Category Archives: Genetic Engineering

Gene and cell therapies are an emerging therapy class. Here, GEN talks to Nneka Onwudiwe, PharmaD, PhD, the PRO/PE Regulatory Review Officer for the FDA, about current and future challenges for manufacturing regulation.

GEN: What do you feel are the main areas where the gene and cell therapy industry are working to meet regulatory requirements?

Onwudiwe: The manufacturing processes [in these industries] are in their infancy, so a lot of validation is needed. [This is the case,] for example, with starting materials, especially if a manufacturer is starting with a small batch and is scaling up ready for commercialization.

Even when a product makes it to clinic, there are lots of other issues that need to be considered. For example, how do you store it within a critical range of temperatures? And how do you ensure safe delivery to clinical sites?

GEN: What do you think is the biggest challenge facing manufacturers in this sector?

Onwudiwe: Capacity constraints is the biggest challenge. If regulations require certain manufacturing processes or additional testing, that becomes a bottleneck if the capacity isnt thereas the manufacturer hasnt anticipated it.

GEN: How would you recommend manufacturers respond?

Onwudiwe: Its important for companies to engage with the health authority at an early stage because the last thing a manufacturer wants to find out is that either their manufacturing process is not in compliance or theres some issue with sourcing raw materials.

The FDA has put out several guidances around cell and gene therapy, but theyre not prescriptive. And, so, manufacturers still need to engage [with the agency] because there may be nuances around their molecule or development process. As these therapies are in their infancy (20 years in this space is still new) then there are things we are still learning. Manufacturers need to have adequate resources to anticipate challenges and a designated team of regulatory professionals who really understand the regulatory pathway for their molecule and can engage with the FDA at each step.

GEN: Where do you see future challenges arising?

Onwudiwe: I think theres a shift, not only in the biotechnology industry, but across other industries too in terms of sourcing much-needed raw materials [due to global supply chain issues]. Thats where I see the challenge in the next couple of years. And that will remain a challenge even if manufacturers have a good understanding of the manufacturing process for their molecule and its been validated by the FDA.

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Outlining the Latest Regulatory Trends in Advanced Therapies, Like Cell and Gene Therapy - Genetic Engineering & Biotechnology News

Saturday, 23 July 2022, 1:12 pmPress Release: GE Free NZ

The Green Party are being called on to support a new ToiTe Taiao with an expanded role to include other emergingtechnologies; Synthetic Biology, Artificial Intelligence,Nano-technology and Geo-engineering.

In an open letterto Green Party members and MPs, former Green Party co-leaderJeanette Fitzsimons is acknowledged for her work in policydevelopment that ensures robust regulation of GeneticEngineering in Aotearoa. [1]

Jeanette advocated forThe Royal Commission on Genetic Modification which led toToi Te Taiao - The Bioethics Council beingestablished.

Toi Te Taiao was tasked with helpingAotearoa navigate the ethical issues of Genetic Engineering,including the use of human genes, but their work ended whenToi Te Taiao was abolished. [2]

Today a new TechnologyEthics Council is needed to navigate the challenges ofinnovation including 'easy, cheap' Gene Editing, SyntheticBiology, Nanotechnology, Artificial Intelligence andGeo-Engineering.

These require deep and ongoingengagement of the public. The abolition of Toi Te Taiaodeprived us of a source of collective wisdom and authenticengagement with tangata whenua and civil society, saidJon Carapiet. spokesman for GE-Free NZ.

Anotherrecommendation of The Royal Commission on GeneticModification was that farm animals should not be GeneticallyEngineered as 'bioreactors' for bulk production ofpharmaceuticals. But the recent call by the ProductivityCommission for deregulation opens up the potential forunacceptable cruelty to animals from GE already seen inAgResearch experiments. [3]

When Jeanette startedher work, it was in the knowledge that commercial interestswere already keen to commercialise these powerful emergingtechnologies, said Jon Carapiet.

To maintainpublic trust an independent body, such as Toi Te Taiao, isneeded to balance the voices of vested interests in bothindustry and academia.

Strict liability is alsoneeded to reign in the appetite for risk that is prevalentin the biotechnology and innovation sectors, driven byIntellectual Property not the public interest.

Astechnologies challenge the integrity of Natural systems andhumanity itself a Technology Ethics Council is needed toguide how society manages risk while navigatingopportunities and regulation in the publicinterest.

Medical use of GE in humans show benefitsand there is a review of settings for regulation inbiomedicine. But the prospect of human Genetic Engineeringalso brings the threat of Eugenics to humanity, including tothe LGBTQIA+ rainbow and disability communities, which mustbe addressed.

These are complex and ongoing issues tonavigate. Having an independent and ethical compass to guideus is vital.

[1] Open Letter to the Green Party The Bioethics Council[2] https://pep.org.nz/wp-content/uploads/2017/03/human-genes.pdf

[3]https://www.nzherald.co.nz/nz/mutant-cows-die-in-gm-trial/UNAM73ED3VXZJFX7MXU7VYAZ74/

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Greens Called To Reinstate Toi Te Taiao -The Bioethics Council | Scoop News - Scoop

There are more viruses in the universe than stars. To get an idea of how big a quantity is, Anyone reading this article would be sitting on top of more than 800 million viruses,

These tiny infectious agents have been with us long before they emerged as a species. And the debate about their presence on Earth is not yet closed: do they predate bacteria, or are they halfway between them and eukaryotic cells?

We dont know yet, but what we do know is that theyve been on everyones lips during these last two years. Its nearly impossible to turn on the television, read the newspaper, or walk into a bar without hearing the word virus.

Viruses are very different from each other, and can infect both human cells and bacteria and even plants, causing endless diseases. Who hasnt caught a cold or the flu and has to spend a few days without getting out of bed?

TookThe history of viruses parallels the history of the development of modern medicine, So much so that nothing has generated such a massive amount of global and unitary effort to achieve protection against them: Vaccination,

In addition, in our recent history, we have achieved another milestone with respect to the virus. Thanks to science, were able to turn the tables and a priori turn an evil agent into his nemesis: a drug.

Some are diseases caused by errors in the genome, including cancer. These errors or mutations cause genes within cells to stop functioning, preventing it from doing its job and causing various problems that end in pathology.

Gene therapy is responsible for the treatment of this type of disease. The idea behind this therapeutic strategy is quite simple: if the genes function has been eliminated, why not carry a copy of the good, functional gene into the cells? That way they can recover and continue to fulfill their mission.

The question is how to do it. The first obstacle in our journey to the nucleus of the cell, where genetic information is stored, is the plasma membrane, an envelope that maintains and protects all cellular components.

To pass this without harm to our genes, lipid vesicles can be used, which due to their nature are integrated into the membrane, releasing their contents towards the interior. Another possibility is to use an electrical discharge that produces transient pores on the surface of the cell.

However, wouldnt it be much cheaper to take advantage of mechanisms already in nature? One that does exactly this: introduce genetic material into human cells? We, of course, mention the virus.

Viruses are able to infect human cells and introduce their genome into them. They use the host cells own machinery to replicate and are thus able to infect nearby cells, until the bodys immune system is able to eliminate this new viral strain.

Thanks to advances in genetic engineering, scientists have been able to modify the genomes of viruses and clip their wings. i.e. transform them so that they can infect but not replicate.

And not only that, but it has also been possible for these agents to carry the genes they want within themselves without waking up the immune system and causing an adverse reaction. In this way it has been achieved that they employ a taxi, a train or a bus carrying passengers (pharmaceuticals) to the destination which one chooses.

Thanks to these advances, diseases that previously had no cure can be treated, such as hereditary retinal dystrophy, which causes vision loss in children and adults due to mutations in the RPE65 gene. Through an injection, the modified virus that contains a functional copy of this gene will travel to the retinal cells, infect them and deposit the dominant copy inside them.

A more recent example is clinical trials that are being done to treat a range of open wounds Those so-called butterflies appear on the whole body of children. These patients lack the gene for collagen VII and, thanks to the modified type I herpes simplex virus, the functional gene can be carried into skin cells, leading to wound closure.

Currently, the number of diseases that can be treated with gene therapy are counted on the fingers of one hand. However, we need many fingers to count the number of clinical trials being conducted in the hope that one day these treatments may reach hospital wards.

Immune response to treatment, drug specificity and its administration are the main challenges that hinder the progress of these trials and gene therapy approaches to patients.

The simple idea of turning one disease-causing agent into another that cures them has succeeded in restoring function to organs that had forgotten how to function. It has given hope to patients whose medical options were scarce and have been able to improve their living conditions.

With time, effort and financial investment, this drawing rooted in science will help to solve various diseases.

,

This article was a finalist in the second edition of the youth promotion competition organized by the Lille Foundation and The Conversation Spain.

This article was originally published on The Conversation. read the original.

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What Doesn't Kill Us Can Make Us Stronger: Gene Therapy With Viruses - Nation World News

BANGKOK: Thailand is considering raising further its health alert for monkeypox by listing it as a so-called serious communicable disease, after the viral disease was classified as a new public health emergency worthy of international concern.

Anutin: No travel curbs for now. Photo: Bangkok Post

The World Health Organisation (WHO) on Saturday (July 23) declared monkeypox as A Public Health Emergency of International Concern and urged member countries to find effective methods that do not stigmatise target groups, reports the Bangkok Post.

The Ministry of Public Health yesterday raised surveillance measures nationwide in response to WHOs announcement, said Public Health Minister Anutin Charnvirakul.

He was speaking after chairing an urgent meeting with health authorities in which they discussed responses to the monkeypox outbreak.

This comes after detection of the first confirmed case in the country, a Nigerian tourist who escaped into neighbouring Cambodia after he was found to have the disease before being nabbed there.

The man has been detained in Cambodia while tracing of his close contacts in Phuket where he stayed in the past month is under way. None have been found infected by monkeypox, said Anutin.

As none of the Nigerian mans close contacts are infected, its a relief to some extent, he said.

We will meet again within hours so the ministrys academic committee comprising medical and public health experts can discuss a proposal to declare monkeypox a new serious communicable disease in Thailand, he said.

Anutin also indicated there are no plans for Thailand to seek the return of the Nigerian man from Cambodia.

Anutin said yesterday he expected the patient to receive treatment in Cambodia. Its good to see him treated there, he said, adding he expected his condition would improve.

In Cambodia, Khuong Sreng, governor of Phom Penh, urged three people, believed to have travelled with Osmond Chihazirim Nzerem, the Nigerian patient, in the same car to Phnom Penh to report to local health authorities.

They should take a health check as they may have contracted the disease, he said, according to the Khmer Times.

The Nigerian man was caught on a security camera when arriving at a guesthouse in Phnom Penh at 5:20am on Saturday accompanied by an African man and an Asian woman, said Cambodian police.

He was caught at 5:30pm at Doeum Thkov market in Phom Penh and taken to the KhmerSoviet Friendship Hospital for treatment and isolation, said the report.

The Thai Department of Disease Control on Sunday began a joint epidemiologic investigation with its Cambodian counterparts, tracking more possible contacts of the man, said a public health source.

As a precautionary measure, disease control and immigration checkpoints nationwide have been instructed to scale up screening for monkeypox, particularly among visitors coming from countries with a high risk of the disease, said Anutin.

However, no travel restrictions are being considered for the time being, he said.

The minister urged the public to not panic about the possible spread of monkeypox in Thailand, saying the same prevention measures used in preventing COVID-19 are sufficient for fending off monkeypox.

Wearing a face mask, washing hands frequently and practising social distancing help protect against both diseases at the same time, he said.

In most cases, patients fully recover from monkeypox after being treated, he said, adding that negative pressure rooms arent necessary.

Before the Nigerian man was confirmed to be Thailands first monkeypox case, a total of 18 foreign visitors to Thailand who had been admitted to private hospitals for treatment were treated as suspected cases of monkeypox infection, said Dr Tares Krassanairawiwong, director-general of the Department of Health Service Support (DHSS).

In another development, Bangkok governor Chadchart Sittipunt said City Hall will meet today to discuss raising preventive measures against monkeypox and scaling up surveillance in the capital.

Certain areas in the city may require special attention and extra disease surveillance measures, he said, apparently referring to communities of foreign visitors coming from countries with high risk of monkeypox.

Anan Jongkaewwattana, a virologist with the National Centre for Genetic Engineering and Biotechnology (Biotec), meanwhile, warned that using a condom while having sex is not sufficient protection against monkeypox.

Although HIV transmissions and monkeypox share some similarities, using a condom alone wont be enough to protect against monkeypox as does it in protecting against HIV, he said.

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Government on high monkeypox alert - Thailand - The Phuket News

image:Topics cover both human and animal viral immunology, exploring viral-based immunological diseases, pathogenic mechanisms, and virus-associated tumor and cancer immunology. view more

Credit: Mary Ann Liebert, Inc., publishers

A new study examined the maintenance of memory B cell responses to SARS-CoV-2, the virus that causes COVID-19, after recovery from natural infection or post-vaccination. The study is published in the peer-reviewed journal Viral Immunology. Click here to read the article now.

The study, coauthored by David Fear, from Kings College London, and colleagues, showed that among those recovered from natural infection, COVID-19 serologically-positive donors had strong antigen-specific memory B cell-associated responses. Post-vaccination, donors showed robust serological antigen-specific antibody responses against spike protein that waned over time. Memory B cell-associated responses against spike protein were also observed but showed less waning over time.

This study is of particular relevance at the moment, because with millions vaccinated, previously infected, or both, studies such as this one may tell us how long we might expect the immunity to last, says Rodney S. Russell, PhD, Editor-in-Chief of Viral Immunology, from Memorial University of Newfoundland, St. Johns.

About the Journal

Viral Immunology is an authoritative peer-reviewed journal published ten times a year in print and online. Topics cover both human and animal viral immunology, exploring viral-based immunological diseases, pathogenic mechanisms, and virus-associated tumor and cancer immunology. The Journal includes original research papers, review articles, and commentaries covering the spectrum of laboratory and clinical research and exploring developments in vaccines and diagnostics targeting viral infections. Tables of content and a sample issue may be viewed on the Viral Immunology website.

About the Publisher

Mary Ann Liebert, Inc., publishers is known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industrys most widely read publication worldwide. A complete list of the firms more than 100 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Case study

People

SARS-CoV-2-specific memory B cell responses are maintained after recovery from natural infection and postvaccination

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

Excerpt from:
Maintenance of immunity to COVID-19 after infection or vaccination - EurekAlert

Replay Bio

Replay Launches with $55 Million Seed to Reprogram Biology by Writing and Delivering Big DNA

KKR and OMX Ventures lead $55 million seed funding round

Replays genomic medicine toolkit encompasses a portfolio of disruptive platform technologies to write and deliver big DNA, including a high payload capacity HSV delivery platform, a hypoimmunogenic cell therapy platform, and a proprietary genome writing platform

Company is led and supported by a world-class team of academics, entrepreneurs, and industry experts

Innovative hub-and-spoke business model separates technology development from product development, leveraging Replays technologies within discrete product companies

Five product companies have been incorporated to date

San Diego, California and London, UK, 25 July 2022 Replay, a genome writing company reprogramming biology by writing and delivering big DNA, today announced its launch with $55 million in seed financing. The round was led by KKR and OMX Ventures, with additional participation from ARTIS Ventures and Lansdowne Partners, SALT, DeciBio Ventures, and Axial.

Replays portfolio of next-generation genomic medicine technologies aims to solve the key challenges currently limiting clinical progress, including the need for increased payload capacity and off-the-shelf cell therapies that substantially reduce cost of goods, improve production speed, volume and consistency, and expand the potential for genome engineering.

Replays genomic medicine toolkit comprises several synergistic technology platforms, including:

synHSV - a high payload capacity HSV vector able to deliver up to 30x the payload of AAV. synHSV facilitates the delivery of large genes, genomic genes, multiple genes, and multiple transcriptional activators and repressors, thereby extending the reach of genomic medicine and opening up the possibility of polygenic therapy

uCell - a universal, renewable, off-the-shelf, genomically rewritten, hypoimmunogenic iPSC-derived cell source for regenerative medicine and cell therapy

DropSynth - a genome writing platform enabling rapid, efficient, and low-cost synthesis of libraries of synthetic genes and big DNA

LASR - an evolutionary inference algorithm platform for rewriting proteins to optimize functionality

Story continues

Replays innovative corporate structure separates technology development from therapeutic product development within disease area-specific product companies. Each product company is co-founded by seasoned entrepreneurs in conjunction with global thought leaders in each therapeutic area. To date, Replay has established four synHSV gene therapy product companies, aimed at bringing big DNA therapies to monogenic diseases affecting the skin, eye, brain and muscle, and an enzyme writing product company using LASR and DropSynth to optimize enzyme functionality.

Replay was co-founded by Dr. Adrian Woolfson BM BCh PhD, formerly Executive Vice President and Head of Research and Development at Sangamo Therapeutics, Chief Medical Officer at Nouscom, Global Clinical Leader of Early and Late Stage Immuno-Oncology/Hematology at Pfizer and Global Medical Lead in Oncology at Bristol Myers Squibb; Lachlan MacKinnon, a member of the founding team at Oxford Science Enterprises (formerly OSI) and founding investor in Base Genomics, ONI and OMass Therapeutics; Professor David Knipe PhD, a world-renowned virologist and pioneer of HSV research; and Professor Ron Weiss PhD, one of the pioneers of synthetic biology and Professor of Biological Engineering at Massachusetts Institute of Technology (MIT).

Adrian Woolfson, Executive Chairman, President, and Co-founder of Replay, commented: Genomic medicine has the potential to transform the future of clinical therapeutics. Over my three decades of experience working in clinical medicine, academia, and the biopharmaceutical industry, it has become clear that we require a more robust and comprehensive toolkit of molecular genetic platform technologies to solve biologys most complex problems and realize its full therapeutic potential. In Replay we have assembled a world-class team of entrepreneurs, subject matter experts, and cutting-edge genomic medicine and synthetic biology technologies into a coherent structure that will enable us to address medicines greatest challenges, including solid tumors and polygenic diseases.

Lachlan MacKinnon, Chief Executive Officer, and Co-founder of Replay, added: Technology and product development have different talent requirements, timelines, costs and cultures. By separating technology development from product development, we have generated a model to accommodate these differences. Our ability to write and deliver big DNA has the potential to disrupt many areas of genomic medicine. We have the right team, corporate structure, portfolio of technology platforms, and financial backing to build an enduring company that shapes the future of the industry.

Kugan Sathiyanandarajah, Managing Director at KKR and Board Member at Replay, said: Replays mission is to create a world-leading company that develops and owns the tools to reprogram biology by writing and delivering big DNA; we believe these capabilities will unlock the largest untapped opportunity in medicine. Replay has tremendous entrepreneurial experience within the Company, as well as a team of seasoned industry players to guide the development of the platform technologies and product companies to bring new treatments to patients.

Nick Haft, Managing Director at OMX Ventures and Board Observer at Replay, added: Replay has assembled an impressive portfolio of step-change technologies to propel the field of genomic medicine forward. We are excited to support these technologies, Replays creative business model and the excellent team of entrepreneurs and investors that brings it all together.

Errik Anderson, CEO of Alloy Therapeutics and Independent Board Member at Replay, stated: Substantial technological advances in biotechnology often create opportunities for new business models. I am very excited to partner with Replays ambitious founders and investors who have devised a new structure around the significant opportunity space afforded by synHSV, uCell, and Replays related genomic medicine and synthetic biology technologies.

Alongside a highly experienced management team and board, which includes serial entrepreneur Errik Anderson, Replay is supported by a distinguished team of entrepreneurs and international experts including product company co-founders: Professor Joe Glorioso PhD, inventor of Replays synHSV technology and Senior Advisor for Gene Therapy Programs at Replay, Co-founder of Oncorus, and Professor of Microbiology and Molecular Genetics at the University of Pittsburgh; Mark Blumenkranz, MD, MMS, the HJ Smead Professor of Ophthalmology, Emeritus, at the Stanford School of Medcine, Co-Director of the Stanford Opthalmology Innovation Program, and former Chairman of the Board and Co-founder of Adverum Biotechnologies; Professor Howard Federoff MD PhD, Co-Founder of Brain Neurotherapy Bio, and former CEO of Aspen Neuroscience and Brooklyn Immunotherapeutics; and Professor David Schaffer PhD, Professor of Chemical and Biomolecular Engineering, Bioengineering and Neuroscience at University of California, Berkeley, and Co-founder of 4D Molecular Therapeutics.

KKR is investing in Replay through KKR Health Care Strategic Growth Fund II, a $4.0 billion fund focused on investing in high-growth health care companies.

Ends

About Replay

Replay is a genome writing company, which aims to define the future of genomic medicine through reprogramming biology by writing and delivering big DNA. The Company has assembled a toolkit of disruptive platform technologies including a high payload capacity HSV platform, a hypoimmunogenic platform, and a genome writing platform to address the scientific challenges currently limiting clinical progress and preventing genomic medicine from realising its full potential. The Companys hub-and-spoke business model separates technology development within Replay from therapeutic development in product companies, which leverage the technology platforms. For example, Replays synHSV technology, a high payload capacity HSV vector capable of delivering up to 30 times the payload of AAV, is utilized by Replays four gene therapy product companies, bringing big DNA treatments to diseases affecting the skin, eye, brain, and muscle. The Company has, additionally, established an enzyme writing product company engaging its evolutionary inference machine learning and genome writing technology to optimize functionality. Replay is led by a world-class team of academics, entrepreneurs and industry experts.

The Company has raised $55 million in seed financing and is supported by an international syndicate of investors that includes KKR, OMX Ventures, ARTIS Ventures, and Lansdowne Partners.

Replay is headquartered in San Diego, CA and London, UK. For further information please visit http://www.replay.bio and follow us on LinkedIn and Twitter.

About KKR

KKR is a leading global investment firm that offers alternative asset management as well as capital markets and insurance solutions. KKR aims to generate attractive investment returns by following a patient and disciplined investment approach, employing world-class people, and supporting growth in its portfolio companies and communities. KKR sponsors investment funds that invest in private equity, credit and real assets and has strategic partners that manage hedge funds. KKRs insurance subsidiaries offer retirement, life and reinsurance products under the management ofGlobal Atlantic Financial Group. References to KKRs investments may include the activities of its sponsored funds and insurance subsidiaries. For additional information aboutKKR & Co. Inc.(NYSE: KKR), please visit KKRs website atwww.kkr.com and on Twitter.

About OMX Ventures

OMX Ventures is an early stage, tech-bio focused venture capital fund a force multiplier for scientists and innovators pushing the boundaries of whats possible in biology and beyond. Visit OMX Ventures website at OMX.VC and follow us on LinkedIn and Twitter.

Contacts:

ReplayDr. Adrian Woolfson/Lachlan MacKinnoninfo@replay.bio

Consilium Strategic Communications Media relationsAmber Fennell/Tracy Cheung/Jessica Hodgsonreplay@consilium-comms.com

KKRAlastair Elwen/Sophia JohnstonFinsbury Glover HeringKKR-LON@fgh.com+44 20 7251 3801

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Replay Launches with $55 Million Seed to Reprogram Biology by Writing and Delivering Big DNA - Yahoo Finance

Hemophilia B is a rare genetic condition caused by a lack of blood clotting factor IX (FIX). Since itprevents blood from clotting effectively, the disease leads to uncontrollable bleeding that can be life-threatening. While most people who suffer from the disease inherit the genetic condition from their parents, approximately one-third of cases are brought on by spontaneous gene mutations.

But now, a new gene therapy could be the key tocutting the risk. In a small-scale trial has been conducted over six months, nine out of ten subjects with severe or moderately severe hemophilia responded to a single treatment one round of FLT180a therapy.It led to the continuous hepatic synthesis of the protein, meaning the participants no longer needed regular injections.

Though the treatment is in its early stages and the trial involved very few people,"[w]e're very excited by the results," said Pratima Chowdary, M.D., one of the doctors who developed the treatment. She also believes that curing hemophilia "will be a reality for the majority of the adults in the next one to three years,"according to BBC.

Elliott Mason, who was one of the subjects of the trial, says the treatment has made his life "completely normal," adding, "I've not had any treatment since I had my therapy, it's all a miracle really, well, it's science, but it feels quite miraculous to me."

The current treatment of hemophilia includes getting injections regularly,generally once a week, tomake up for the lack of clotting factor IX.However, even that onerous treatment doesn't always prevent debilitating joint damage.

The new method, on the other hand,delivers several copies of the gene that codes for the clotting factor to liver cells. Rather than requiring the protein from an outside source, it provides the body with the blueprints needed to produce it on its own.So that a single injection of gene treatment could treat the condition for the long term.

Mason also toldBBC that receiving the treatment took about an hour.

The results of the clinical trial have demonstrated that out of the 10 individuals, five patients had normal levels of blood-clotting factors while the other three had elevated levels that were still below normal. Only one of them had excessively high factor IX levels, which caused a blood clot to form.

"Removing the need for hemophilia patients to regularly inject themselves with the missing protein is an important step in improving their quality of life," Chowdary stated in a press release.

"This initial data is promising, but we continue to monitor gene therapy trials closely and cautiously, as with all new treatments," Clive Smith, chairman of the Haemophilia Society told BBC. "If they are shown to be safe and effective, NICE [National Institute for Health and Care Excellence] and the NHS [National Health Service] must work together to make these innovative treatments available."

Though the study offers hope for the treatment of hemophilia B, it's been tested on a very small group of subjects. Importantly, one of the patients suffered from aserious blood clot. Therefore, the next stepis the Phase III trial which is testing the treatment on a wider group of people.

The results of the study have been published in the journalthe New England Journal of Medicine.

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A new gene therapy could pave the way to finally curing hemophilia B - Interesting Engineering

The new report by Expert Market Research titled, Global Non-GMO Seeds Market Report and Forecast 2022-2027, gives an in-depth analysis of the globalnon-GMO seeds market growth, assessing the market based on its segments like natures, forms, seed types, packaging types, and major regions. The report tracks the latest trends in the industry and studies their impact on the overall market. It also assesses the market dynamics, covering the key demand and price indicators, along with analyzing the market based on the SWOT and Porters Five Forces models.

Request a free sample copy in PDF or view the reportsummary@https://bityl.co/CUIX

The key highlights of the report include:

Market Overview (2017-2027)

Farmers are increasingly adopting non-GMO seeds since they can save money while boosting their yields, which is driving the market growth. With the increasing preference for clean and better-produced food goods with non-GMO Project Certified certifications over GMO food products by consumers due to their cost-effectiveness and more sustainable production, the market is growing. The rising popularity of vegetarian and vegan lifestyles, along with the increasing health-consciousness, is surging the demand for foods manufactured using organic and non-GMO seeds. Over the forecast period, the popularity of organic food products is expected to expand, which is anticipated to aid the growth of the non-GMO seeds industry.

Industry Definition and Major Segments

Non-GMO seeds, variously known as non-genetically modified seeds, refer to seeds whose DNA are not modified by genetic engineering. They are typically cultivated through natural processes like pollination. They also maintain soil and crop health without utilising fertilisers and pesticides. Moreover, non-GMO seeds are more cost-effective than their genetically modified counterparts, due to which they are extensively adopted in the agricultural industry.

Explore the full report with the table ofcontents@https://bityl.co/CUIW

By nature, the market is divided into:

Based on form, the market is categorised into:

The market, based on seed type, is segmented into:

On the basis of packaging type, the market is divided into:

The regional markets for the product include:

Market Trends

As non-GMO seeds are cost-effective than genetically modified seeds, they are increasingly preferred by farmers, which is bolstering the industry growth. The favourable agriculture economics of non-GMO seeds are surging the demand for their end-use products in the food processing industry, therefore propelling the market growth. With the growing focus on high-yielding crops and seeds to meet the rising food demand for the increasing global population, the demand for non-GMO seeds owing to their high yield is significantly surging. Furthermore, the increasing launches of non-GMO products in the food and beverage industry due to the emerging trend of health and wellness is predicted to propel the market growth in upcoming years.

Key Market Players

The key players in the market are Cargill Inc., BASF SE, Prairie Hybrids, Spectrum Premium Non-GMO, Albert Lea Seed, and Sentinel Seeds, LLC, among others. The report covers the market shares, capacities, plant turnarounds, expansions, investments and mergers and acquisitions, among other latest developments of these market players.

About Us:

Expert Market Research (EMR) is leading market research company with clients across the globe. Through comprehensive data collection and skilful analysis and interpretation of data, the company offers its clients extensive, latest and actionable market intelligence which enables them to make informed and intelligent decisions and strengthen their position in the market. The clientele ranges from Fortune 1000 companies to small and medium scale enterprises.

EMR customises syndicated reports according to clients requirements and expectations. The company is active across over 15 prominent industry domains, including food and beverages, chemicals and materials, technology and media, consumer goods, packaging, agriculture, and pharmaceuticals, among others.

Over 3000 EMR consultants and more than 100 analysts work very hard to ensure that clients get only the most updated, relevant, accurate and actionable industry intelligence so that they may formulate informed, effective and intelligent business strategies and ensure their leadership in the market.

Media Contact

Company Name: Claight CorporationContact Person: Louis Wane, Corporate Sales Specialist U.S.A.Email:sales@expertmarketresearch.comToll Free Number:+1-415-325-5166 | +44-702-402-5790Address: 30 North Gould Street, Sheridan, WY 82801, USAWebsite:https://www.expertmarketresearch.com

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K-12 Online Tutoring Market: https://www.designerwomen.co.uk/global-k-12-online-tutoring-market-to-be-driven-by-the-growing-popularity-of-online-microlearning-in-the-forecast-period-of-2021-2026/

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Global Non-GMO Seeds Market To Be Driven By Increasing Health Consciousness Among Consumers In The Forecast Period Of 2022-2027 This Is Ardee - This...

The Research Brief is a short take about interesting academic work.

Damage to the ends of your chromosomes can create zombie cells that are still alive but cant function, according to our recently published study in Nature Structural and Molecular Biology.

When cells prepare to divide, their DNA is tightly wound around proteins to form chromosomes that provide structure and support for genetic material. At the ends of these chromosomes are repetitive stretches of DNA called telomeres that form a protective cap to prevent damage to the genetic material. However, telomeres shorten each time a cell divides. This means that as cells divide more and more as you age, your telomeres become increasingly shorter and more likely to lose their ability to protect your DNA.

Damage to genetic material can lead to mutations that cause cells to divide uncontrollably, resulting in cancer. Cells avoid becoming cancerous when their telomeres become too short after dividing too many times and potentially accruing damage along the way, however, by entering a zombielike state that stops cells from dividing through a process called cellular senescence.

Because they are resistant to death, senescent or zombie cells accumulate with age. They can be beneficial to health by promoting senescence in nearby cells at risk of becoming cancerous and attracting immune cells to clear out cancer cells. But they can also contribute to disease by impairing tissue healing and immune function and by secreting chemicals that promote inflammation and tumor growth.

We wanted to know if direct damage to telomeres can be sufficient to trigger senescence and make zombie cells. In order to figure this out, we needed to confine damage just to the telomeres. So we attached a protein to the telomeres of human cells grown in the lab. Then we added a dye to the protein that makes it sensitive to light. Shining a far-red light (or light with a wavelength slightly shorter than infrared light) on the cells induces the protein to produce oxygen free radicals highly reactive molecules that can damage DNA right at the telomeres, sparing the rest of the chromosome and the cell.

We found that direct damage to the telomeres was sufficient to turn cells into zombies, even when these protective caps werent shortened. The reason for this, we discovered, was likely a result of disrupted DNA replication at the telomeres that leaves chromosomes even more susceptible to damage or mutations.

Telomeres naturally shorten with age. They limit how many times a cell can divide by signaling cells to become zombies when they reach a certain length. But an excess of free radicals produced from both normal bodily processes as well as exposure to harmful chemicals like air pollution and tobacco smoke can lead to a condition called oxidative stress that can accelerate telomere shortening. This can prematurely trigger senescence and contribute to age-related diseases, including immunodeficiency, cardiovascular disease, metabolic disease, and cancer.

Our study reveals that telomeres not only serve as alarm clocks that indicate a cell divided too many times but also as warning bells for harmful levels of oxidative stress. Age-related shortening of telomeres isnt the only thing that triggers senescence; telomere damage is also sufficient to turn a cell into a zombie.

Researchers are studying treatments and interventions that can protect telomeres from damage and prevent zombie cell accumulation. A number of studies in mice have found that removing zombie cells can promote healthy aging by improving cognitive function, muscle mass and function, and recovery from viral infections.

Researchers are also developing drugs called senolytics that can either kill zombie cells or prevent them from developing in the first place.

This study focuses on the consequences of telomere damage in actively dividing cells, like kidney and skin cells. Were now looking at how this damage will play out in cells that dont divide, like neurons or heart muscle cells. While researchers have shown that the telomeres of nondividing cells and tissues become more dysfunctional with age, its unclear why this happens when these telomeres should not be shortening in the first place.

Patricia Opresko, Professor of Environmental and Occupational Health, University of Pittsburgh Health Sciences, and Ryan Barnes, Postdoctoral Researcher in Environmental and Occupational Health, University of Pittsburgh Health Sciences

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Zombie cells fight against cancer as you age here's why researchers want to kill them - Interesting Engineering

Its been 30 years since I was asked to do a study comparing organic farming and conventional production and the use of genetically modified breeds such as transgenic Bt maize.At that time, theCenter for Biosafety and Sustainabilityin Basel was conducting a study to assess the impact of genetic engineering.Exactly the same questions were discussed as today.

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Since these first debates, there have been countless national and EU-wide studies on the risks and potential of genetic engineering involving scientists, politicians, NGOs, consumers and organic and conventional farmers.The data is clear: In principle, the new breeding methods do not differ from classic crossbreeding in terms of their effect on (agricultural) ecosystems and human health.

30 years of scientific progress, countless studies involving dialogue with society, thousands of forums and debates for which scientists left their laboratories, and yet: a majority of the population is still convinced that we do not yet know enough to Open Pandoras Box. The image of a risky technology with little benefit for the great challenge of the century to ensure safe feeding of soon ten billion people while at the same time greatly reducing the consumption of the natural resources of air, water, soil and biodiversity persists.

[Editors note: This article has been translated from German and edited for clarity.]

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By 2050, there will be 10 billion people to feed. To meet the challenge, we need all technologies on deck including molecular biology and genetic...