First EPA-approved outdoor field trial for genetically engineered algae Science Daily "Just as agricultural experts for decades have used targeted genetic engineering to produce robust food crops that provide human food security, this study is the first step to demonstrate that we can do the same with genetically engineered algae," said ... |
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First EPA-approved outdoor field trial for genetically engineered algae - Science Daily
"During acute infection, HIV actively replicates," explained co-senior study investigator Kamel Khalili, Ph.D., professor and chair of the department of neuroscience at LKSOM. "With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection." The excision efficiency of their strategy reached 96% in EcoHIV mice, providing the first evidence for HIV-1 eradication by prophylactic treatment with a CRISPR/Cas9 system.
In the third animal model, a latent HIV-1 infection was recapitulated in humanized mice engrafted with human immune cells, including T cells, followed by HIV-1 infection. "These animals carry latent HIV in the genomes of human T cells, where the virus can escape detection, Dr. Hu explained. Amazingly, after a single treatment with CRISPR/Cas9, viral fragments were successfully excised from latently infected human cells embedded in mouse tissues and organs.
In all three animal models, the researchers employed a recombinant adeno-associated viral (rAAV) vector delivery system based on a subtype known as AAV-DJ/8. "The AAV-DJ/8 subtype combines multiple serotypes, giving us a broader range of cell targets for the delivery of our CRISPR/Cas9 system," remarked Dr. Hu. Additionally, the researchers re-engineered their previous gene-editing apparatus to now carry a set of four guide RNAs, all designed to efficiently excise integrated HIV-1 DNA from the host cell genome and avoid potential HIV-1 mutational escape.
To determine the success of the strategy, the team measured levels of HIV-1 RNA and used a novel and cleverly designed live bioluminescence imaging system. "The imaging system, developed by Dr. Won-Bin Young while at the University of Pittsburgh, pinpoints the spatial and temporal location of HIV-1-infected cells in the body, allowing us to observe HIV-1 replication in real time and to essentially see HIV-1 reservoirs in latently infected cells and tissues," stated Dr. Khalili.
The researchers were excited by their findings and are optimistic about their next steps. The next stage would be to repeat the study in primates, a more suitable animal model where HIV infection induces disease, in order to further demonstrate the elimination of HIV-1 DNA in latently infected T cells and other sanctuary sites for HIV-1, including brain cells," Dr. Khalili concluded. "Our eventual goal is a clinical trial in human patients."
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CRISPR Eliminates HIV in Live Animals - Genetic Engineering & Biotechnology News
In uncertain times, we are witnessing one of the greatest moments in the history of science.
A projected timeline for treatment and prevention of the novel coronavirus. Although we are living ... [+] through uncertain times, we are also witnessing one of the greatest moments in science history.
Scientists are breaking speed records in their race to develop treatments for the new coronavirus. Some are panning through old molecules hoping to find effective drugs. Others are applying the latest breakthroughs in synthetic biology to engineer sophisticated treatments and vaccines.
Ive previously talked about some synthetic biology companies are racing to create treatments. Others like Mammoth Biosciences are developing much-needed testing. Every day brings additional reports of the latest breakthroughs from around the world. But how can we make sense of all this information?
To provide a big-picture perspective, SynBioBeta and Leaps by Bayer have partnered to help visualize the overall progress of the research community. At the heart of the project is an infographic showing the timeline to the various treatments and preventions (click here to download it). Its based on data from The Milken Institute, which recently released a detailed tracker to monitor the progress of each of the more than 60 known COVID-19 treatments and preventions currently in development.
One takeaway: the progress to develop coronavirus treatments and preventions is moving at an unprecedented pace, with historic records being broken nearly every week.
The crisis response from the global biotech community has been truly inspiring, says Juergen Eckhardt, SVP and Head of Leaps by Bayer, a unit of Bayer AG that leads impact investments into solutions to some of todays biggest challenges in health and agriculture. We are excited to partner on this visual timeline to help a broader audience understand how and when scientific innovation may bring us through this deeply challenging time.
COVID19: Projected timeline for treatment and prevention. Three paths: pre-existing drugs, antibody ... [+] therapies, and vaccines.
There are standard stages to getting a drug approved. In Phase 1 trials, a drugs safety is assessed in a small group of healthy subjects. In later stages (Phase II & III), efficacy is measured in a larger number of people, often versus a placebo. The situation with COVID-19 is predicted to become so dire so quickly, however, that many are looking to fast-track testing. This could include granting experimental drugs expanded access, for compassionate use, which would allow physicians to give them to patients who are critically ill before testing is complete.
The fastest way to safely stop COVID-19 would be to discover that an already-approved medication works against it. Repurposed drugs do not require the same extensive testing as novel medicines and may already be available in large quantities. The Milken Institutes tracker identifies 7 candidate drugs in this category.
One is the malarial medicine chloroquine, which in recent days has been touted by some as a possible miracle drug against the coronavirus. German pharmaceutical company Bayer last week donated three million tablets of chloroquine to the U.S. The FDA and academics are together investigating whether it can provide relief to COVID-19 patients.
There are hundreds if not thousands of other FDA-approved drugs on the market that are already proven safe in humans and that may have treatment potential against COVID-19, so many scientists are rapidly screening the known drug arsenal in hopes of discovering an effective compound.
Antibodies are proteins that are a natural part of the human immune system. They work around the clock in blood to block viruses and more. The problem at the moment is that because the novel coronavirus (known as SARS-CoV-2) is new, no one has had time to develop antibodies against it.No one, that is, except those who have recovered from COVID-19.
Antibodies taken from those people could help patients who are still infected. Such patient-to-patient transfers can be performed without extensive testing or lengthy approval processes so long as standard protocols are followed. It is yet unknown whether this treatment option will work for COVID-19, nor whether there will be enough recovered donors to deal with the infection at scale.
To improve this process, companies like Vancouver, Canada-based AbCellera are applying new biotechnologies.
AbCellera is using proprietary tools and machine learning to rapidly screen through millions of B cells from patients who recovered from COVID-19. B cells are responsible for producing antibodies. The company has announced a partnership with Eli Lilly on this project and aims to bring its hottest antibodies those that neutralize the virus to the clinic.
AbCellera's platform has delivered, with unprecedented speed, by far the world's largest panel of anti-SAR-CoV-2 antibodies," said Carl Hansen, Ph.D., CEO of AbCellera, in a statement. "In 11 days, we've discovered hundreds of antibodies against the SARS-CoV-2 virus responsible for the current outbreak, moved into functional testing with global experts in virology, and signed a co-development agreement with one of the world's leading biopharmaceutical companies. We're deeply impressed with the speed and agility of Lilly's response to this global challenge. Together, our teams are committed to delivering a countermeasure to stop the outbreak."
James Crowe at Vanderbilt University is also sifting through the blood of recovered patients. Using a new instrument called Beacon from a company called Berkeley Lights. Crowes team has been scouring through B cells to find antibodies that neutralize SARS-CoV-2. The technology behind this project was developed in recent years with funds from the Department of Defense.
Normally this would be a five year program, Crowe told me. But in the rapid process his team is following, animal studies could be done in as fast as two months.
This morning, Berkeley Lights announced a Global Emerging Pathogen Antibody Discovery Consortium (GEPAD) to attack COVID-19 and other viruses. It is partnering with Vanderbilt University, La Jolla Institute for Immunology, and Emory University to accelerate the work above to the broader research community.
This collaboration also included commercial partners, including Twist Bioscience, who synthesized DNA for the project.
Our mission is to provide the raw material needed for biologists to make breakthroughs, said Twists CEO Emily Leproust. If DNA is needed, we want to make it, quickly and perfectly
Another company that specializes in DNA synthesis, SGI-DNA, is offering its tools at much reduced cost to researchers developing COVID-19 treatments. The company said that people from around the world are coming to them for help.
"There is zero time to waste," said Todd R. Nelson, Ph.D., CEO of SGI-DNA. He said that researchers need synthetic DNA and RNA, which its Bio-XP machine can provide in as little as eight hours.
Nelson continued, "In a matter of a day or two, we have built the genes thought to be critical to the development of successful vaccines against SARS-CoV-2. SGI-DNA has made them available in the form of different genetic libraries, which researchers can use to find druggable targets in a matter of hours, dramatically accelerating the time to market for therapeutics and vaccines.
Beyond searching for antibodies in recovered patients, biotechnologists have other tricks up their sleeves.
One approach involves genetically engineering laboratory mice to mimic the human immune system. These animals can then be presented with the virus or parts of the virus and allowed to recover. The hope is that their B cells would then produce effective antibodies. Because this happens in a controlled setting, biologists can better understand and engineer the process.
A company called GenScript was pursuing this strategy as early as February 4,
when police escorted 8 transgenic mice immunized with the 2019 nCoV antigen to research labs in China. In 12 hours, its researchers successfully found specific antibodies in the mice that could recognize the novel virus and potentially block it from binding to cells. In less than 24 hoursagain using Berkeley Lights new Beacon instrument for working with thousands of individual, live cellsGenScript completed a series of steps that would have taken three months using previous technology.
Yet another approach involves computational approaches and artificial intelligence. Firms like Distributed Bio are using computers to reengineer antibodies to better target SARS-CoV-2. The company is optimizing antibodies that are known to target SARS-CoV-1, the virus behind the 2003 outbreak of SARS.
We believe broadly neutralizing antibodies with engineered biophysical properties will become key weapons to win the war against all coronaviruses said Jake Glanville, CEO of Distributed Bio.
Vaccines work by simulating infection, which allows the body to mount its own defense against a virus. Effective vaccines take time to develop, and they can take even longer to test. But recent progress in biotechnology is again accelerating these efforts.
Notably, Moderna has launched a Phase 1 vaccine trial against COVID-19 in record time. Patients in Seattle have already begun receiving injections of an experimental mRNA vaccine. Moderna cranked out doses of this and won approval from the FDA for testing in just 44 days an all-time record.
These programs show a massive focus on a common enemy, and a coming together of disparate firms.
Ginkgo Bioworks, a giant in the emerging field of synthetic biology, has announced a $25 million fund to help spur even more collaboration. The company is offering its laboratory equipment and know-how to anyone with a good idea of how to stop COVID-19. We dont want any scientists to have to wait. The pandemic has already arrived, so the time for rapid prototyping and scale-up is right now, said Jason Kelly, CEO of Ginkgo.
These effortsand the infographic aboveshould give you hope. Although we are all now living in uncertain times, we are also witnessing one of the greatest moments in the history of science.
It's a terrible time, and simultaneously a fantastic time to see the global science community working together to conquer this very hard and challenging disease, said Berkeley Lights CEO Eric Hobbs. We are also learning and developing the tools and technologies to ensure that we can react faster to the next threat, so that we don't get to this point again in the future.
Follow me on twitter at @johncumbers and @synbiobeta. Subscribe to my weekly newsletters in synthetic biology.
Thank you to Ian Haydon and Kevin Costa for additional research and reporting in this article. Im the founder of SynBioBeta, and some of the companies that I write aboutincluding Leaps by Bayer, Mammoth Biosciences, Distributed Bio, Twist Bioscience, SGI-DNA, Genscript, Berkeley Lights, and Ginkgo Bioworksare sponsors of the SynBioBeta conference and weekly digest heres the full list of SynBioBeta sponsors.
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Timeline Shows 3 Paths To COVID-19 Treatment And Prevention (INFOGRAPHIC) - Forbes
An international research team has identified a candidate thinness gene that could help to explain why some people can seemingly stay slim no matter what they eat. A genetic association study that analyzed data from more than 47,000 people in an Estonian biobank implicated ALK as a key gene that may regulate thinness and play a role in resisting weight gain in metabolically healthy thin people. Further studies in animal models showed that deleting ALK resulted in thinner flies and thinner mice, and demonstrated that ALK expression in the brain may be involved in regulating energy expenditure.
ALK is already a recognized anticancer target, and the researchers suggest that targeting the gene may represent a future therapeutic strategy against obesity. If you think about it, its realistic that we could shut down ALK and reduce ALK function to see if we did stay skinny, said Josef Penninger, PhD, director of the Life Sciences Institute and professor of the department of medical genetics at the University of British Columbia. ALK inhibitors are used in cancer treatments already. Its targetable. We could possibly inhibit ALK, and we actually will try to do this in the future. Penninger is senior author of the teams published paper in Cell, which is titled, Identification of ALK in Thinness. The reported studies involved a multidisciplinary team of researchers in Austria, Switzerland, Estonia, China, Australia, Canada, and Sweden, and the U.S.
Theres considerable variability in how susceptible different people are to putting on weight. We all know these people: its around one percent of the population, said Penninger. They can eat whatever they want and be metabolically healthy. They eat a lot, they dont do squats all the time, but they just dont gain weight.
Body mass index (BMI), which is commonly used to classify weight categories, is a highly complex trait that is impacted by genes and environmental cues, the researchers wrote. And while more than 700 common single nucleotide polymorphisms (SNPs) have been linked with BMI, only a limited number of genes involved in regulating human body weight have been identified and validated. To date, most studies have focused on susceptibility to obesity, and only a few have looked at the genetic basis of thinness in humans or animal models. Everybody studies obesity and the genetics of obesity, Penninger pointed out. We thought, Lets just turn it around and start a new research field. Lets study thinness.
To do this Penningers team analyzed data from the Estonian Biobank, which includes 47,102 people aged 2044 years. The investigators carried out a genome-wide association study (GWAS) to compare the DNA samples and clinical data of healthy thin individualswho were in the lowest 6th percentilewith normal-weight individuals, in the search for genetic variants linked with thinness. Their results highlighted genetic variants in the ALK gene that were specific to the thin individuals.
Scientists have known that the ALK gene frequently mutates in various types of cancer, and while it is viewed as an oncogene that can drive the development of tumors, the role of ALK outside of cancer isnt understood. ALK has been extensively studied in cancer, but little is known about the biological role of ALK outside the context of cancer, they wrote. The new finding suggested that the ALK gene might play a role as a thinness gene involved in weight-gain resistance.
The researchers investigated the association between ALK and thinness through a series of studies in Drosophila fruit flies, and in mice. Their experiments demonstrated that mice in which the ALK gene was knocked out remained thin and were resistant to diet-induced obesity. Intriguingly, Alk knockout mice were significantly protected against HFD-induced obesity, the researchers wrote. ALK deficiency was also linked with reduced weight gain in a genetic obesity mouse model. Even when the ALK knockout mice had the same diet and activity levels as normal mice, they still demonstrated lower body weight and body fat from an early age, which persisted into adulthood.
Further studies in mice suggested that ALK, which is highly expressed in the brain, plays a role in instructing the fat tissues to burn more fat from food. Expression analysis revealed high Alk mRNA levels in the hypothalamus, especially in the PVN, which is also true for humans, the investigators wrote. Mechanistically, we found that ALK expression in hypothalamic neurons controls energy expenditure via sympathetic control of adipose tissue lipolysis Our genetic and mechanistic experiments identify ALK as a thinness gene, which is involved in the resistance to weight gain.
The findings could help scientists develop therapeutics against ALK as a future strategy against obesity. The team also plans to further study how neurons that express ALK regulate the brain at a molecular level to balance metabolism and promote thinness.
The Estonian Biobank that the team studied was ideal because of its wide age range and its strong phenotype data. We took advantage of the wide age range of the unique Estonian biobank recruitment as well as its strong phenotypic datasets, making ECGUT [Estonian Genome Center of the University of Tartu] an ideal starting point to identify potential variants and genes playing a role in thinness, the scientists noted. Even so, one limitation for replicating these findings is that biobanks that collect biological or medical data and tissue samples dont have a universal standard in data collection, which makes comparability a challenge. The researchers say they will need to confirm their findings with other data banks through meta-analyses. You learn a lot from biobanks, said Penninger. But, like everything, its not the ultimate answer to life, but theyre the starting points and very good points for confirmation, very important links and associations to human health.
The team suggests its work is unique in its combination of populationand genome-wide-scale analyses into the genetic basis of thinness, with in vivo analyses of gene function in mice and flies. Its great to bring together different groups, from nutrition to biobanking, to hardcore mouse and fly genetics, stated Penninger. Together, this is one story including evolutionary trees in metabolism, the evolutionary role of ALK, human evidence, and hardcore biochemistry and genetics to provide causal evidence.
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Gene for "Thinness" Identified that May Help to Resist Weight Gain - Genetic Engineering & Biotechnology News
French startup Vivet Therapeutics raised 37.5 million (about $41 million) in a Series A round of financing to support the development of gene therapies for rare inherited metabolic diseases. The firm was set up in 2016 to develop treatments based on adeno-associated virus (AAV) vector technology licensed exclusively from its close collaborator, the Fundacin para la Investigacin Mdica Aplicada (FIMA), at the Center for Applied Medical Research (CIMA) in Pamplona, Spain, and from Massachusetts Eye and Ear (MEE) in Boston.
Novartis Venture Fund and Columbus Venture Partners led the Series A investment round. Roche Venture Fund, HealthCap, Kurma Partners, and Ysios Capital also participated.Florent Gros, managing director at Novartis Venture Fund, commented, "We have searched extensively for next-generation AAV technologies and clinical applications. We are very excited by Vivet Therapeutics' clinical and commercial prospects; the company has outstanding management, assets, and capabilities."
Based in Paris, and with a wholly owned subsidiary in Spain, Vivetaims to develop gene therapies targeting disorders including Wilson disease, progressive familial intrahepatic cholestasis (PFIC), and citrullinemia.The firm is usinga novel, synthetic AAV, AAV-Anc80, to introduce genes into hepatocytes.Lead Wilson disease gene therapy program VTX801 comprises a truncated, functional version of the defective ATP7B gene, delivered directly into liver cells using the AAV vector technology. First-in-human trials with VTX801 are projected to start by the end of 2018.
Jean-Philippe Combal, Pharm.D., Ph.D., Vivet co-founder and CEO, noted, Early results from preclinical studies with VTX801 are very promising, and we are now well funded to advance this candidate into the clinic, while developing our portfolio and technologies."
Vivet's co-founders includeCombal (ex-Gensight Biologics, Sanofi),Jens Kurth, Ph.D. (ex-Anokion, Novartis), and Gloria Gonzlez-Aseguinolaza, Ph.D. (CIMA, University of Navarra).On announcement of Series A fundraising, Gloria Gonzlez-Aseguinolaza, Vivet CSO, said, By collaborating with leading institutions such as CIMA in Spain and MEE in the United States, Vivet has secured superior and novel gene therapy technologies and liver disease expertise. We believe these capabilities, combined with the international development expertise of the management team, create a company with very exciting prospects."
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Vivet Raises 37.5M to Develop Gene Therapies for Rare Liver Diseases - Genetic Engineering & Biotechnology News
SAN JOSE, Calif., Feb. 18, 2020 /PRNewswire/ --Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening bacterial infections, announced today that it will host an Investor Day on Thursday, March 12th, 2020 in New York City from 12:00PM-2:00PM EST.
The event will provide development updates on the Company's key clinical programs and feature key opinion leader (KOL) panels moderated by leading healthcare analysts, Louise Chen of Cantor Fitzgerald and Jason McCarthy from Maxim Group. Participating experts include Dr. Steven Opal of Brown University and Dr. Lisa Saiman of Columbia University who will provide insights on the acute pneumonia and cystic fibrosis indications and the current therapeutic landscape. Medical reimbursement will also be discussed.
In addition to the KOL panel discussion, the forum is intended to provide investors and analysts with a comprehensive profile of APEX, the Company's cutting-edge antibody discovery and production platform along with an update on lead programs AR-301 for the treatment of ventilator associated pneumonia (VAP) and AR-501, an inhalable therapy for chronic lung infections in cystic fibrosis patients. Aridis is advancing AR-301 in a global clinical trial which remains on track to report top line data in 1H 2021, and enrolling AR-501's Phase 1/2a clinical trial with top-line data expected in 1H 2020 (healthy subjects), and in 2H 2021 (cystic fibrosis subjects).
To learn more about the event or to register for attendance, please email RSVP@aridispharma.com.
About Aridis Pharmaceuticals, Inc.Aridis Pharmaceuticals, Inc. discovers and develops anti-infectives to be used as add-on treatments to standard-of-care antibiotics. The Company is utilizing its proprietary APEX and MabIgX technology platforms to rapidly identify rare, potent antibody-producing B-cells from patients who have successfully overcome an infection, and to manufacture mAbs for therapeutic treatment of critical infections. These mAbs are already of human origin and functionally optimized for high potency by the donor's immune system; hence, they do not require genetic engineering or further optimization to achieve full functionality.
The Company has generated multiple clinical stage mAbs targeting bacteria that cause life-threatening infections such as VAPand HAP. The use of mAbs as anti-infective treatments represents an innovative therapeutic approach that harnesses the human immune system to fight infections and is designed to overcome the deficiencies associated with the current standard of care which is broad spectrum antibiotics. Such deficiencies include, but are not limited to, increasing drug resistance, short duration of efficacy, disruption of the normal flora of the human microbiome and lack of differentiation among current treatments. The mAb portfolio is complemented by a non-antibiotic novel mechanism small molecule anti-infective candidate being developed to treat lung infections in cystic fibrosis patients. The company's pipeline is highlighted below:
Aridis'Pipeline
AR-301(VAP).AR-301 is a fully human immunoglobulin 1, or IgG1, mAb currently in Phase 3 clinical development targeting gram-positiveS. aureusalpha-toxin in VAPpatients.
AR-101(HAP).AR-101 is a fully human immunoglobulin M, or IgM, mAb targetingP. aeruginosaliposaccharides serotype O11, which accounts for approximately 22% of allP. aeruginosahospital acquired pneumonia cases worldwide.
AR-501(cystic fibrosis).AR-501 is an inhaled formulation of gallium citrate with broad-spectrum anti-infective activity being developed to treat chronic lung infections in cystic fibrosis patients. This program is currently in a Phase 1/2a clinical study in healthy volunteers and CF patients.
AR-401(blood stream infections).AR-401 is a fully human mAb preclinical program aimed at treating infections caused by gram-negativeAcinetobacter baumannii.
AR-201(RSV infection). AR-201 is a fully human IgG1 mAb preclinical program aimed at neutralizing diverse clinical isolates of respiratory syncytial virus (RSV).
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For additional information on Aridis Pharmaceuticals, please visithttps://aridispharma.com/.
Forward-Looking StatementsCertain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. These statements may be identified by the use of words such as "anticipate," "believe," "forecast," "estimated" and "intend" or other similar terms or expressions that concern Aridis' expectations, strategy, plans or intentions. These forward-looking statements are based on Aridis' current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the timing of regulatory submissions, Aridis' ability to obtain and maintain regulatory approval of its existing product candidates and any other product candidates it may develop, approvals for clinical trials may be delayed or withheld by regulatory agencies, risks relating to the timing and costs of clinical trials, risks associated with obtaining funding from third parties, management and employee operations and execution risks, loss of key personnel, competition, risks related to market acceptance of products, intellectual property risks, risks associated with the uncertainty of future financial results, Aridis' ability to attract collaborators and partners and risks associated with Aridis' reliance on third party organizations. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Aridis' 10-K for the year ended December 31, 2018and Aridis' other filings made with the Securities and Exchange Commission.Forward-looking statements included herein are made as of the date hereof, and Aridis does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.
Contact:
Investor RelationsJason WongBlueprint Life Science Groupjwong@bplifescience.com(415) 375-3340 Ext. 4
Aridis Pharmaceuticals, Inc. Logo (PRNewsfoto/Aridis Pharmaceuticals, Inc.)
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One morning in 2005, Shelley Beverley woke up to find that she had gone deaf. She was 21, and living in Johannesburg with her older brother Neil. I was very scared, she says. It was just so sudden. She struggled through the rest of the day, hoping that her hearing would come back, but it didnt. In one sense, her hearing loss wasnt entirely a surprise: Beverleys grandmother had been deaf, Neil had lost his hearing when he was 13, and her mum, Mary, had lost hers when she was 32. We knew it ran in the family, she says, but I thought Id been lucky and not inherited it.
Beverley, 35, lives in Margate, a semi-rural district south of Hobart, with her husband James. The couple migrated to Australia from South Africa in 2010, looking for space, buying 2 hectares of lush green grass at the foot of a forested ridge near the mouth of the Derwent River. We love the wildlife here, says James, looking out the living room window. Weve seen pademelons, echidnas, quolls, blue-tongue lizards, even a Tassie devil. At dusk, hundreds of kangaroos emerge from the forest to gorge on the grass. Its very peaceful, says James. Its really helped us after everything thats happened.
Apart from their deafness, Beverleys family had largely enjoyed good health. Then, in September 2015, her mother, Mary, then 62, started experiencing fatigue and stomach pain. Doctors in Durban ordered a colonoscopy, but the procedure made her worse. Her feet became swollen and purple. Because of their hearing problems, Shelley and Mary had communicated mainly in text messages. But soon I began noticing that her wording got a bit funny, says Beverley. It didnt always make sense.
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Beverley flew to Durban in February 2016, but by that time her mother could no longer talk or walk. She was so weak that she couldnt move her hands or lift her neck. Two days after Beverley arrived in Durban, her mother caught a virus that caused fluid to build up on her lungs. The doctors tried unsuccessfully to drain it. Shortly afterwards, she died. She weighed just 36 kilograms. It was so fast, Beverley says. And we were still in the dark about what she had.
Shortly before Marys death, Neil had also fallen ill. He developed a number of mysterious symptoms, including facial twitches and seizures. He kept falling over and tripping, and experienced vomiting and headaches so severe he lost his vision for weeks at a time. His behaviour became strange showering with his clothes on, and hallucinating.
One day, Dad was driving him around and Neil started talking to all these little people he thought were around his feet, says Beverley. Doctors in Durban had trouble diagnosing him, so they sent a biopsy to London, where he was found to have a type of mitochondrial cytopathy one of a large family of chronic and progressive diseases that affect the muscles, brain and nervous system. As the family soon learnt, the condition has no cure and no effective therapies. One of the common early symptoms is hearing loss.
Neil died in June 2017, aged 34, by which time Beverley had discovered she also had the condition. It was fear, so much fear, she says. She began experiencing symptoms, including migraines and vision loss. She has since developed diabetes, hypertension, gastro-paresis (when your stomach muscles dont work), and pharyngeal dysphagia (difficulty swallowing). Every time I get sick now, the flu or something, I think, When am I going to need a wheelchair or a feeding tube? When will my legs stop working?
Mito has taken everything from me, she says. If I die, at least James will still have a part of me.
Beverley has bright blue eyes and long, straight, ash-brown hair. Shes got a lazy left eye and uncommonly pale skin, which she attributes to her condition. Oh, and I had bunions out in 2010, she says, laughing wryly.
She doesnt know how long shes got left, but she is determined to make it count. She has joined mito awareness groups, and is an active member of the Mito Foundation, which supports sufferers, and funds research. She has exhaustively researched the condition and takes every opportunity to educate doctors. Youd be surprised by how little they know about it, she says.
But her overriding focus has been on a cutting-edge, and currently illegal, procedure called mitochondrial donation, a form of IVF which would allow those with the condition to have children, safe in the knowledge they would not be passing it on. Mito has taken everything from me, she says. If I die, at least James will still have a part of me. I would like him to look at our child, and say, You have your mums smile or your mums eyes.
An IVF treatment known as mitochondrial donation could potentially save up to 60 Australian children a year from being born with the condition. Credit:
Mitochondrial donation has been labelled immoral and unethical, a slippery slope to designer babies, not to mention potentially unsafe. The only country in the world to have legalised it is the UK. A report by medical experts into the technologys potential application in Australia is due to be delivered to Health Minister Greg Hunt this month.
This fight is really personal to me, Beverley says. Short of a cure, people with mito should at least have the option of having healthy children.
Mitochondria are microscopic structures in human cells that provide the body with energy. For this reason, they are often described as the cells powerhouse. They are crucially important: if your mitochondria fail or mutate, your body will be starved of energy, causing multiple organ failure and premature death.
A stylised representation of a mitochondrion, which provides the body with energy. Malfunction can lead to organ failure and death.Credit:Josh Robenstone
Mito, which is maternally inherited, usually affects the muscles and major organs such as the brain, heart, liver, inner ears, and eyes. But it can cause any symptom in any organ, at any age. Indeed, the term mito includes more than 200 disorders, the symptoms of which are maddeningly varied and seemingly unrelated, leading to delayed diagnoses or incorrect diagnoses or, indeed, no diagnosis.
Many of these people have been fobbed off by doctors or laughed off by people who think they are hypochondriacs, says Dr David Thorburn, a mitochondrial researcher at the Murdoch Childrens Research Institute, in Melbourne, who has diagnosed some 700 cases over the past 28 years. Most people are relieved to finally know what it is, because that is the end of that part of their journey.
Its sometimes said babies produced as a result of mitochondrial donation would have three parents the mother, the father, and the donor.
Up to two million people worldwide have some form of mito. - Others, like Beverley, who have a less severe type of the disease, will get adult onset, and can expect to become ill in their 30s, 40s or 50s.
According to Thorburn, One of the things that most dismays families with mito is the lack of control they have over passing the condition down to future generations of their family.
Remaining childless is one way to stop the condition from being passed down, as is adopting, but as Thorburn acknowledges, There is an innate desire in many individuals to have their own children. For these people, mito donation offers the very real prospect that the condition is eliminated from future generations.
Mitochondrial replacement is a highly specialised procedure, requiring a level of manual dexterity sufficient to manipulate a womans egg, which is roughly the width of a human hair. Within that egg is a nucleus, where a persons genes are located, and the cytoplasm, the jelly-like substance that surrounds it. Mitochondria are found in the cytoplasm.
Mitochondrial replacement involves taking a donor females healthy egg, removing its nucleus and replacing it with the nucleus of the woman affected by mitochondrial disease, but whose nucleus is healthy. The egg is then fertilised using her pa
rtners sperm. (Another option is to fertilise the egg first, and then swap the nucleus.) The resulting embryo is then implanted into the mother.
Researcher David Thorburn: "Mito donation offers the very real prospect that the condition is eliminated from future generations."Credit:Josh Robenstone
Since more than 99.9 per cent of our genes are found in the eggs nucleus, which remains unaffected, the procedure will have no impact on the childs height, hair colour or mannerisms. Despite that, its sometimes said that babies produced as a result of mitochondrial donation would have three parents the mother, the father, and the donor.
The technology has been tested in mice for more than 30 years, but only since 2009 has research been done on human embryos, mainly in the UK. Almost from the start, the research was subject to sensational headlines about scientists playing God, and the possibility of genetic engineering, with much of the hysteria being fuelled by anti-abortion groups. The Catholic Church described it as a further step in commodification of the human embryo and a failure to respect new individual human lives.
In 2012, the Human Genetics Alert, an independent watchdog group in London, wrote a paper comparing any baby produced with mitochondrial replacement to Frankensteins creation, since they would be produced by sticking together bits from many different bodies. According to the Conservative British MP Jacob Rees-Mogg, the procedure was not a cure for disease, it is the creating of a different person.
Regulators subjected the technology to four separate scientific reviews, together with rounds of ethical debate and community consultation. In 2015, the UK Parliament voted to legalise the technology for use in humans, on the proviso that it only be available to those women at high risk of passing on the disease. Since then, 13 couples in the UK have received the go-ahead to undergo the procedure.
Its unclear how many children, if any, have been born: the parents have asked that details not be published. Meanwhile, scientists like Thorburn wait eagerly for news of any developments. I know the UK researchers well and have asked several of them, and they are keeping completely quiet about it in respecting the families wishes, he says.
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If there have been babies born in the UK using the procedure, they arent the first. In April 2016, a child was born using the technique in Mexico, to a Jordanian mother who carried a fatal mitochondrial condition known as Leigh syndrome. The doctor in charge, an American fertility specialist called Dr John Zhang, later admitted that he had gone to Mexico because the procedure is illegal in America. In Mexico, he admitted, There are no rules.
Even those who want mitochondrial donation legalised in Australia concede that much remains unknown about the procedure. Its long-term risks can only be understood through lifelong health check-ups, but this is impossible until any children conceived via this procedure become adults. Implications for subsequent generations also remain unclear.
No medical procedure is 100 per cent safe, says Sean Murray, CEO of the Mito Foundation. But we think we are at the stage now where the benefits of the technology are greater than the risks.
One of the issues around safety concerns the compatibility of the donors mitochondria with the recipients nuclear genes. A 2016 study in mice suggested that mismatched mitochondria affected their metabolism and shortened their lives. Another concern is known as carryover, whereby a tiny amount of mutant mitochondria is inevitably transferred from the affected mothers egg into the donor egg during the procedure.
Instead of it being wiped out, the mutation might then reappear in the descendants of any girls born as a result. For this reason, some people have proposed that the procedure be restricted to male embryos only, but this raises all kinds of ethical issues around selective breeding and sex selection.
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Indeed, it often seems as if the term ethical minefield was coined especially with mitochondrial donation in mind.
My primary ethical concern has to do with the sanctity of human life, says Father Kevin McGovern, a Catholic priest and member of the National Health and Medical Research Councils Mitochondrial Donation Expert Working Committee.
If mitochondrial donation is permitted here, the technique most likely to be used is pronuclear transfer, which requires that both the donors egg and the affected mothers egg be fertilised. [This is to ensure that both eggs are at the same developmental stage.] But once the nucleus is removed from the donors fertilised egg, it is discarded. For people who believe that life begins at conception, this is akin to murder. You are creating two lives and destroying one for spare parts.
The Catholic Church has consistently opposed mitochondrial donation. In a Senate inquiry into the technology in 2018, Dr Bernadette Tobin, director of the Plunkett Centre for Ethics at the Australian Catholic University, suggested the process was intrinsically evil.
The inquiry also heard from Father Anthony Fisher, Catholic Archbishop of Sydney, who raised concerns about the moral right of the child to know how he or she was conceived the problem of what he called genealogical bewilderment and the donors right to remain anonymous. He also worried that women might effectively become egg vending machines: The availability of human ova is often assumed when people talk about reproductive technology as if they were somehow there in a cupboard to be used. In fact, it means women have to be used to obtain these eggs. They are extracted by invasive procedures that do carry some risk.
A report by medical experts into mitochondrial donation and its potential application in Australia is due to be delivered to Health Minister Greg Hunt this month. Credit:Alex Ellinghausen
Equally troubling for the Australian Catholic Bishops Conference, the peak national body for the churchs bishops, was the fact that mitochondrial donation involved conceiving babies not by marital intercourse [but by] a technical procedure.
Most of these concerns are redundant, argues the Mito Foundations Sean Murray. We already have a well defined regulatory framework for dealing with all this, he says. As far as the donors right to remain anonymous, we would defer to the appropriate federal or state and territory regulations that apply for sperm or egg donations. In regard to a kids right to know they had a mitochondrial donor, societally there seems to be a preference to inform kids. Its important for them to understand their genetic lineage.
Then theres the matter of consent. The parents can wrestle with the ethical issues and weigh up all the risks, but the only person who cant consent to the procedure is the unborn child. Well, says Murray, they cant consent to being born with mito, either.
The Mito Foundations Sean Murray: "In regard to a kids right to know they had a mitochondrial donor, societally there seems to be a preference to inform kids."Credit:Joshua Morris
Murray, 47, is one of the founding directors of the Mito Foundation, which was established in Sydney in 2009. Mito runs in my family, he says. My older brother, Peter, died of it in 2009 at 45, and my mum passed away in 2011, at 70. What people often dont understand is that even in families that have mito, each member can have different mutational loads basically, different amounts of bad mitochondria. Peter got a high load, but I didnt. Thats why Im still here.
A computer scientist by training, Murray now works full-time on the foundation. Much of his job involves travelling around the country, explaining mito to politicians, journalists and philanthropists, raising funds for research and, most crucially, advocating for a change to the laws.
Mitochondrial donation falls foul of two pieces of legislation: the Research Involving Human Embryos Act 2002, and the Prohibition of Human Cloning for Reproduction Act 2002. The laws p
rohibit the implantation of a human embryo that contains more than two peoples genetic material. The laws were subject to a mandatory review in 2010, but the then Labor government recommended they remain the same.
In 2013, the Mito Foundation urged the government to revisit its decision. Two years later, it began lobbying in earnest. What we tried to get across was that the science around mito donation has come a long way since 2010, says Murray. Also, the process that the UK went through to legalise it really reassured us that the procedure is safe and effective.
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In the past five years, Murray and his colleagues have consulted with more than 100 MPs and senators. Only one of them, according to Murray, said I dont like this. They have also talked to dozens of industry experts, including academics and medical and research bodies, about the benefits of mitochondrial donation. Most of them get it straight away, he says. We are talking about a technique that will prevent the chance of having a morbidly ill child.
Now, a breakthrough appears imminent. In February 2019, Health Minister Greg Hunt asked the National Health and Medical Research Council to look into the matter, review the science and conduct public consultation. The NHMRC is due to hand its report to Hunt this month. The expectation among the mito community is that he will recommend the laws be changed. Any proposals would then need to be debated in Parliament, where issues around reproductive medicine have, in the past, been hotly contested.
Murray expects some opposition from more conservative MPs, but nothing like the rancour seen in the NSW Parliament during last years debate over legalising abortion. Shadow health minister Chris Bowen has, for his part, said that Labor will support changing the laws.
Mitochondrial sufferer Shelley Beverley at home in Tasmania. This fight is really personal to me. Credit:Peter Mathew
Whether this will help people like Shelley Beverley is unclear. If Hunt gives it the green light, it will take two years at least for mitochondrial donation to become available to prospective parents, given the time involved in drafting and passing legislation, establishing a regulatory regime and getting doctors up to speed with the technology.
This will probably be too late for Beverley. I really only have about a year left to give it a go, she tells me. After that, my symptoms may progress and biologically things get worse after 35. She says she would consider going to the UK for the treatment, but that at present they are not accepting international patients.
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In the meantime, she watches TV, and reads a little, but not too much. (It puts me to sleep.) She gardens: she has a bed of huge white and pink roses out the back of her house, as a memorial to her mother and brother. And she eats. James cooks for me. He lets me choose the best meat and potatoes! Ive put on weight since I met him. She describes James as something close to an angel. He will listen to every problem I have or feeling I experience. He will always put me first.
Beverley started going out with James when she was 21, right around the time she first went deaf. I was so scared that he wouldnt like me as much. I remember calling him and saying I was scared he would leave me. But James is still here. Im very lucky to have him, she says. If I go, I want him to have a part of me.
To read more from Good Weekend magazine, visit our page at The Sydney Morning Herald, The Age and Brisbane Times.
Tim Elliott is a senior writer with Good Weekend.
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How far should genetic engineering go to allow this couple to have a healthy baby? - Brisbane Times
CAMBRIDGE, Mass.(GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, is presenting new data from two of its development programs at Keystone Symposias Engineering the Genome Conference, a joint meeting with the Emerging Cellular Therapies: Cancer and Beyond Conference, taking place Feb. 8-12, 2020, in Banff, Canada. Intellia researchers are presenting data in support of the companys lead engineered cell therapy development candidate, NTLA-5001 for the treatment of the hematological cancer, acute myeloid leukemia (AML). Intellia also is sharing preclinical results for its hereditary angioedema (HAE) program, which is Intellias third CRISPR/Cas9 development program, announced in January 2020.
Intellia continues to demonstrate strong progress across both our engineered cell therapy and in vivo pipelines, said Intellia President and Chief Executive Officer John Leonard, M.D. We are observing very favorable preclinical data with our engineered T cells, and we are moving ahead with IND-enabling studies and manufacturing for NTLA-5001, to enable a regulatory submission in the first half of 2021.
On the in vivo side, the data from our HAE development program reinforce the modularity of Intellia's non-viral delivery genome editing platform and how it is enabling independent, single-dose therapies for multiple monogenic diseases. For HAE, we expect to nominate a development candidate in the first half of this year, continued Dr. Leonard.
New Data from Intellias Engineered Cell Therapy Development Program for AML
NTLA-5001, which is Intellias first engineered T cell therapy development candidate and is wholly owned, utilizes a T cell receptor (TCR)-directed approach to target the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. The companys WT1-TCR T cell approach aims to develop a broadly applicable treatment for AML patients, regardless of mutational background of a patients leukemia.
The company is presenting data demonstrating that the selection of a natural, high-affinity TCR, in combination with CRISPR-enabled engineering and targeted insertion, results in an engineered T cell capable of specific and potent killing of primary AML blasts. Todays presentation at Keystone builds on data previously presented last fall at the Annual Congress of the European Society of Gene and Cell Therapy (ESGCT).
The data being presented at the Keystone conference substantiate the advantages that a homogeneous T cell product developed through CRISPR engineering, like NTLA-5001, may have over traditional T cell engineering approaches. In particular, traditional T cell engineering methods typically result in a T cell product that carries two different TCRs, one endogenous and one transferred, which can pair in various combinations of alpha and beta chains and form mixed TCRs with unknown specificities. Intellia researchers are sharing today that the precise replacement of the endogenous TCR with the transgenic TCR (tgTCR) resulted in T cells with improved tgTCR expression levels and in 95% of edited T cells carrying exclusively the desired pairs of the tgTCR alpha and beta chains. This therapeutic TCR profile is expected to yield improved T cell product homogeneity, as researchers showed that Intellias T cell editing approach results in superior function of the engineered T cells toward WT1-positive targets in vitro. This therapeutic TCR profile is also expected to result in lower reactivity against unwanted targets on normal tissues that could lead to toxicities, including graft-versus-host disease (GvHD).
Researchers identified that the selected lead WT1 TCR exhibits high avidity (in the nM range) to its target epitope and shows tight epitope specificity. Being a natural TCR isolated from a healthy donor, it may have a lower cross-reactivity risk than many affinity-matured TCRs. Cells engineered with Intellia's lead WT1 TCR also demonstrated no detectable cytotoxicity toward bone marrow CD34+ cells, which express WT1 at low levels. This is an advantage over current CAR-T cell approaches targeting CD33 or CD123 in AML, which have been shown to induce severe bone marrow toxicity.
Furthermore, the data demonstrate that specific and potent killing of WT1-positive primary AML blasts result from T cells expressing Intellias lead WT1 TCR when cocultured in vitro. This outcome was observed across multiple patient samples that harbor the frequent HLA-A*02:01 allele and that express different WT1 levels as well as AML characteristics. These data validate that the epitope targeted by the lead WT1 TCR, which is distinct from a previously evaluated RMF epitope, is presented efficiently and broadly by AML tumor cells that carry the correct human leukocyte antigen (HLA) restriction. Intellias lead WT1 TCR also has the potential to target WT1-positive solid tumors, such as ovarian cancer, glioblastoma, lung cancer and mesothelioma.
The company plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the first half of 2021 for NTLA-5001 for the treatment of AML. Details on todays presentations on WT1 TCR T cells, including data from ongoing collaborations with researchers at IRCCS Ospedale San Raffaele, Milan, at Keystone are as follows:
First Data Presented on Potential CRISPR/Cas9-Based Therapy for HAE, Intellias Third Development Program
Researchers presented yesterday at the Keystone conference the companys first dataset in support of Intellias development program for HAE. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. The disease is caused by increased levels of the bradykinin protein. Most patients with HAE have a C1 esterase inhibitor (C1-INH) protein deficiency, which normally prevents the unregulated release and buildup of bradykinin.
Intellias HAE treatment hypothesis involves knocking out the kallikrein B1 (KLKB1) gene to reduce kallikrein activity, which is involved in the biological pathway for release of bradykinin. Intellia expects this reduction to correlate with a decrease in bradykinin activity, thus, preventing the activation of endothelial cells that causes vascular leakage and angioedema in HAE patients. The data presented at the Keystone conference showed that the knockout of KLKB1 produces in non-human primates (NHPs) a 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates (Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least five months in an ongoing NHP study, in a highly reproducible manner observed across both rodent and NHP studies.
Similar to its lead in vivo program, for the treatment of transthyretin amyloidosis (ATTR), Intellias potential HAE therapy utilizes the companys modular non-viral lipid nanoparticle (LNP) system to deliver CRISPR/Cas9. Intellias proprietary LNP-based delivery system includes two basic components: Cas9 messenger RNA (mRNA) and a guide RNA (gRNA). The gRNA is the only variable portion of the LNP delivery system and is the sole component that needs to be changed from the LNP-based delivery system that forms the foundation of NTLA-2001, Intellias development candidate for the treatment of ATTR for which the company intends to submit an IND application in mid-2020.
Intellia continues to evaluate several potential guide RNAs and expects to nominate a development candidate for HAE in the first half of 2020. Intellias KLKB1 HAE program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party.
Yesterdays short talk, titled In Vivo Delivery of CRISPR/Cas9 to the Liver Using Lipid Nanoparticles Enables Gene Knockout Across Multiple Targets in Rodent and Non-Human Primates, was made
by Jessica Seitzer, director, genomics, Intellia. These data included results from ongoing collaborations with researchers at Regeneron.
All of Intellias presentations can be found here, on the Scientific Publications & Presentations page of Intellias website.
About Intellia Therapeutics
Intellia Therapeuticsis a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.com and follow us on Twitter @intelliatweets.
Forward-Looking Statements
This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020; its plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; its plans to nominate a development candidate for its hereditary angioedema (HAE) program in the first half of 2020; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; its presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML and HAE programs, in any future studies, including human clinical trials; its ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; its business plans and objectives for its preclinical studies and clinical trials, including the therapeutic potential and clinical benefits thereof, as well as the potential patient populations that may be addressed by its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc.(Regeneron) and Regenerons ability to enter into a Co/Co agreement for the HAE program; statements regarding the timing of regulatory filings for its development programs; its use of capital, including expenses, future accumulated deficit and other financial results during 2019 or in the future; and the ability to fund operations through the end of 2021.
Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.
Intellia Contacts:
Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com
Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917lynnea.olivarez@intelliatx.com
Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com
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Intellia Therapeutics Presents New Data From Its Engineered Cell Therapy and In Vivo Programs at Keystone Symposia's Engineering the Genome Conference...
Such is the extent of our dominion on Earththat the answers to questions around whether we are still part of nature and whether we even need some of it rely on an understanding of what we want as Homo sapiens. And to know what we want, we need to grasp what we are.
It is a huge question but they are the best. And as a biologist, here is my humble suggestion to address it, and a personal conclusion. You may have a different one, but what matters is that we reflect on it.
Perhaps the best place to start is to consider what makes us human in the first place, which is not as obvious as it may seem.
Sharing the full story, not just the headlines
Many years ago, a novel written by Vercors called Les Animaux Dnaturs (Denatured Animals) told the story of a group of primitive hominids, the Tropis, found in an unexplored jungle in New Guinea, who seem to constitute a missing link. However, the prospect that this fictional group may be used as slave labour by an entrepreneurial businessman named Vancruysen forces society to decide whether the Tropis are simply sophisticated animals or whether they should be given human rights. And herein lies the difficulty.
Human status had hitherto seemed so obvious that the book describes how it is soon discovered that there is no definition of what a human actually is. Certainly, the string of experts consulted anthropologists, primatologists, psychologists, lawyers and clergymen could not agree. Perhaps prophetically, it is a layperson who suggested a possible way forward.
She asked whether some of the hominids habits could be described as the early signs of a spiritual or religious mind. In short, were there signs that, like us, the Tropis were no longer at one with nature, but had separated from it, and were now looking at it from the outside with some fear.
Pluto has a 'beating heart' of frozen nitrogen that is doing strange things to its surface, Nasa has found.The mysterious core seems to be the cause of features on its surface that have fascinated scientists since they were spotted by Nasa's New Horizons mission."Before New Horizons, everyone thought Pluto was going to be a netball - completely flat, almost no diversity," said Tanguy Bertrand, an astrophysicist and planetary scientist at NASA's Ames Research Center and the lead author on the new study."But it's completely different. It has a lot of different landscapes and we are trying to understand what's going on there."
Getty
The ancient invertabrate worm-like species rhenopyrgus viviani (pictured) is one of over 400 species previously unknown to science that were discovered by experts at the Natural History Museum this year
PA
Jackdaws can identify dangerous humans from listening to each others warning calls, scientists say. The highly social birds will also remember that person if they come near their nests again, according to researchers from the University of Exeter. In the study, a person unknown to the wild jackdaws approached their nest. At the same time scientists played a recording of a warning call (threatening) or contact calls (non-threatening). The next time jackdaws saw this same person, the birds that had previously heard the warning call were defensive and returned to their nests more than twice as quickly on average.
Getty
The sex of the turtle is determined by the temperatures at which they are incubated. Warm temperatures favour females.But by wiggling around the egg, embryos can find the Goldilocks Zone which means they are able to shield themselves against extreme thermal conditions and produce a balanced sex ratio, according to the new study published in Current Biology journal
Ye et al/Current Biology
African elephant poaching rates have dropped by 60 per cent in six years, an international study has found. It is thought the decline could be associated with the ivory trade ban introduced in China in 2017.
Reuters
Scientists have identified a four-legged creature with webbed feet to be an ancestor of the whale. Fossils unearthed in Peru have led scientists to conclude that the enormous creatures that traverse the planets oceans today are descended from small hoofed ancestors that lived in south Asia 50 million years ago
A. Gennari
A scientist has stumbled upon a creature with a transient anus that appears only when it is needed, before vanishing completely. Dr Sidney Tamm of the Marine Biological Laboratory could not initially find any trace of an anus on the species. However, as the animal gets full, a pore opens up to dispose of waste
Steven G Johnson
Feared extinct, the Wallace's Giant bee has been spotted for the first time in nearly 40 years. An international team of conservationists spotted the bee, that is four times the size of a typical honeybee, on an expedition to a group of Indonesian Islands
Clay Bolt
Fossilised bones digested by crocodiles have revealed the existence of three new mammal species that roamed the Cayman Islands 300 years ago. The bones belonged to two large rodent species and a small shrew-like animal
New Mexico Museum of Natural History
Scientists at the University of Maryland have created a fabric that adapts to heat, expanding to allow more heat to escape the body when warm and compacting to retain more heat when cold
Faye Levine, University of Maryland
A study from the University of Tokyo has found that the tears of baby mice cause female mice to be less interested in the sexual advances of males
Getty
The Intergovernmental Panel on Climate Change has issued a report which projects the impact of a rise in global temperatures of 1.5 degrees Celsius and warns against a higher increase
Getty
The nobel prize for chemistry has been awarded to three chemists working with evolution. Frances Smith is being awarded the prize for her work on directing the evolution of enzymes, while Gregory Winter and George Smith take the prize for their work on phage display of peptides and antibodies
Getty/AFP
The nobel prize for physics has been awarded to three physicists working with lasers. Arthur Ashkin (L) was awarded for his "optical tweezers" which use lasers to grab particles, atoms, viruses and other living cells. Donna Strickland and Grard Mourou were jointly awarded the prize for developing chirped-pulse amplification of lasers
Reuters/AP
The Ledumahadi Mafube roamed around 200 million years ago in what is now South Africa. Recently discovered by a team of international scientists, it was the largest land animal of its time, weighing 12 tons and standing at 13 feet. In Sesotho, the South African language of the region in which the dinosaur was discovered, its name means "a giant thunderclap at dawn"
Viktor Radermacher / SWNS
Scientists have witnessed the birth of a planet for the first time ever. This spectacular image from the SPHERE instrument on ESO's Very Large Telescope is the first clear image of a planet caught in the very act of formation around the dwarf star PDS 70. The planet stands clearly out, visible as a bright point to the right of the center of the image, which is blacked out by the coronagraph mask used to block the blinding light of the central star.
ESO/A. Mller et al
Layers long thought to be dense, connective tissue are actually a series of fluid-filled compartments researchers have termed the interstitium. These compartments are found beneath the skin, as well as lining the gut, lungs, blood vessels and muscles, and join together to form a network supported by a mesh of strong, flexible proteins
Getty
Working in the Brazilian state of Mato Grosso, a team led by archaeologists at the University of Exeter unearthed hundreds of villages hidden in the depths of the rainforest. These excavations included evidence of fortifications and mysterious earthworks called geoglyphs
Jos Iriarte
More than one in 10 people were found to have traces of class A drugs on their fingers by scientists developing
a new fingerprint-based drug test.Using sensitive analysis of the chemical composition of sweat, researchers were able to tell the difference between those who had been directly exposed to heroin and cocaine, and those who had encountered it indirectly.
Getty
The storm bigger than the Earth, has been swhirling for 350 years. The image's colours have been enhanced after it was sent back to Earth.
Pictures by: Tom Momary
Pluto has a 'beating heart' of frozen nitrogen that is doing strange things to its surface, Nasa has found.The mysterious core seems to be the cause of features on its surface that have fascinated scientists since they were spotted by Nasa's New Horizons mission."Before New Horizons, everyone thought Pluto was going to be a netball - completely flat, almost no diversity," said Tanguy Bertrand, an astrophysicist and planetary scientist at NASA's Ames Research Center and the lead author on the new study."But it's completely different. It has a lot of different landscapes and we are trying to understand what's going on there."
Getty
The ancient invertabrate worm-like species rhenopyrgus viviani (pictured) is one of over 400 species previously unknown to science that were discovered by experts at the Natural History Museum this year
PA
Jackdaws can identify dangerous humans from listening to each others warning calls, scientists say. The highly social birds will also remember that person if they come near their nests again, according to researchers from the University of Exeter. In the study, a person unknown to the wild jackdaws approached their nest. At the same time scientists played a recording of a warning call (threatening) or contact calls (non-threatening). The next time jackdaws saw this same person, the birds that had previously heard the warning call were defensive and returned to their nests more than twice as quickly on average.
Getty
The sex of the turtle is determined by the temperatures at which they are incubated. Warm temperatures favour females.But by wiggling around the egg, embryos can find the Goldilocks Zone which means they are able to shield themselves against extreme thermal conditions and produce a balanced sex ratio, according to the new study published in Current Biology journal
Ye et al/Current Biology
African elephant poaching rates have dropped by 60 per cent in six years, an international study has found. It is thought the decline could be associated with the ivory trade ban introduced in China in 2017.
Reuters
Scientists have identified a four-legged creature with webbed feet to be an ancestor of the whale. Fossils unearthed in Peru have led scientists to conclude that the enormous creatures that traverse the planets oceans today are descended from small hoofed ancestors that lived in south Asia 50 million years ago
A. Gennari
A scientist has stumbled upon a creature with a transient anus that appears only when it is needed, before vanishing completely. Dr Sidney Tamm of the Marine Biological Laboratory could not initially find any trace of an anus on the species. However, as the animal gets full, a pore opens up to dispose of waste
Steven G Johnson
Feared extinct, the Wallace's Giant bee has been spotted for the first time in nearly 40 years. An international team of conservationists spotted the bee, that is four times the size of a typical honeybee, on an expedition to a group of Indonesian Islands
Clay Bolt
Fossilised bones digested by crocodiles have revealed the existence of three new mammal species that roamed the Cayman Islands 300 years ago. The bones belonged to two large rodent species and a small shrew-like animal
New Mexico Museum of Natural History
Scientists at the University of Maryland have created a fabric that adapts to heat, expanding to allow more heat to escape the body when warm and compacting to retain more heat when cold
Faye Levine, University of Maryland
A study from the University of Tokyo has found that the tears of baby mice cause female mice to be less interested in the sexual advances of males
Getty
The Intergovernmental Panel on Climate Change has issued a report which projects the impact of a rise in global temperatures of 1.5 degrees Celsius and warns against a higher increase
Getty
The nobel prize for chemistry has been awarded to three chemists working with evolution. Frances Smith is being awarded the prize for her work on directing the evolution of enzymes, while Gregory Winter and George Smith take the prize for their work on phage display of peptides and antibodies
Getty/AFP
The nobel prize for physics has been awarded to three physicists working with lasers. Arthur Ashkin (L) was awarded for his "optical tweezers" which use lasers to grab particles, atoms, viruses and other living cells. Donna Strickland and Grard Mourou were jointly awarded the prize for developing chirped-pulse amplification of lasers
Reuters/AP
The Ledumahadi Mafube roamed around 200 million years ago in what is now South Africa. Recently discovered by a team of international scientists, it was the largest land animal of its time, weighing 12 tons and standing at 13 feet. In Sesotho, the South African language of the region in which the dinosaur was discovered, its name means "a giant thunderclap at dawn"
Viktor Radermacher / SWNS
Scientists have witnessed the birth of a planet for the first time ever. This spectacular image from the SPHERE instrument on ESO's Very Large Telescope is the first clear image of a planet caught in the very act of formation around the dwarf star PDS 70. The planet stands clearly out, visible as a bright point to the right of the center of the image, which is blacked out by the coronagraph mask used to block the blinding light of the central star.
ESO/A. Mller et al
Layers long thought to be dense, connective tissue are actually a series of fluid-filled compartments researchers have termed the interstitium. These compartments are found beneath the skin, as well as lining the gut, lungs, blood vessels and muscles, and join together to form a network supported by a mesh of strong, flexible proteins
Getty
Working in the Brazilian state of Mato Grosso, a team led by archaeologists at the University of Exeter unearthed hundreds of villages hidden in the depths of the rainforest. These excavations included evidence of fortifications and mysterious earthworks called geoglyphs
Jos Iriarte
More than one in 10 people were found to have traces of class A drugs on their fingers by scientists developing a new fingerprint-based drug test.Using sensitive analysis of the chemical composition of sweat, researchers were able to tell the difference between those who had been directly exposed to heroin and cocaine, and those who had encountered it indirectly.
Getty
The storm bigger than the Earth, has been swhirling for 350 years. The image's colours have been enhanced after it was sent back to Earth.
Pictures by: Tom Momary
It is a telling perspective. Our status as altered or denatured animals creatures who have arguably separated from the natural world is perhaps both the source of our humanity and the cause of many of our troubles. In the words of the books author:
All mans troubles arise from the fact that we do not know what we are and do not agree on what we want to be
We will probably never know the timing of our gradual separation from nature although cave paintings perhaps contain some clues. But a key recent event in our relationship with the world around us is as well documented as it was abrupt. It happened on a sunny Monday morning, at precisely 8.15am.
A new age
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The atomic bomb that rocked Hiroshima on 6 August 1945 was a wake-up call so loud that it still resonates in our consciousness many decades later.
The day the sun rose twice was not only a forceful demonstration of the new era that we had entered buta reminder of how pa
radoxically primitive we remained: differential calculus, advanced electronics and almost godlike insights into the laws of the universe helped build, well a very big stick. Modern Homo sapiens seemingly had developed the powers of gods, while keeping the psyche of a stereotypical Stone Age killer.
We were no longer fearful of nature, but of what we would do to it, and ourselves. In short, we still did not know where we came from but began panicking about where we were going. We now know a lot more about our origins but we remain unsure about what we want to be in the future or, increasingly, as the climate crisis accelerates, whether we even have one.
Arguably, the greater choices granted by our technological advances make it even more difficult to decide which of the many paths to take. This is the cost of freedom. I am not arguing against our dominion over nature nor, even as a biologist, do I feel a need to preserve the status quo. Big changes are part of our evolution. After all, oxygen was first a poison which threatened the very existence of early life, yet it is now the fuel vital to our existence.
Similarly, we may have to accept that what we do, even our unprecedented dominion, is a natural consequence of what we have evolved into, and by a process nothing less natural than natural selection itself. If artificial birth control is unnatural, so is reduced infant mortality.
I am also not convinced by the argument against genetic engineering on the basis that it is unnatural. By artificially selecting specific strains of wheat or dogs, we had been tinkering more or less blindly with genomes for centuries before the genetic revolution. Even our choice of romantic partner is a form of genetic engineering. Sex is natures way of producing new genetic combinations quickly.
Even nature, it seems, can be impatient with itself.
Changing our world
Read the original:
Have humans evolved beyond nature? - The Independent
In both pre-Christianity Rome and China, the countryside was a place of retreat where those seeking respite from the bustle and grime of the city would go for rest, relaxation, and creative inspiration. The Chinese founders of Taoism called this freedom and wondering Xiaoyao, while Roman philosophers referred to time away as Otium: and idealized existencesfrom off-the-grid hippy utopias to the peaceful bliss of Arcadiahave continued to crystallize in the natural landscapes of the rural. Contemporary ideas around wellness, mindfulness, ayahuasca startup retreats, and glamping at Burning Man fill the same role in our society as a full-circle return to pre-industrial, pre-capitalist, nature-centric lifestyles that are paradoxically a product of our neoliberal consumerist culture and sold as an antidote to it.
This lineage, from the beginning of western civilization and ancient eastern philosophy to 21st-century marketing culture, is just part of Rem Koolhaass ten-year transcultural, transhistorical research and analysis of non-urban territories, or what he calls the ignored realm. On view at New Yorks Guggenheim Museum through August 14, Countryside: The Future is a project of Koolhaas, AMO director Samir Bantal, and Troy Conrad Therrien of the Guggenheim. The show fills the museums entire main rotunda. It is meant to upend traditional notions of the countryside by investigating the places where the influence, as well as the oddities, normally associated with the urban can be found outside the city. If, at one time in the not-so-distant past, the countryside was an idyllic place where each human had a role, Koolhaas posits that the romantic landscape of creek beds, hillsides, and family farms is now unrecognizable as a stable, human-centered place, but rather a hyper-efficient, inorganic, non-place where Cartesian technological systems define life.
The show reverses course on much of what we have come to accept as the baseline for thinking about development. Take that famous statistic: by 2050, 70- to-80 percent of humanity would live in cities. Are we really heading for this absurd outcome, where the vast majority of humanity lives on only 2% of the earths surface, and the remaining 98%, inhabited by only one-fifth of humanity, exists to serve cities? Of course, Rem is not the first person to do research on the rural. But he has the resources (5 partner schools and AMO), the storytelling ability, and the platform (an entire museum in NYC) to reorient the conversation, as he has on other topics such as cities, Dubai, and toilets.
New nature; Highly artificial and sterile environments are employed to create the ideal organic specimen. Todays glass houses can contain all the essential ingredients of life but none of the redundancies: sun, soil, and water are emulated, optimized, and finally, automated. (Pieternel van Velden)
The exhibition starts outside the museum, with a tractor next to a small, high-tech indoor tomato farm under pink lights that illuminate passing pedestrians. In the lobby, a requisite hanging sculpture in the rotunda is made from a bale of hay, an imaging satellite akin those used by Google Maps, and an underwater robot that kills fish threatening coral reefs. Land, sea, and even space are all implicated in this broad survey of the rural, as this sculpture sets the tone for the rest of the show, which launches into an outpouring of information. It is reminiscent of OMA/AMO publications Content, Volume, or the Elements exhibition and books, as visitors are greeted by a wall text of 1,000 questions posed by Koolhaas. Nearby is a table showcasing publications that provided context: The Red Book and the Great Wall, The Future of the Great Plains, Golf Courses of the World, and a German publication about Muammar al-Gaddafi.
At the core of the show, the Guggenheims iconic ramp houses a set of themed vignettes. Political Redesign is a catalog of heroic 20th-century geopolitical operations, ranging from the founding of several United States federal agencies during the Dust Bowl, to German Architect Herman Srgels plan to unite Europe and Africa by lowering the level of the Mediterranean Sea and building a bridge over the resulting span. Stalins Plan for the Transformation of Nature and the evolution of the Jeffersonian grid from squares to circles are also highlights.
Countryside then moves away from these governmental models into more polyvalent experiments with nature, technology, politics, planning, and preservation. Many of these we might normally associate with the urban, such as the anarchist community in Tarnac, France that was raided by police in 2008 but is now home to an informal university hidden in the forest. There are also glimpses of rural China, most beautifully Taobao Live, Alibabas live streaming channel that allows sellers in the countryside to broadcast their produce and foodstuffs to audiences in the cities. Arcosanti, afro-futurism, and Chinas Belt and Road Initiative are among the other kaleidoscopic ways that the narrative extends beyond industrial farming into a host of other social and political spheres.
Working through contemporary preservation methods, proposals, and scenarios, including a curious example from Siberia where valuable mammoth tusks are becoming exposed in the ground by climate change and creating new economies for local, amateur archaeologists, the exhibitions closes on cartesian euphoria, a kind of paranoiac-critical reading of the technologies and systems that are rearranging nature and politics in the countryside, complete with a full-scale installation of a PhenoMate, a cutting-edge farming tool that uses machine learning to identify which plants in a nursery bed photosynthesizing the most, and selectively breeds stronger strains without genetic modification.
The show operates politically in a context where the countryside, and those who live in it are a marginalized group, at least culturally. Urban elites deride rural areas as many things, most out-of-touchedly as fly-over states. After a decade or more or the architectural world focusing on cities and urban areas as the main spaces of inquiry, Rems turn to the countryside most likely born from a desire to look where most others are not and his ability to show the public that the so-called hinterlands are a place where not only are some of the most important agricultural, industrial, and social mechanisms of society operating, but it is also where many of the interesting intersections of experimental politics, economics, engineering, and social relationships are taking place.
To ignore the rural because we dont agree with the politics of those who live there, or think that their culture is not sophisticated is not only missing out on experiencing a countryside beyond a luxury faux-rustic retreat, but it is also disregarding the fact that the countryside and the city are and always will be inextricably linked, as elucidated by a brilliant provocation that cities have become stuck in frivolity, while supported by complex, managed landscapes in the countryside. For example, urbanites underneath Londons ArcelorMittal Orbit leisurely eat ice cream brought in from factory farms in the outskirts.
The frivolity of urban life has necessitated the organization, abstraction, and automation of the countryside at a vast and unprecedented scale. Left: Mishka Henner, Feedlots, 2013. Right: Queen Elizabeth Olympic Park, London, 2018. (Luca Locatelli)
It is also a show with a decidedly top-down lens on the countryside. Some will not like the relative lack of representation of small-scale communities in the show, but the acknowledgment of systems and technology is an important way of seeing these territories. Had the curators included more grassroots narratives, it likely would have watered down the larger, geopolitical stories being told, and the show is better off for staying focused on larger-scale issues rather than getting into the folk aspects of the countryside, which would be more predictable and less compelling.
Countryside is definite
ly a magazine- or book-on-the-wall type of exhibition, but not in a bad way. The texts are snappily written in typical Koolhaasian style, and there are not too many complex maps or charts, making the exhibition feel more like a journalistic analysis of what is interesting about the countryside, not necessarily a theoretical treatise or prescriptive path forward. It could be read as a transformation of the museum into a publication, a curatorial strategy that upturns not only our ideas about the Guggenheim but about how to leverage a hyper-didactic exhibition into an aesthetic experience. The show is literally distorted by the Guggenheims double-curved surfaces, spiraling ramp, and constantly shifting vantage points, with a string of text spiraling around the underside of the ramps like a dizzying thesis statement, always to be revisited.
If there is a sticking point, it is that the aesthetic of the exhibition will be familiar to many, as it harkens back to previous OMA/AMO publications. Koolhaas has long collaborated with Dutch graphic designer Irma Boom, who created a custom Countryside typeface for the show, which resembles both handwriting and her Neutral typeface used throughout. In an exhibition that is really a publication, typefaces matter, and the familiar layouts and fonts make the exhibition seem more like the work of a signature architect or firm, not a global coalition. No, but seriously, folks, go see the show!
Taschen has published an accompanying publication, available for 24.95 online or at the gift shop.
When Ridley Scott set about making Philip K. Dick's novelDo Androids Dream of Electric Sheep?into a film, he knew there'd be some changes that had to be made to properly adapt it to the silver screen.One of the main alterations had to do with the "androids" in the novel, which Scott feared would be too similar to the Ash character he'd created forAlien.His daughter, studying biology at the time suggested something to do with "replication" and thus the term "Replicants" was born.
RELATED:Blade Runner: 5 Fan Theories That Have Been Debunked (& 5 That Have Been Confirmed)
WhenBlade Runnerwas released, it changed the state of the science fiction genre with its ideas on artificial intelligence, futurism, and empathy. And while several aspects where altered from the concepts of the novel, as the "blade runner" Deckard hunted the rogue Replicants, he discovered what it meant to truly be human in both. Philip K. Dick died shortly before the film was released, but K.W. Jeter wrote several sequels to both the novel and the film. Below you'll find 10 facts about Replicants from the books the movies leave out.
The theatrical version ofBlade Runnerincluded much more exposition in the preamble, as well as a voice-over narration provided by Harrison Ford as Rick Deckard to make sure the plot wasn't too convoluted for viewers to follow. Much of that was lost in theDirector's Cut, so was the extent to which Replicants were mistreated.
One of the main reasons that Roy Batty, Pris, and the other four Replicants escaped the Martian colonies were because they were slaves. They were made as slaves to serve humans, and mistreated egregiously as "skin jobs".Their abuse was largely dropped from the film for making them too sympathetic.
Though it isn't shown in the films, replicant aging can be slowed if the subject is placed into stasis before they begin to shut down, like the Roy Batty unit. This comes up inBlade Runner 2: The Edge of Human,when Rachael is placed into a Tyrell Corp transport container by Deckard after they leave the city at the end of theBlade Runner.
In the book, Deckard lives in a shack outside the city with Rachael, who had to be put into the container to slow her aging process. As long as she's kept inside, she's frozen in time exactly as she was, until Deckard can find a way to permanently keep her from aging. Her ability to reproduce isn't mentioned as a part of her model untilBlade Runner 2049,and is taken fromBlade Runner 3: Replicant Night.
When the Replicants are introduced in Blade Runner,audiences aren't informed about the creative process behind their conception, or why they were created to look the way they do. In the bookBlade Runner 2: The Edge of Human, Deckard meets Sarah, Eldon Tyrell's niece and the template used for Rachael, when she tasks him with finding the "missing" sixth replicant.
RELATED:Rutger Hauer's 10 Most Memorable Roles, Ranked
He also meets the template for Roy Batty, who also desires to find the sixth Replicant. He believe sit to be Deckard. The Batty template has the same superior physical stamina and ruthless intelligence as his Replicant, which begs the question - is he just another improved model?
While Eldon Tyrell is said to be the genius behind the Replicant, other corporations initially created similar synthetic beings, Tyrell Corp simply had a monopoly on the market. Th origins of Tyrell isn't explored inBlade Runner, Blade Runner 2049,or any of the books untilBlade Runner 4: Eye and Talon.
A female blade runner named Iris is given an explanation that the UN destroyed the other manufacturers of replicants in order to create the Tyrell Corp artificially. Eldon Tyrell had simply discovered a way to manufacture them the best, and even help them house the gestalt of a human's consciousness, allowing them to transfer their mind into a new "body" every four years.
InBlade Runner 2: The Edge of Humanand its supplicant sequel novels, it's revealed that a Replicants' personality could be stored in the event that the host unit was destroyed. Rick Deckard kept Roy Batty's personality tucked away in his briefcase.
RELATED:10 Best Sci-Fi Movies About Artificial Intelligence, Ranked
InBlade Runner 4: Eye and Talon, it's revealed that Eldon Tyrell's personality was stored inside the mysterious replicant owl that always fluttered around Tyrell Corp inBlade Runner.Taking the owl and a suitable replicant host body, Eldon Tyrell could be "brought back to life".
In the fourth novel,Blade Runner 4: Eye and Talon,in which a female blade runner Iris searches for the Replicant owl that observed so much in the glory days of Tyrell Corp, readers are introduced to the concept of Replicants as a means to ensure immortality.
RELATED:Master of Sci-Fi: 10 Ways Philip K. Dick Influenced Sci-Fi Movies & TV
Iris finds several Eldon Tyrell Replicants, each with no eyes. This is to reflect when Roy Batty gouged out the eyes of the real Eldon Tyrell. They're waiting for Tyrell's personality (stored in the owl) to be merged, allowing Tyrell to live forever via Replicants.
Something that may not come across inBlade Runneris that humans are encouraged to go Off World by the UN in order to preserve the "genetic integrity" of humans. To entice them to move away from Earth, humans are given Replicants to assist them in their new life on an Off World colony.
These Replicants are called "andys" (short for android) are created by The Rosen Association on Mars, and are released to their human owners when they arrive. The Replicants aren't permitted to return to Earth, but assist their humans until their life span runs out (4 years).
When Deckard meets Rachael and administers the Voight-Kampff questionnaire, with 20-30 questions designed to determine whether or not she's a Replicant, she exceeds 100 questions before he determines that she is. She's been implanted with human memories to make her the most human-like Replicant in existence.
Unless you saw the theatrical version, which included a voice over, you won't have known that Rachael (and all Nexus-7's) have an open-ended life span. This was removed from theDirector's Cutto be left open ended, and in the sequel novel, Deckard is forced to put her in a Tyrell transport container to stop her aging process. InBlade Runner 2049,Nexus-9 Replicants' life spans are also open ended.
Though the preamble inBlade Runnerexplained that Replicants were created as the culmination of advanced robotics and genetic engineering, they can't be compared to the androids in Ridley Scott'sAlienfranchise. Ash and Bishop were full of tubing, wiring, and microchip processors, whereas Scott wanted Roy Batty and Pris to be full of organic tissue.
RELATED:Blade Runner: 5 Things the Movies Left Out (& 5 Things They Added In)
In essence, they needed to appear "grown" in a lab, not fabricated in a machine shop.Scott wanted it visually conveyed that they couldappear more trustworthy to humans by being similar to them. When they're injured, they bleed, and the only reason they were given empathy inhibitors was to prevent them from overriding theVoight-Kampff test. In Philip K. Dick's novel, they're described in much more robotic terms.
In the novelDo Androids Dream of Electric Sheep?Deckard is more a bounty hunter, less a detective. He agrees to "retire andys" because it'll make him enough money to buy a larger real animal. Since radiation killed most life on Earth, organic animals (as opposed to those that are Replicants) are highly prized for their status.
Deckard lacks any empathy for the Replicants as a contract killer, which is appropriate for his level of hypocrisy, since he rarely shows emotion for his wife or fellow humans. At some point, as in the filmBlade Runner,Deckard has to find a point of change, and learn to become more human by hunting those made in that image.
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Kayleena has be
en raised on Star Wars and Indiana Jones from the crib. A film buff, she has a Western collection of 250+ titles and counting that she's particularly proud of. When she isn't writing for ScreenRant, CBR, or The Gamer, she's working on her fiction novel, lifting weights, going to synthwave concerts, or cosplaying. With degrees in anthropology and archaeology, she plans to continue pretending to be Lara Croft as long as she can.
See the rest here:
Blade Runner: 10 Facts About Replicants From The Books The Movies Leave Out - Screen Rant
Dr. Reshma Shetty is no stranger within the synthetic biology community. In 2008 she co-foundedGinkgo Bioworksa company youll definitely hear about if you havent alreadyalong with fellow MIT grad students Austin Che, Barry Canton, and Jason Kelly, and their graduate adviser, Professor Tom Knight. They started with a simple but revolutionary goal: help people design and build organisms. A decade later,Ginkgo achieved unicorn statusa private company valued at over $1 billionand it finds itself at the fore of the synthetic biology revolution with customers seeking to build organisms for use in fields as diverse as health, food, agriculture, cosmetics and materials.
Shetty has been through the whole journey and has been a major influence in the synthetic biology community. She had a majorrole in the firstInternational Genetic Engineering Machine (iGEM) Competition with her co-founders. In 2008, she was named one of Eight People Inventing the Future by Forbes and, in 2011, one of the 100 Most Creative People in Business by Fast Company.
Shetty is an upbeat talker. If theres any stress or jadedness from navigating a company from birth to unicorn over a decade, it doesnt show. There is a sincere enthusiasm in her voice, especially when we discuss the science. When I caught up with her a few weeks back, one of things I wanted to know was: what do you do when you realize youre riding a biotech unicorn?
What was the moment when you realized that Ginkgo was going to be big?
It was when we closed ourSeries B financing. It was a $45 million round or roughly speaking, so that was more dollars dumped into our bank account at one instance than we ever had before.
My thought was, well pretty serious people withserious capital are choosing to take a bet on us.
This was confirmed for her in 2017 whenBayer chose to work with Ginkgoon engineering biologicals for agriculture, proving the intrinsic value of their platform and cementing Ginkgo as a platform company.
It proved three things at the time. One, that engineered microbes in the environment could be a thing, that [they] could be a product category. There are serious people taking serious bets that were going to be able to release engineered microbes in the future. Two, that Ginkgos platform had value even in areas that we hadnt previously been in. Three, it proved to the world that Ginkgo was really a platform company, that we werent simply going after a few products in the industrial biotech market.
It wasnt easy sailing for Gingko from the start though. Right after the company was founded, the global economy took a nosedive.
I think we incorporated in July of 2008 and, like literally, within the next month or two, the fiscal crisis hit,says Shetty.
In many ways this was not the ideal time to be starting a business and looking for investment, leading to creative thinking in getting the company going.
What did you learn in those early days that biotech companies could benefit from?
At the time everybody said that the way to start a biotech start-up is to go raise money immediately because you need some amount of money to be able to start a lab and get going. The thing I had to learn and realize was that no, actually, it is possible. If youre creative enough, savvy enough and patient enough, then you can in fact bootstrap even a biotech start-up.
Shetty stresses the importance of having the space to figure out their technology platform and business model and ask themselves how to take it forward. Having Knight and his wealth of experience on the team certainly helped.
Tom always said Oh, its a good idea to bootstrap in the early years regardless, based on his prior experience starting companies. But circumstances certainly reinforced that and I think that was really helpful that we spent the first few years bootstrapping the company.
Was it natural having your former advisor on the team?
Yeah, very natural. Tom, hes a pretty low-key guy, but hes also been very ahead of his time when it comes to thinking about the technology and technology trends. Early on it was great because Tom has started and run a company before and there were some obvious pitfalls that he could help us avoid and talk a bit about options.
And your other co-founders, what is it about them that makes them special?
I think probably for me the biggest thing is that weve now been working together for almost 20 years, says Shetty, referencing their time at MIT in the years before Ginkgo.
And even now, if Im struggling with something or Im trying to dig through how to solve a problem, I would want to talk to Tom, Barry, Austin, and Jason. I always come away having learned something or clarified my thinking or somehow changed how I was approaching a problem. To me, that is the real hallmark of excellence.
Despite all those shared experiences, they still learn from one another and solve problems together. Shetty considers her colleagues to be mentors too, saying shes benefitted from them as much as from her supervisors through the years.
Anybody can be a mentor, she says.
They are all engineers at heart, so the most exciting things for the Ginkgo team are around potentially world-changing technologies that can jump quickly from dream to reality.
What are the engineering challenges youre most excited about these days?
Bayer and Ginkgo, through our joint venture inJoyn, are going after nitrogen fixation. It has long been a dream of folks. Could we reduce fertilizer usage by using biological nitrogen fixation instead?
This project has been close to Shetty since her academic days, but therapeutics and Ginkgoscollaboration with Synlogic, who develop bacteria as living medicines, has also piqued her interest.
Theres all these areas of metabolism that lead to devastating diseases and the idea that you could engineer microbes to basically treat them is a cool idea!
Is there any particular problem youd like to solve through engineering biology?
How do you think about leveraging biology to make a positive impact on the environment? Thats one I think has been on our wish list for a while.
Enabling the future of synthetic biology is a big part of how Ginkgo operates, even since the early days. The founders were involved in establishing iGEM and their platform is well suited to collaborative efforts.
How do you see Ginkgos role to give back and enable the next generation of synthetic biology?
I think one thing that has been a longstanding ask from folks in the community is how are we going to open up our cell programming platform to more people? Early on, that seemed crazy to even think about, she says, citing the skill set required to use and build it. I think weve come a long way since then so we can say actually maybe we get started thinking about opening up the platform to more folks.
Shetty says initial collaborations like Joyn, (Ginkgo spin-out)Motif, andSynlogicmean they can learn how to open their platform better. Relationships with accelerators likeYCombinatorandPetriare the next steps. They acknowledge that opening their platform will only benefit and accelerate biological engineering.
Our conversation then moves onto a more human element of running a company, a reminder that its never all about the science.
Do you have any mistakes or regrets in how youve done things?
The biggest regret I have is actually not thinking consciously about diversity and inclusion issues earlier in Ginkgos history. We started thinking about them seriously in about 2015 or so, when we were still relatively small, about 30 people. But we could have thought about diversity and inclusion even earlier.
Shetty reveals its easier to change the balance in a company when its just a handful of people.
Can we be doing better on diversity as a whole?
I would say that synthetic biology as a field has always been pretty good in that it thought about issues outside of just the science and engineering itself. I think the
field always fosters that broader perspective. So I think its been more natural and more normal to think about diversity and inclusion issues in the synthetic biology community as a result, says Shetty, Were by no means beyond reproach but theres more of a willingness to talk about these issues and really try to take proactive steps.
Do you have any advice for those starting a company?
The thing I like to tell people is that, if youre going to start a company, dont do it for the money. There are a lot of easier ways to make money in the world. Start a company because you think a company is really the best way to go tackle a problem that youre passionate about.
Any final thoughts?
I think that weve come a long way in terms of our ability to engineer biology, but we still have a long way to go. Fundamentally, biology is still not yet a predictable engineering discipline and its important to remember that. Because its still not yet predictable, we have to iterate through different designs and search for a functional design whenever were trying to engineer a GMO. We have more work to yet do to bring down the cost of doing genetic engineering so that we can explore more and more of design space.
Follow me on twitter at@johncumbersand@synbiobeta. Subscribe to my weekly newsletters insynthetic biologyandspace settlement.
Thank you toDavid KirkandKevin Costafor additional research and reporting in this article. Im the founder ofSynBioBeta, and some of the companies that I write about includingGinkgo Bioworks are sponsors of theSynBioBeta conferenceandweekly digestheres the full list of SynBioBeta sponsors.
Excerpt from:
An Interview with Ginkgo Bioworks' Reshma Shetty On Co-Founding Synthetic Biology's First Unicorn - SynBioBeta
More than 150 New Zealand scientists under 30 have signed a letter to the Green Party urging a rethink of its stance on the regulation of genetic modification. The full text of the letter follows.
To the members and supporters of the Green Party of Aotearoa New Zealand and their representatives in government
Climate change is one of the greatest crises in human history, and our current law severely restricts the development of technologies that could make a vital difference. In 2003 the 1996 Hazardous Substances and New Organisms Act was modified to tightly regulate research into genetic modification (GM). This legislation and the surrounding public debate was driven by uncertainty about the risks that these new technologies posed to biodiversity and human health, and resulted in creating one of the toughest regulatory environments in the world for this field of research.
We, an emerging generation of New Zealand scientists with expertise in and/or undertaking research in the biological sciences*, are writing to request that the Green Party reconsider its position on the regulation of these technologies. We are addressing this letter to the Greens because of a history of leading in science-based policy such as climate action, even when that path is difficult. We believe that GM based research could be decisive in our efforts to reduce New Zealand and global climate emissions as well as partially mitigating some of the impacts of climate change. At the same time, we emphasise that potential reduction of impact is not a substitute for emission reduction.
The period since the introduction of the 2003 legislation has seen important GM related research in the areas of agricultural efficiency, carbon sequestration, and alternative protein production. The existing regulation in New Zealand inhibits application of advances such as these, blocking not only the development of green technology, but the potential for a just transition away from extractive and polluting industries. New Zealand has the opportunity to be a world leader in such a transition: for example, the development and demonstration of effective technologies to reduce agricultural emissions could have an international impact and set an example for other countries.
While such a powerful technology as targeted genetic modification certainly requires controls, existing frameworks do not enable public and environmental benefits from these technologies to be realised. The gene editing expert advice panel supported by The Royal Society Te Aprangi, the Prime Ministers Chief Science Advisor, and the interim climate change committee have recently called for public discussion on potential reform of New Zealands laws around modern gene editing techniques.
As a confidence and supply member of the current government the Greens have the ability to drive this reform: the members can persuade the party to reconsider its policy position, and the Members of Parliament can influence the government it supports to revise the legislation. The Greens have been strong advocates of both climate action and evidence based policy informed by science. In this light we call upon its members, supporters, ministers, and MPs to add their voices to the cause of a science-based approach to climate, on behalf of the people and environment of both Aotearoa and the world.
Ng mihi
PhD
Kyle Webster, University of Auckland, Bio-nanotechnology
Luke Stevenson, Victoria University of Wellington, Biotechnology
Emilie Gios, University of Auckland, Microbial ecology
Morgane Merien, University of Auckland, Biological Sciences Entomology
Lucie Jiraska, University of Auckland, Environmental Microbiology
Victor Yim, University of Auckland, Peptide chemistry
Zach McLean, University of Auckland, Genetic engineering
Declan Lafferty, Plant and FoodResearch/University of Auckland, Genetics and Molecular Biology
Samarth Samarth, University of Canterbury, Plant Biology
Juliane Gaviraghi Mussoi, University of Auckland, Avian Behaviour
Alex Noble, University of Canterbury, Biology
Kelsey Burborough, University of Auckland, Genetics
Matthew Mayo-Smith, University of Auckland, Plant Molecular Biology
Moritz Miebach, University of Canterbury, Plant-microbe interactions
Olivia Ogilvie, University of Auckland, Food Biotech / Biochemistry
Rachel Bennie, University of Canterbury, Human Toxicology
Sean Mackay, University of Otago, Chemistry and Nanotechnology
Georgia Carson, Victoria University of Wellington, Cell and Molecular Biology
Ruby Roach, Massey University
Jeremy Stephens, Massey University, Biology
Zidong (Andy) Li, Massey University, Molecular Cancer Biology
Aqfan Jamaluddin, University of Auckland, Molecular Pharmacology
Michael Fairhurst, Victoria University of Wellington, Microbiology
Nikolai Kondratev, Massey University, Plant Biology
Mariana Tarallo, Massey University, Plant pathology
Ellie Bradley, Massey University, Plant pathology
Mercedes Rocafort Ferrer, Massey University, Plant pathology
Yi-Hsuan Tu, Massey University, Biochemistry & Microbiology
Sean Bisset, Massey University, Biochemistry
Patrick Main, Massey University, Biological sciences
Abigail Sharrock, Victoria University of Wellington, Biotechnology
Alvey Little, Victoria University of Wellington, Molecular Microbiology
William Odey, Victoria University of Wellington, Biotechnology
Gabrielle Greig, Victoria University of Wellington, Molecular Microbiology
Melanie Olds, Victoria University of Wellington, Biotechnology
Jennifer Soundy, Victoria University of Wellington, Biological Sciences
Matire Ward, Victoria University of Wellington, Cell and molecular bioscience
Tom Dawes, Victoria University of Wellington, Plant Ecology
Hamish Dunham, Victoria University of Wellington, Biomedical science
Amy Alder, Victoria University of Wellington, Neuroscience
Caitlin Harris, University of Otago, Plant genetics
Lucy Gorman, Victoria University of Wellington, Coral reef biology
Vincent Nowak, Victoria University of Wellington, Biotechnology
Brandon Wright, University of Otago, Biochemistry
Anna Tribe, Victoria University of Wellington, Cancer cell biology
Conor McGuinness, University of Otago, Breast Cancer
Genomics/Immunology Kelsi Hall, Victoria University of Wellington, Biotechnology
Andrew Howard, University of Waikato, Biochemistry
Mitch Ganley, Victoria University of Wellington, Biotechnology/vaccines
Matt Munro, Victoria University of Wellington, Biomedical Science
Prashath Karunaraj, University of Otago, Genetics
Pascale Lubbe, University of Otago, Evolutionary genetics
Mackenzie Lovegrove, University of Otago, Genetics, Insect evolution
Nicholas Foster, University of Otago, Ecology
Taylor Hamlin, University of Otago, Antarctic Marine Ecosystem & Movement Ecology
Fionnuala Murphy, Massey University, Proteomics
Amanda Board, University of Canterbury, Protein Biochemistry
Esther Onguta, Massey University, Food Technology
Nomie Petit, University of Auckland, Proteins
Liam Le Lievre, University of Otago, Plant Reproduction
James Hunter, University of Otago, Ecology
Samarth Kulshrestha, University of Canterbury,
Rebecca Clarke, University of Otago, Whole body regeneration
Sarah Killick, University of Auckland, Environmental Science
Stephanie Workman, University of Otago, Developmental Genetics
Erik Johnson, University of Otago, Oceanography
Declan Lafferty, University of Auckland, Molecular Biology
Laurine van Haastrecht, Victoria University of Wellington, Glaciology
Leo Mercer, Victoria University of Wellington, Environmental Studies
Aidan Joblin-Mills, Victoria University of Wellington, Chemical Genetics
Gabrielle Keeler-May, University of Otago, Marine Science
Aqfan
Jamaluddin, University of Auckland, Pharmacology
Spencer McIntyre, University of Auckland, Biological Sciences
Sarah Inwood, University of Otago, Genetics
Isabelle Barrett, University of Canterbury, Freshwater ecology
Olivia Angelin-Bonnet, Massey University, Biostatistics
Hannah McCarthy, Massey University, Plant Pathology
Sofie Pearson, Massey University, Plant Science
Zac Beechey-Gradwell, Lincoln University, Plant physiology
Hannah Lee-Harwood, Victoria University of Wellington, Biotechnology
Euan Russell, University of Otago, Microbiology
Masters
Kelly Styles, University of Auckland, Biological Sciences
Merlyn Robson, University of Auckland, Virology
Andra Popa, University of Auckland
James Love, University of Auckland, Bioinformatics
Evie Mansfield, University of Auckland, Molecular Microbiology
Ash Sargent, University of Auckland, Immunology
Sabrina Cuellar, University of Auckland, Plant Genetics
Renji Jiang, University of Canterbury, Plant pathology
Morgan Tracy, University of Canterbury, Ecology
Continued here:
GM could be decisive: An open letter to the Green Party from young NZ scientists - The Spinoff
Nov. 14, 2019 21:00 UTC
ALAMEDA, Calif.--(BUSINESS WIRE)-- AgeX Therapeutics, Inc.(AgeX; NYSE American: AGE), a biotechnology company developing therapeutics for human aging and regeneration, reported financial and operating results for the third quarter ended September 30, 2019.
Driven by our vision of being the leading biotechnology company with a focus on human aging, in the third quarter we advanced our product development on numerous fronts, said Michael D. West, Ph.D., founder and Chief Executive Officer. Our recent build-out of GMP-compliant manufacturing laboratory space will facilitate our manufacture of master cell banks such as those that carry the UniverCyteTM genetic modification for off-the-shelf cell-based regenerative therapy.
Recent Highlights
Second Quarter 2019 Operating Results
Revenues: Total revenues for the three months ended September 30, 2019 were $411,000, as compared with $380,000 in the same period in 2018. AgeX revenue is primarily generated from subscription and advertising revenues from the GeneCards online database through its subsidiary LifeMap Sciences, Inc. 2019 revenues also included $41,000 of grant revenue from the NIH. AgeX had no grant revenues in the same period in 2018.
Operating expenses: Operating expenses for the three months ended September 30, 2019 were $3.6 million, as compared with $2.6 million for the same period in 2018. On an as-adjusted basis, operating expenses for the three months ended September 30, 2019 were $2.9 million as compared to $2.2 million for the same period in 2018.
The reconciliation between operating expenses determined in accordance with accounting principles generally accepted in the United States (GAAP) and operating expenses, as adjusted, a non-GAAP measure, is provided in the financial tables included at the end of this press release.
Research and development expenses for the three months ended September 30, 2019 were $1.4 million, as compared with $1.3 million in the same period in 2018.
General and administrative expenses for the three months ended September 30, 2019 were $2.2 million, as compared with $1.3 million in the same period in 2018.
Net loss attributable to AgeX: The net loss attributable to AgeX for the three months ended September 30, 2019 was $3.2 million, or ($0.09) per share (basic and diluted) compared to $2.2 million, or ($0.06) per share (basic and diluted), for the same period in 2018.
Balance Sheet Highlights
Cash, and cash equivalents, including restricted cash totaled $3.8 million as of September 30, 2019, in addition to which we have $1.5 million remaining available for borrowing under a credit facility provided by Juvenescence Limited, which brought our total available capital to $5.3 million. However, under accounting standard ASC 205-40 Presentation of Financial Statements-Going Concern, AgeXs cash and cash equivalents of $3.8 million as of September 30, 2019 and the loan facility by Juvenescence may not be sufficient, without raising additional capital and reducing expenditures, to satisfy AgeXs anticipated operating and other funding requirements for the next twelve months from the issuance of its interim condensed consolidated interim financial statements.
About AgeX Therapeutics
AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.
For more information, please visit http://www.agexinc.com or connect with the company on Twitter, Facebook, and YouTube.
Forward-Looking Statements
Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the Risk Factors section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.
AGEX THERAPEUTICS, INC. AND SUBSIDIARIES
CONDENSED CONSOLIDATED BALANCE SHEETS
(IN THOUSANDS, EXCEPT PAR VALUE AMOUNTS)
September 30, 2019
December 31, 2018
(Unaudited)
ASSETS
CURRENT ASSETS
Cash and cash equivalents
$
3,768
$
6,707
Accounts and grants receivable, net
234
131
Prepaid expenses and other current assets
688
1,015
Total current assets
4,690
7,853
Go here to see the original:
AgeX Therapeutics Reports Third Quarter 2019 Financial Results and Provides Business Update - BioSpace
Science and technology are the great drivers of innovation in the worldaround us.Technological and scientific breakthroughs help people every day, bringing drinking water to the needy, access to information through the internet to remote villages, cures for obscure diseases.
Many aspects of scientific discovery are under no ethical questions. But there are alsoa number of scientific endeavors that push the ethical lines of what science should revolve around. While all the areas of controversy we'll look into have great benefits, they also come with a lot of ethical burdens, like harm to animals, people, or the environment.
It all should make us stop and think, at what point do the negatives of innovation overshadow the good that it brings. And is there ever an innovation so beneficial to the world and mankind that it would be worth ethical tragedy on the road to scientific and technological progress? Ponder these questions as we look into 7 ethically controversial areas of science and technology...
Artificial intelligence is at the forefront of techno-jargon these days. Every company that has anything to do with technology is using it as a buzzword to sell their product. "New dog collar with built-in AI to detect when your dog is in distress! Install our simple computer plug-in and we'll optimize your workday."
AI certainlyhas its applications and benefits, but there are areas where it has some extensive drawbacks. Take two key AI technologies that have questionable benefits, or rather extensive drawbacks: deep fakes and Neuralink.
You've probably heard of deep fakes, the face-swapping technology that is used to make world leaders say things they never didor for less family-friendly things.
You might not know about Neuralink though. It's one of Elon Musk's technological endeavors that aims to improve brain-machine interfaces, record memories, and other technological advancements with the brain.
Focusing in on Neuralink first, questions surround the ethics of connecting human brains to machines and utilizing AI to make human brains function better. Ethical questions primarily focus on the development of said technology and potential side-effects. The company's goal is to optimize human brain function, but the testing that will be needed to get there will be extensive. This means human testing, on human brains, with unknown consequences. At what point is the potential promise of drastic technological advancement not worth the potential human loss in the development of the technology?
RELATED: AI CONTINUES TO ACT IN UNPREDICTABLE WAYSSHOULD WE BE CONCERNED?
Moving from Neuralink, we're met with technology, deep fakes, that pose less benefit to humanity. There's arguably little reason that anyone needs to replace someone's face with another's in a videoat least, little reason that isn't nefarious.
Yet, technology exists thanks to artificial intelligence and machine learning. It continues to be researched under the guise of benefits through improved video editing technology, but at the end of the day,there's no way to keep it from being used for negative purposes.
At the end of the day, artificial intelligence has the potential to completely change how we interact with the worldbut are there too many negatives? Time will tell...
Through CRISPR, scientists are able to edit human genomes. That means researchers can alter DNA sequences and alter how our genes function. That means the potential to correct genetic defects, preventing the spread of diseaseorrr for making designer babies.
CRISPR is short for CRISPR-Cas9, a gene-editing tool that utilizes the Cas9 enzyme to cut strands of DNA. It's basicallylike molecular scrapbooking.
The idea and implementation of CRISPR came from how bacteria defend themselves, by chopping up and destroying the DNA of foreign invaders before they are able to take hold of the organism.
CRISPR was just a theory until in 2017, a paper was published demonstrating just how CRISPR worked.
Chinese scientists have started using CRISPR to engineer designer babies or create human babies withedited genes, primarily lacking any tendency towards genetic defects. All of this seems noble and can potentiallyimprove humanity's quality of life, but at what cost? We largely don't know any potential side-effects and if there are any, we're talking about human life.
Designing humans also brings into question what exactly a human is. Are we naturally occurring beings, or does being a human just mean thinking like we do and form or process doesn't matter?
Moving on from human gene editing in CRISPR, we can examine the ethical issues with gene editing on other beings, like plants. Gene editing encompasses anytime a scientist intervenes in an organism's genetics.
This intervention creates GMOs or genetically modified organisms. This results in stronger, more drought-resistant crops. Or crops that have higher yields per acre, among a bounty of other things.
Today, gene editing happens across the world and it is done on both plants and animals, mostly in the pursuit of better food production. Looking into the animal realm,gene editing has been usedto create pigs that aren't susceptible to Porcine Reproductive and Respiratory Syndrom, or PRRS. Gene editing has been used to create pigs that are naturally very resistant to the disease, improving animal welfare.
The gene-editing process for all organisms is overseen by various federal agencies, obviously depending upon the country you're practicing this science in. It raises many ethical concerns, primarily along with the side-effects that might be caused by it, and it is still a much-debated topic by ethicists.
Animal testing is likely the most controversial area of scientific research on this list. Many people couldn't care less while others vehemently oppose it. For years, animal testing has been used to create newer and better pharmaceuticals, better makeup, better shampoos, etc.
The keyword here is "better" as it means better for humans. At the end of the day, animal testing places the prevention of human suffering over the importance of the prevention of animal suffering. In certain cases, the ethical argument for animal testing is easier, i.e. cancer research, or other pursuits that would prevent human death. In other cases, the argument is harder, as the development of a better lipstick.
The ethical debate around animal testing is essentially a real-life trolley problem. On one hand, you have human suffering and on the other, you have animal suffering. And we seem to have no problem with animal suffering as long as it is for a greater cause.
In introducing the subject, we've made it seem fairly cut and dry, but as science goes, it rarely ever is. An increasing number of scientists are starting to question the relevance of continued animal testing at a time where AI and other tech is starting to be able to accurately model and predict biological interfaces. A great deal of animals are harmed in the creation of many of the chemicals, and we musk as ourselves, is it worth it?
The natural progression from animal testing is human testing or trials of medication on human test subjects. Human subject research is often necessary to get drugs to the final phase of regulatory approval. It serves as the final check of how a givenmedicine or chemical will interact with the human system. Yet, time and time again it has hurt, maimed, or killed individuals. And we have to ask ourselves again, at what point does it become not worth it?
History hasn't been kind to the reputation of human trials, though scientists are making aconstant effort to create safety standardsin the process.
In 1947, it was discovered that two German physicians conducted deadly experiments on concentration camp prisoners during WWII. They were prosecuted as war criminals in the Nuremberg Trials. The Allies then established the Nuremberg Code, being the first international document for voluntary human consent for research.< /p>
With human testing today, the testing proceeds onlyif the patient consents to the study. Though this often leads to people with lesser fortune signing up for human trials to earn some extra cash. The ethics of the entire research situation can still be hotly debated.
Military weapon development is another major crossroad of science and ethics. Take, for example, the development of the atomic bombs during the Manhattan Project during WWII. In many ways, the research conducted during these experiments furthered humanity's understanding of atoms, molecules, and quantum. In other ways, this research killed tens of thousands of people.
Military power and weapon technology pose an ethical dilemma largelydue to the nature ofhumankind. If a givencountry doesn't invest resources into developing the best weapons technology, then another more powerful country will simplyswoop in and overpower them. That's the way it works nowadays. It's the unfortunate truth of the interaction of global superpowers. And once again, we're met with a real-life trolley problem.
Do we invest scientific resources into developing better weapons to protect ourselves and thus kill others, or do we let ourselves be killed and "protect" others? We would certainlynot opt for the latter, would we?
Since it seems like the earth has seen better days, maybe it's time to just abandon our planet and move to a new clean slate, like Mars. We know that there is flowing liquid water on Mars somewhere, and we know there are also other resources to help us survive.
So, why not just up and move humanity there?
The biggest ethical questions around Mars colonization are presented when you consider the potential of life on Mars or the potential of future life on Mars. We can't state with absolute certainty that there is life on the planet. Moving humanity there could harm it. We also don't definitively know that life won't occur on the planet through natural means. If humanity moving there interrupts the natural progression of Mars life, isn't that an ethical issue?
RELATED: SPACEX IS PREPARING A MISSION TO COLONIZE MARS BY 2026
The answers to those suppositions largely have to do with how humanity in total should approach its ethical responsibility. If you believe humanity's only ethical responsibility is to themselves, then it's likely not an issue. If you believe that we're responsible for all lesser life forms, then you'll run into countless ethical dilemmasin the process.
Closing out this discussion of ethical dilemmas in science and technology we're left again wonderingwhat are innovation and the betterment of humanity worth? The answer to that question will vary depending upon who you ask... but ask yourself, what is innovation worth?
The rest is here:
7 Ethically Controversial Research Areas in Science and Technology - Interesting Engineering
GREG WILPERT: Its The Real News Network and Im Greg Wilpert, coming to you from Baltimore.
A scientist in China made what should be a momentous announcement on Monday. He claimed to have successfully edited the genes of a pair of twins who were born earlier this month. The Chinese scientist He Jiankui said that he altered the twins genes so that they would be resistant to the HIV virus, using a gene editing technique known as CRISPR. Heres how he justified the project in an interview with The Associated Press.
HE JIANKUI: I feel a strong responsibility that its not just to make it the first, but also make it an example how to perform like this, consider morality of the society and consider its impact to the public.
GREG WILPERT: Genetic engineering of human genes is illegal in the United States and in most other countries with the potential technology to do so. However, in China, theres no law against it, even though many scientists have expressed strong opposition to the practice. Joining me now to discuss the implications of this announcement is Professor Stuart Newman. Hes professor of cell biology and anatomy at New York Medical College in Valhalla, and he is a founding member of the Council for Responsible Genetics and editor-in-chief of the journal Biological Theory. He is also the author of the forthcoming book, Biotech Juggernaut. Thanks, Stuart, for joining us today.
STUART NEWMAN: Thank you.
GREG WILPERT: So the scientist who did this, He Jiankui, he said that he succeeded in this engineering project, but he did not provide any proof that it actually worked. How likely do you think it is that it actually did work?
STUART NEWMAN: Well, I think hes a serious scientist. I wont comment right now on the morality of what he did, but I think that he knows what hes doing scientifically. And Ive met him, and I think that his claim, as far as I can tell, is probably valid.
GREG WILPERT: So in an article that you published last year, you expressed skepticism that the CRISPR technology could actually do some of this kind of genetic engineering that was used in this particular test. Why is it, what is the issue around well get to the morality later, but I just want to get into the technique for a second. Why are you skeptical about this project of genetic engineering using this kind of technique?
STUART NEWMAN: Well, theres a difference between modifying a gene, even accurately modifying a gene, and bringing about a phenotypic effect that has a biological effect. So in the article that you probably saw, I said that CRISPR wont be useful in bringing about the results that people want because the way genes operate in embryos is not the way that they operate in adult organisms. In an adult organism, you can look at a gene and say it more or less does one thing or it does two things. During embryonic development, it interacts with many other genes in a very quickly changing system and the proteins that the gene specifies dont necessarily do the same thing during development that they do in the adults. So I was skeptical about the ability to bring about desired results. But if its claimed that CRISPR can take a piece of DNA and change it in a specific way, yes it can do that.
GREG WILPERT: So as we saw in the clip of He Jiankui, he says that he felt it was important to do this and to do it for basically what he considered to be a good cause or a good reason. Whats your reaction to this argument and what do you see as being the dangers of this type of work?
STUART NEWMAN: I think its very unjustified that he did it. First of all, He is just looking at the known function of the gene in adult humans, hes not looking at the function during embryonic development. Theres a whole set of unknowns in the developmental process and we dont really have good scientific control over manipulating it, and we may never do because its so complicated. So He has taken a gene with a known function in the adult and hes said its bad to have that gene active, so he inactivated it. But really, theres lot of misconceptions, kind of unthinking, kind of moving ahead in what he did that he should have never done it.
GREG WILPERT: So what is at stake here, basically, and what do you see as being the best way to avoid the worst kinds of consequences of this technology?
STUART NEWMAN: Well I think you know hes taking something that I guess he would say everybody agrees it would be good to be resistant against, AIDS or other viral diseases. So hes looking for some kind of agreement in what he did by the particular problem that he addressed. But in fact, what some people consider an impairment other people dont consider an impairment. In particularly American society, I cant speak for Chinese society, theres a kind of a consumerist ethic which says that if somebody wants to pay for something and its possible to do, they should be allowed to do it.
And in fact, you said at the top of the segment that there are laws against it in the United States, but there really arent. There are not laws in the United States against genetically modifying embryos. So we would have to pass such laws in order to prevent it from happening. And even passing the laws wont prevent it from happening because therell people who do it surreptitiously. So I think that we really have to talk about it a lot. It has to be stigmatized it has to be something that a lot of opprobrium falls on somebody who would attempt such a thing because in many cases it will turn out badly. And then what do you do with one of the unfortunate outcomes that turned out worse rather than better than not and hoped for. This is really a totally poor and motivated project.
GREG WILPERT: Well, what do you think, first of all, are the motivations behind this technology and this project?
STUART NEWMAN: Well, its just kind of a simple-minded approach to a medical problem. I mean, its like saying that AIDS is bad, this gene is associated with AIDS, get rid of this gene and we wont have AIDS or something. So really, it wont affect AIDS and the population, it will affect it in a couple of individuals. And if those individuals that have been genetically modified if it works and I doubt that it will work as intended. But if even if it does work itll just give a license to those resistant individuals to act responsibly and not use precautions and get themselves tested if theyre at risk and so on. So its really crazy, actually, I would call it crazy to try to do this. And scientifically, its based on a poor understanding of science.
GREG WILPERT: I guess the main issue here, perhaps, is that theres a lot of potential for unintended consequences and that the biology is a lot more complicated than people make it out to be if you look only at the individual genes. Is that more or less it in a nutshell?
STUART NEWMAN: Thats absolutely true, yes. And theres a kind of a false notion that if you understand an organisms genes, if you can modify the organisms genes, you can understand how the organism works and you can get it to work in a new way, in kind of an engineering paradigm. And this is not true at all. Genes are not the only thing that are controlling what goes on in an organism, particularly during early development. There are many forces, there physical forces, environmental forces involved in molding the embryos, not just the genes. And the other thing thats not recognized in these attempts is that genes dont always do the same thing in the same context. So the very same gene acting at different stages in the life history of an individual can do very different things, and this is not taken into account at all in these experiments.
GREG WILPERT: OK. Well, well leave it there for now. I was speaking to Stuart Newman, professor of cell biology and anatomy at New York Medical College in Valhalla. Thanks again, Stuart, for having joined us today.
STUART NEWMAN: Thank you.
GREG WILPERT: And thank you for joining The Real News Network. If you like Real News Network stories such as this one, p
lease keep in mind that we have started our winter fundraiser and need your help to reach our goal of raising four hundred thousand dollars. Every dollar that you donate will be matched. Unlike practically all other news outlets, we do not accept support from governments or corporations. Please do what you can today.
Read more from the original source:
Chinese Scientist's Human Genetic Engineering Experiment ...
Human genetic engineering pertains to the practice of adding new DNAs to a person to give him certain traits that he would not naturally have. Due to its concept, it has gotten much attention lately, making it one of the hottest topics in debates all around the world. Curious of its story? Here are its main advantages and disadvantages:
1. It can eliminate diseases.Though it may seem impossible now, but this technology can take diseases out of the equation. By detecting and removing bad genes inherited from parents, the next generation would be healthier. Any genetic mutations caused by environmental mutagens can be corrected, keeping mutations under control and making the human body less susceptible to infections.
2. It helps the pharmaceutical industry to advance.Aside from paving the way for xenotransplantation or the process of transplanting living tissues or organs through biotechnology, genetic engineering also acts as an aid for genetics, enabling the pharmaceutical industry to develop highly graded products that can help fight health conditions.
3. It has the potential to increase human life span.As human genetic engineering has the potential to make diseases a thing of the past, it can allow for a fuller and healthier life, not to mention a longer life span. As research suggests, it has the ability to increase the life span of humans anywhere between 100 and 150 years by slowing down the aging process through altering the genome of a healthy individual. This technology also pinpoints desirable traits of a certain person and then integrated them to others DNA.
1. It is surrounded with moral issues.The initial reaction of people to the practice of genetic engineering is whether it is morally right or not. As many people religiously believe in God, they see the technology as playing God, and expressly forbid it to be performed on their on their children. Aside from the religious arguments, there are also ethical objections, where opponents believe that diseases exist for a reason. While many of these conditions are to be dealt with, illnesses are generally needed, or else we would soon face the problem of overpopulation.
2. It can limit genetic diversity.Diversity is very important in all species of animals and the ecosystem, and human genetic engineering will a detrimental effect on peoples genetic diversity.
3. It poses possible irreversible effects and consequences.Even among scientists and researchers, genetic engineering is believed to have irreversible side effects, especially in the aspect of hereditarily modified genes. As you can see, the process involves the use of viral factors to carry functional genes to the human body, and naturally, these viral genes will likely leave certain side effects. Moreover, placing functional genes in the genome still does not present definite effects, which means that they can replace other important genes, rather than the mutated ones, causing other forms of health conditions to develop.
Genetic engineering is one of the most controversial topics of today, and keeping yourself informed about all its aspects can help you form a well-informed opinion on the matter.
---
See the article here:
6 Advantages and Disadvantages of Human Genetic ...
Prof John Shine in 2015. Shine discovering a sequence of DNA now called the Shine-Dalgarno sequence which allows cells to produce proteins the basis for how all our cells operate. Photograph: Mal Fairclough/AAP
A man whose discovery was essential for the development of genetic engineering, and used that technology to create several therapies now helping many thousands of people, says receiving a Queens Birthday honour is a great recognition from the community of the value of scientific research.
John Shine started his career by discovering a sequence of DNA now called the Shine-Dalgarno sequence as part of his PhD in the mid 1970s.
That sequence, while a minuscule part of the human genome, allows cells to produce proteins the basis for how all our cells operate.
The discovery was essential for genetic engineering, spawned an entire biotech industry, and has now been used to produce therapies that have helped millions of people. In his own work, Shine used those techniques to clone of human insulin and growth hormone for the first time.
Other scientists honoured on Monday included astronomer Ken Freeman, who founded the field of galactic archaeology, and ethnobotanist Beth Gott.
Shine, who was appointed a Companion of the Order of Australia today, told the Guardian he has been unusually lucky in his career to have been able to oversee discoveries he made in basic sciences, be translated into real therapies and become commercialised.
My PhD was really esoteric research, he said, referring to his discovery of the Shine-Dalgarno sequence . But then I went over to San Francisco when gene cloning was just beginning right place, right time.
Shine had discovered how to clone the human gene that produces insulin, but to make that useful, it needed to be inserted into another organism that could be farmed in this case, bacteria, which would be farmed in large vats.
But if you want to put [the gene] into bacteria to make human insulin, you needed to trick the bacteria into thinking the gene was one of its own, he said.
It turned out Shines earlier discovery of the Shine-Dalgarno sequence was essential for making that final leap. Although the genetic code is the same in animals and bacteria, the regulatory code was very different. Thats where the Shine-Dalgarno sequence comes in, Shine said.
He needed to find the bacterias version of the Shine-Dalgarno sequence, and put that on either side of the human insulin gene, inside the bacteria.
You needed to put the right Shine-Dalgarno sequence just in front in the right place in the insulin gene to make the bacteria produce human insulin.
The fact that both problems were so closely related was mostly an accident, Shine says.
But throughout the rest of his career, Shine continued to be involved in the translation of his discoveries in esoteric science, all the way through to commercialisation.
Since stepping down as the head of the Garvan Institute in 2011 one of Australias top medical research institutes Shine has been the chair of the biotech giant CSL, one of Australasias largest companies.
So Ive come full circle, Shine said. CSL ... in more recent years, were moving into genetic engineering and weve released several genetically modified proteins for haemophilia that are changing the lives of thousands of people around the world.
Ive been very lucky to be able to go through the basic research in my career, and now see a lot of these real health care products come to fruition and improve the lives of thousands of people. Its wonderful when you can have all the excitement of research but also the satisfaction of seeing something very good coming out of it.
It is not the first time Shine has been recognised publicly for his work. In 2010 he won the prime ministers prize for science something his brother Rick Shine won in 2016.
Apart from the obvious personal honour, its a demonstration that the community does appreciate the benefits that come from research, Shine said. The wellbeing of any society is intimately linked to good healthcare.
Another winner of the prime ministers science prize, astronomer Ken Freeman, was appointed a Companion of the Order of Australia for his founding contributions to the field of galactic archaeology and his teaching work at the Australian National Universitys Mount Stromlo Observatory.
Honours were also awarded to Royal Melbourne hospitals Peter Grahame Colman (AM), for his work in endocrinology and diabetes research; aeronautical engineer Graeme Bird (AO), the former department head at the University of Sydney and a NASA consultant for 40 years; and Peter Klinken (AC), the chief scientist of Western Australia.
Ethnobotanist Beth Gott was made a Member of the Order of Australia for her work studying native plants and their use by Indigenous people. Gott founded Monash Universitys Aboriginal education garden in 1986 and has assembled databases of native plants in south-eastern Australia.
A paper she wrote in 2005 for the Journal of Biogreography found Indigenous fire-farming was crucial to the growth of plant tubers in southeastern Australia, allowing them to make up half of the local peoples diet.
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Scientist John Shine honoured for discovery that formed basis of genetic engineering - The Guardian
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On Aug. 3, the scientific article in Nature finally gave us some facts about the much-hyped experiments that involved editing the genomes of human embryos at the Center for Embryonic Cell and Gene Therapy at Oregon Health and Science University. The story had broken in late July in Technology Review, spurring profuse hand-wringing and discussion. But until we saw the scientific paper, it was not clear what cells and methods were used, what genes were edited, or what the results were.
Now we know more, and while the paper demonstrates the possibility of genome editing of human embryos, it raises more questions than it answers. It is a useful demonstration of technical promise, though not an immediate prelude to the birth of a genome-edited baby. But the process by which the news emerged is also an ominous harbinger of the discombobulated way the debate about genetically altering human embryos is likely to unfold. We need open, vigorous debate that captures the many, often contradictory, moral views of Americans. Yet what we are likely to get is piecemeal, fragmented stories of breakthroughs with incomplete details, more sober publication in science journals that appear later, news commentary that lasts a few days, and very little systematic effort to think through what policy should be.
The science underlying this news cycle about human genome editing builds on a technique first developed six years ago by studying how bacteria alter DNA. CRISPR genome editing is the most recent, and most promising, way to introduce changes into DNA. It is faster, easier, and cheaper than previous methods and should eventually be more precise and controllablewhich is why it may one day be available for clinical use in people.
Though headlines about the study discussed designer babies, researchers prefer to emphasize how these techniques could help stop devastating genetic disorders. The Oregon experiments with human embryo cells corrected disease-associated DNA variants associated with heart muscle wasting that can cause heart failure. The treated embryos were alive for only a few days and were never intended to become a human baby. They were, however, human embryos deliberately created for the research.
U.S. guidance in this area is sparse and reflects the lack of societal consensus. In 1994, when the federal government was contemplating funding for research involving human embryos, the NIH Embryo Research Panel concluded that just this kind of experiment was ethically appropriate. But within hours of that reports release, then-President Bill Clinton announced he did not agree with creating embryos in order to do research on them.
The United States currently has just two policies relevant to genomic editing of human embryos. The first blocks federal funding: On April 28, 2015, Francis Collins, director of the National Institutes of Health, stated, NIH will not fund any use of gene-editing technologies in human embryos. This is not embedded in statute or formal executive order, but members of Congress are fully aware of it and it is, in effect, a federal policy. NIH can (and does) fund genome editing of nonembryonic cells that might be used to treat cancer and for other possible therapeutic purposes, but not embryonic cells that would have their effect by creating humans with germline alterations.
Second, Congress has prohibited the Food and Drug Administration from reviewing research in which a human embryo is intentionally created or modified to include a heritable genetic modification. This language comes from a rider to FDAs annual appropriations. Yet use of human embryonic cells for treatment should be subject to FDA regulation. So this language in effect means alterations of embryonic cells cannot be done in the United States if there is any intent to treat a human being, including implantation of an altered embryo into a womans uterus. This will remain true so long as the rider is included in FDAs annual appropriations. The federal government thus has two relevant policies, both of which take federal agencies out of the action: One removes NIH funding, and the other precludes FDA oversight of genome-edited human embryos.
This leaves privately funded research that has no direct therapeutic purpose, such as with the Oregon experiments. The funding came from OHSU itself; South Korean Basic Research Funds; the municipal government of Shenzhen, China; and several private philanthropies (Chapman, Mathers, Helmsley, and Moxie). The research complies with recommendations to study the basic cellular processes of genome editing, keeping an eye on possible future clinical use but only so long as the work does not attempt to create a human pregnancy.
By coincidence, on the same day the Nature paper came out, the American Journal of Human Genetics also published a thoughtful 10-page position statement about germline genome editing from the American Society for Human Genetics endorsed by many other genetic and reproductive medicine organizations from all over the world. It reviews recommendations of the National Academies of Sciences, Engineering, and Medicine, several international and U.S.-based organizations and commissions, and makes several recommendations of its own, concluding it is inappropriate to perform germline gene editing that culminates in human pregnancy, but also there is no reason to prohibit in vitro germline genome editing on human embryos and gametes, with appropriate oversight and consent from donors, to facilitate research on the possible future clinical applications. Indeed, the statement argues for public funding. Finally, it urges research to proceed only with compelling medical rationale, strong oversight, and a transparent public process to solicit and incorporate stakeholder input.
So is there a problem here? It is truly wonderful that medical and scientific organizations have addressed genome editing. It is, however, far from sufficient. Reports and scientific consensus statements inform the policy debate but cannot resolve it. All of the reports on genome editing call for robust public debate, but the simple fact is that embryo research has proven highly divisive and resistant to consensus, and it is far from clear how to know when there is enough thoughtful deliberation to make policy choices. Its significant that none of the reports have emerged from a process that embodied such engagement. The Catholic Church, evangelical Christians, and concerned civic action groups who view embryo research as immoral are not likely to turn to the National Academies of Sciences, Engineering and Medicine, the American Society for Human Genetics, the Hinxton Group, the Nuffield Council on Bioetics, or other scientific and medical organizations for their primary counsel. They may well listen to scientists, but religious and moral doctrine will get greater weight. Yet religious groups highly critical of embryo research are part of the political systemand whether we embrace this sort of genome editing in the United States is a political question, not a purely technical one.
Reports and scientific consensus statements inform the policy debate but cannot resolveit.
Addressing the political questions will be extremely difficult. The U.S. government is poorly positioned to mediate the policy debate in a way that recognizes and addresses our complex moral pluralism. NIH and FDA are two of the most crucial agencies, but current policies remove them from line authority, and with good reason, given that engaging in this debate could actually endanger the agencies other vital missions. International consensus about genome editing of human embryos remains no more likely than about embryo research in general: Some countries ban it while others actively promote and fund it. Private foundations dont have the mandate or incentive to mediate political debate about a controversial technology that rouses the politics of abortion. What private philanthropic organization would willingly take on such a thankless and politically perilous ta
sk, and what organization would be credible to the full range of constituencies?
So who can carry out the public engagement that everyone seems to agree we need? The likely answer is no one. This problem occurs with all debate about fraught scientific and technical innovations, but its particularly acute when it touches on highly ossified abortion politics.
The debate about genomic editing of human embryos is unlikely to follow the recommendations for systematic forethought proposed by illustrious research bodies and reports. Given the reactions weve seen to human embryonic stem-cell research in the past two decades, we have ample reason for pessimism. Rather, debate is more likely to progress by reaction to events as researchers make newsoften with the same lack of information we lived with for the last week of July, based on incomplete media accounts and quotes from disparate experts who lacked access to the details. Most of the debate will be quote-to-quote combat in the public media, leavened by news and analysis in scientific and medical journals, but surrounded by controversy in religious and political media. It is not what anyone designing a system would want. But the recommendations for robust public engagement and debate feel a bit vacuous and vague, aspirations untethered to a concrete framework.
Our divisive political system seems fated to make decisions about genomic editing of human embryos mainly amidst conflict, with experts dueling in the public media rather than through a thoughtful and well-informed debate conducted in a credible framework. As the furor over the Oregon experiments begins to dissipate, we await the event that will cause the next flare-up. And so it will continue, skipping from news cycle to news cycle.
History shows that sometimes technical advances settle the issues, at least for most people and in defined contexts. Furor about in vitro fertilization after Louise Brown, the first test tube baby, was born in 1978 gave way to acceptance as grateful parents gave birth to more and more healthy babies and welcomed them into their families. Initial revulsion at heart transplants gave way in the face of success. Anger about prospects for human embryonic stem-cell research might similarly attenuate if practical applications emerge.
Such historical examples show precisely why reflective deliberation remains essential, despite its unlikely success. Momentum tends to carry the research forward. Yet at times we should stop, learn more, and decide actively rather than passively whether to proceed, when, how, and with what outcomes in mind. In the case of genome editing of human embryos, however, it seems likely that technology will make the next move.
This article is part of Future Tense, a collaboration among Arizona State University, New America, and Slate. Future Tense explores the ways emerging technologies affect society, policy, and culture. To read more, follow us on Twitter and sign up for our weekly newsletter.
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Listening for the Public Voice - Slate Magazine
QUESTION: What are the benefits of human genetic engineering?
ANSWER:
The benefits of human genetic engineering can be found in the headlines nearly every day. With the successful cloning of mammals and the completion of the Human Genome Project, scientists all over the world are aggressively researching the many different facets of human genetic engineering. These continuing breakthroughs have allowed science to more deeply understand DNA and its role in medicine, pharmacology, reproductive technology, and countless other fields.
The most promising benefit of human genetic engineering is gene therapy. Gene therapy is the medical treatment of a disease by repairing or replacing defective genes or introducing therapeutic genes to fight the disease. Over the past ten years, certain autoimmune diseases and heart disease have been treated with gene therapy. Many diseases, such as Huntington's disease, ALS (Lou Gehrig's disease), and cystic fibrosis are caused by a defective gene. The hope is that soon, through genetic engineering, a cure can be found for these diseases by either inserting a corrected gene, modifying the defective gene, or even performing genetic surgery. Eventually the hope is to completely eliminate certain genetic diseases as well as treat non-genetic diseases with an appropriate gene therapy.
Currently, many pregnant women elect to have their fetuses screened for genetic defects. The results of these screenings can allow the parents and their physician to prepare for the arrival of a child who may have special needs before, during, and after delivery. One possible future benefit of human genetic engineering is that, with gene therapy, a fetus w/ a genetic defect could be treated and even cured before it is born. There is also current research into gene therapy for embryos before they are implanted into the mother through in-vitro fertilization.
Another benefit of genetic engineering is the creation pharmaceutical products that are superior to their predecessors. These new pharmaceuticals are created through cloning certain genes. Currently on the market are bio-engineered insulin (which was previously obtained from sheep or cows) and human growth hormone (which in the past was obtained from cadavers) as well as bio-engineered hormones and blood clotting factors. The hope in the future is to be able to create plants or fruits that contain a certain drug by manipulating their genes in the laboratory.
The field of human genetic engineering is growing and changing at a tremendous pace. With these changes come several benefits and risks. These benefits and risks must be weighed in light of their moral, spiritual, legal, and ethical perspectives. The potential power of human genetic engineering comes with great responsibility.
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Benefits of Human Genetic Engineering - Popular Issues