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Category Archives: Gene Medicine

Gene Editing System Revamped to Target RNA Aggregates Found in Inherited ALS – ALS News Today

Posted: August 15, 2017 at 11:45 am

Researchers have found a way to break down aggregated RNA molecules that cause diseases such as certain inherited forms of amyotrophic lateral sclerosis (ALS).

As the technique has the potential to treat several diseases which currently lack treatment options, the research team from theUniversity of California, San Diego (UCSD) made sure to engineer the new system so that it could be delivered to specific tissues with non-infectious viruses.

The method builds on a well-known gene-editing system, called CRISPRCas9, but was adapted to target RNA instead of DNA. The new method is called RNA-targeting Cas9, or simply, RCas9.

This is exciting because were not only targeting the root cause of diseases for which there are no current therapies to delay progression, but weve re-engineered the CRISPR-Cas9 system in a way thats feasible to deliver it to specific tissues via a viral vector, the studys senior author, Gene Yeo, said in a press release. He is aprofessor of cellular and molecular medicine at UCSD School of Medicine.

The study, Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9, published in the journal Cell, described how the team rebuilt the Cas9 system to find and chop up disease-causing RNA molecules.

In gene editing, the CRISPRCas 9 system uses an RNA probe that matches a specific stretch of DNA. Once bound to the right gene, the Cas9 enzyme cuts the DNA, which then can be inactivated or edited. The new system targets RNA, and chops it upinstead of editing it.

RNA, whichis largely composed of similar building blocks as DNA, has numerous roles in a cell. For instance, it is used to take a copy of a gene to provide instructions for the cells protein-making machinery.

At times, however, RNA molecules start accumulating what researchers call microsatellite repeat expansions. These are stretches of repeat RNA letters that disrupt the normal activity of the RNA. When found in messenger RNAs, they prevent necessary proteins from being made.

Anabnormal sequence also makes the RNA accumulate in cells, disrupting other cell operations. This can be seen in ALS that runs in families, andin diseases such as myotonic dystrophy and Huntingtons.

In ALS, such repeats are found in the C9orf72 gene, and cause about a third of familial ALS cases, or those that run in families,according to the ALS Association.

Testing the new tool in lab-grown cells derived from ALS patients with such mutations, the team showed that RCas9 could eliminate at least 95 percent of accumulated RNA, seen as dense clusters, or foci, in the cells.

They also discovered that using RCas9 freed proteins that normally bind to RNA in cells. When abnormal RNA starts accumulating in a cell, these proteins get tied up interacting with the aggregates, instead of binding to their natural targets. Researchers said that treated patient-derived cells eventually resembled healthy cells.

For the system to be useful as a human therapy, it needs to fit into a virus the most common way to deliver gene therapy. Normal Cas9 is too large to fit into thevirus typically used. The team solved the issue by removing parts of the Cas9 enzyme required for cutting DNA, making the enzyme small enough to fit.

Yet, many more questions need to be answered before the method can be tried in patients.

The main thing we dont know yet is whether or not the viral vectors that deliver RCas9 to cells would elicit an immune response, Yeo said. Before this could be tested in humans, we would need to test it in animal models, determine potential toxicities and evaluate long-term exposure.

The group has launched a company, Locana, that will work onpreclinical-trial development of the method with the aim of bringing it to patients.

We are really excited about this work because we not only defined a new potential therapeutic mechanism for CRISPR-Cas9, we demonstrated how it could be used to treat an entire class of conditions for which there are no successful treatment options, said David Nelles, PhD, one of two lead studyauthors.

There are more than 20 genetic diseases caused by microsatellite expansions in different places in the genome. Our ability to program the RCas9 system to target different repeats, combined with low risk of off-target effects, is its major strength, added Ranjan Batra, PhD, the studys other lead author.

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Studying How Genes, Environment Contribute to Juvenile Arthritis – UB School of Medicine and Biomedical Sciences News

Posted: August 14, 2017 at 11:45 am

James N. Jarvis, MD, is conducting a study of the gene-environment paradigm for juvenile idiopathic arthritis pathogenesis.

Published August 14, 2017

James N. Jarvis, MD, clinical professor of pediatrics, will use an Arthritis Foundation grant to study how genes and environment work together to influence the immune dysfunction in juvenile arthritis.

After asthma, juvenile idiopathic arthritis (JIA) is the most common chronic disease condition in children. While genetics play a small role in the disease, environmental factors are also known to be important.

The study, titled Interplay Between Genetics and Epigenetics in Polyarticular JIA, builds upon previous work by Jarvis and his fellow researchers.

The epigenome refers to the features of DNA and the proteins that DNA is wrapped around that do not control the genetic makeup of a person but do influence how cells respond to the environment, says Jarvis, principal investigator on the grant.

Specifically, the epigenome determines what genes a cell will turn on or turn off in response to environmental cues, he notes.

Like most complex traits, genetic risk for JIA is principally located within non-coding regions of the genome.

Our preliminary studies present the hope that we can finally understand the gene-environment paradigm for JIA pathogenesis, Jarvis says.

Rather than regarding JIA as an autoimmune disease, triggered by inappropriate recognition of a self protein by the adaptive immune system, Jarvis hypothesizes that JIA emerges because leukocytes suffer genetically and epigenetically mediated perturbations that blunt their capacity to regulate and coordinate transcriptions across the genome.

This loss of coordinate regulation leads to inappropriate expression of inflammatory mediators in the absence of the normal external signals typically required to initiate or sustain an inflammatory response, he says.

Our field has been dominated by a single hypothesis for JIA pathogenesis for 30 years, Jarvis notes. However, as the field of functional genomics becomes increasingly wedded to the field of therapeutics, our work carries the promise of completely new approaches to therapy based on a completely different paradigm of pathogenesis.

The researchers are recruiting 30 children with newly diagnosed polyarticular JIA for its study to survey the epigenome and CD4+ T cells in them and compare the results with findings in 30 healthy children.

We plan to build a multidimensional genomic map that surveys the functional epigenome, examines underlying genetic variation and examines the effects of genetic and epigenetic variation on gene expression, Jarvis says.

He notes the work will focus on CD4+ T cells because the researchers have already identified interesting interactions between their epigenome and transcriptome in the context of therapeutic response in JIA.

Because the epigenome is the medium through which the environment exerts its effects on cells, Jarvis believes that characterizing the epigenome in pathologically relevant cells, ascertaining where epigenetic change is linked to genetic variation and determining how genetic and epigenetic features of the genome regulate or alter transcription is the key to truly understanding this disease.

This project addresses a question that parents always ask, which I never thought wed begin to answer in my lifetime: What causes JIA? This study wont provide the whole answer, but it will go a long way toward taking us there, he says.

The project has three specific aims:

The two-year, $730,998 grant is part of the Arthritis Foundations 2016 Delivering on Discovery awards. It was one of only six projects out of 159 proposals chosen for funding. For the first time, arthritis patients helped the foundation select projects.

Including patient input as part of the selection process was a new milestone in patient engagement for the Arthritis Foundation and allowed us to select projects that hold the most promise from an arthritis patients point of view, says Guy Eakin, senior vice president, scientific strategy.

Collaborators from the Jacobs School of Medicine and Biomedical Sciences are:

Other collaborators include researchers from the Childrens Hospital of Philadelphia.

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Ten Weird Ways Scientists Are Changing the World With Gene Editing – Newsweek

Posted: August 13, 2017 at 1:45 am

CRISPR-Cas9 gene editing has captured the publics imagination. As this powerful technology becomes even more popular, it has also incited plenty of fears about what the future may bring. But a closer look at recent milestones and studies demonstrate the future of gene editing is already happening right now.

This experimental technique, known as CRISPR (pronounced crisper) for short,utilizes snippets of bacteria as a pair of molecular scissors. The technology allows scientists to selectively modify DNA segments, disable or alter genes or correct mutations in the genome of any living organism. In a controversial landmark study published earlier this month in Nature, scientists eliminated a genetic abnormality in a human embryo.

Gene editing is proving to be a nimble and versatile technology for redesigning the world. This area of research is certain to change nearly every field of biological sciencesincluding agriculture, medicine and zoologyand touch every aspect of our lives. Here are 10 ways scientists have already used CRISPR gene editing to do what once seemed impossible.

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Here’s how CRISPR-Cas9 works. REUTERS

A potential cure for diabetes. Scientists created genetically modified skin grafts to protect lab mice from diabetes. The experiment could help researchers identify a suitable substitute for insulin.

Eliminated disease from mosquitoes. In one experiment, researchers bred mosquitos that are resistant to the parasite that causes malaria.

Created a new type of seafood. The U.S. Food and Drug Administration approved a genetically modified salmon, known as AquaAdvantage salmon. Gene editing gave the fish the taste and texture of Chinook salmon and the efficient, rapid growth of ocean pout. Canadians are already eating them.

Super-strength animals. By deleting the relevant gene, scientists in China bred goats with more muscle (for meat) and hair (for wool).

Several changes to pigs. Scientists edited out all traces of porcine endogenous retrovirus (PERV), which brings us one step closer to creating a sustainable organ supply for transplant patients. Another new strain of pigs, known as Enviropigs produces manure that is low in phosphorus.Scientists can also now cut off the gene responsible for growth hormone in the animals, which could make micropigs the next pet.

Treated cancer. CRISPR has been used in a number pilot studies in China to treat aggressive cancers. In astudyof head and neck cancer, scientists tweaked genetic mutations in a patients blood, andthen injected the blood back into thepatient in order to suppress tumor growth.

Bolster the wine supply. At Rutgers University, researchers developed a way to cultivate grapes that can resist a type of mildew that can spoil the crop.

Make antisocial ants. Ants rely on their keen sense of smell to communicate. When scientists edited out the gene responsible for their sense of smell, the bugs behaviors changed. The researchers found their antennae and brain circuits didnt fully develop. Productivity in the colony, such as food foraging, also went down dramatically because the bugs were unable to work together effectively.

Eliminate cattle dehorning. The practice of removing the horns from cattle is especially painful to the animal. It’salso costly and time-consuming for farmers. Some scientists used CRISPR to breed cows that dont have horns.

Disable HIV. Though not yet studied in humans, scientists used gene editing to excise the HIV virus from the genomes of mice.

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How Gene Editing And Pig Organs Can End The Human Transplant Shortage – Newsweek

Posted: at 1:44 am

Each year, some 30,000 patients undergo transplant surgery to receive an organ from a donor. Transplant medicine saves lives, but not enough people are willing to donate. Patients cant rely on the generosity of fellow humans to replace a heart, kidney or lungs. According to the United Network for Organ Sharing (UNOS), one patient is added to the U.S. transplant waiting list every 10 minutes, and 20 people on the national list die each day.

For decades, scientists have been hoping to address the organ shortage in more innovative ways, namely by tweaking the innards of other mammals to make them compatible with humans. Successfulanimal-to-human transplants (also known as xenotransplantation) would create a sustainable organ supply.

Pigs are the strongest contender for xenotransplantation because they have organs similar in size and physiological function to those found in humans. But pig organs on their own arent suitable for transplant. Human immune systems would most definitely reject pig organs. But an even greater challenge is the risk of animal viruses infectinghumans. Pigs carry active porcine endogenous retrovirus, and it remains unclear whether these viruses could becommunicable or fatal in humans. PERV infection would be dangerous becausetransplant recipients are routinely put on immunosuppressant drugs that make it difficult to fight off any bacteria or viruses.

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If animal-to-human transplants can be achieved successfully, it would create a sustainable organ supply. Thanks to gene editing, this may be possible in the future. REUTERS

Nowa team of researchers affiliated with Harvard Medical School appear to have solved one of these problems. Not only have these scientistsmade a controversial possibilityanimal organs in humansmore likely, but theyve done so using a controversial technology: CRISPR-Cas9 gene editing.

Through gene editing, the team eliminated all traces of the PERV virus from the cell line and conducted in vitro fertilization. There are 25 strains of PERV, which is the only known active retrovirus found in pigs. In the study, published Thursday in the journal Science, biologist Luhan Yang and her team implanted the PERV-free embryos into surrogates. The fetuses did not become reinfected with the virus, and the newbornpiglets are the first animals born without PERV. Yangwho founded eGenesis a few years ago to harness advances in CRISPR-Cas9 for the worldwide organ shortagewill now monitor the animals for any long-term effects.

Im a strong believer that science can help us improve health care if we look holistically for a solution, says Yang, lead author on the paper and chief science officer of eGenesis, the biotechnology company funding advancements in the research. Because there are millions of patients who suffer from end-stage organ failure, their life could potentially be saved, or largely improved, by this potential organ resource.

CRISPR-Cas9, or CRISPR (pronounced crisper) for short, is an experimental biomedical technique. The technology utilizes snippets of certain bacteria that allow for selective modifications of DNA segments, such as changingthe misspellings of a gene that contributeto mutations. Since CRISPR was identified several years ago, scientists have been using it in the laboratory to alter the genetic codes of living organisms. The new technologyis already leading advances once considered the stuff of science fiction. In astudy published last week in Nature,scientistseliminated a genetic abnormality in a human embryo.

Yang has been determined tousegene editing to solve the organ shortage problemfor several years. In 2013, sheand her team published the first paper showing CRISPR could be used to accurately and effectively alter the immune system. In 2015, she eradicated 62 copies of the PERV virus from a pig cancer cell line, which she says is a world record for researchers using CRISPR. The next step, she says, is to tweak the porcine genome further to prove the organs can be compatible with the human immune system.

Resurrecting aScientific Vision

For decades, xenotransplantation research seemed impossibly dangerous and financially risky both for small biomedical companies and large pharmaceutical companies. In the early 2000s, Novartis stopped funding xenotransplantation research. The U.S. Food Administration, fearing a public health disaster, began placing regulations on research facilities, whichmade studies even more challenging. The projects were costly andtoo complicated, and animal rights activists frequentlytargeted the scientists. But CRISPR is reviving the area of research once again, says Yang.

Transgenic PERV-free pigs could provide a source for solid organs as well as islet cells, which are tiny cells scattered throughout the pancreas that secrete insulin. Some successful pilot studies looked at porcine islet cell transfusions as a potential treatment for diabetes.

Dixon Kaufman, president-elect of the American Society of Transplant Surgeons and a transplant surgeon at the University of Wisconsin School of Medicine and Public Health, says its only a matter of timeprobably a few yearsbefore xenotransplant studies are open to patients. I think it is a realistic, almost palpable opportunity, he says. Anything that will improve safety, such as deleting this risk of the PERV infection, makes this more viable.

Kaufman thinks kidneys and pancreases will be the first solid animal organs transplanted into humans. Because these are non-vital organs, failure wouldnt necessarily lead to death. Patients who need a kidney could still receive dialysis, and those who need a pancreas could still access insulin.

These advances are a boon for transplant surgeons like Kaufman, who regularly have to tell patientsthey will probably die before a donated organ becomes available. He doesnt think a pig organ would be a hard sell to most of these patients, who are otherwise facing certain death.

The field is inherently sort of risky to begin with, and I think a lot of patients have already processed that, he says. I tell patientsin the grand designwe were not meant to swap body parts between ourselves.

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Researchers use CRISPR to manipulate social behavior in ants – Phys.Org

Posted: August 11, 2017 at 5:45 pm

August 10, 2017 This photograph shows Ooceraea biroi workers tagged with color dots for individual behavioral tracking. Credit: Daniel Kronauer The Rockefeller University

The gene-editing technology called CRISPR has revolutionized the way that the function of genes is studied. So far, CRISPR has been widely used to precisely modify single-celled organisms and, more importantly, specific types of cells within more complex organisms. Now, two independent teams of investigators are reporting that CRISPR has been used to manipulate ant eggsleading to germline changes that occur in every cell of the adult animals throughout the entire ant colony. The papers appear August 10 in Cell.

“These studies are proof of principle that you can do genetics in ants,” says Daniel Kronauer, an assistant professor at The Rockefeller University and senior author of one of the studies. “If you’re interested in studying social behaviors and their genetic basis, ants are a good system. Now, we can knock out any gene that we think will influence social behavior and see its effects.”

Because they live in colonies that function like superorganisms, ants are also a valuable model for studying complex biological systems. But ant colonies have been difficult to grow and study in the lab because of the complexity of their life cycles.

The teams found a way to work around that, using two different species of ants. The Rockefeller team employed a species called clonal raider ants (Ooceraea biroi), which lacks queens in their colonies. Instead, single unfertilized eggs develop as clones, creating large numbers of ants that are genetically identical through parthogenesis. “This means that by using CRISPR to modify single eggs, we can quickly grow up colonies containing the gene mutation we want to study,” Kronauer says.

The other team, a collaboration between researchers at New York University and the NYU School of Medicine, Arizona State University, the Perelman School of Medicine at the University of Pennsylvania, and Vanderbilt University. , used Indian jumping ants (Harpegnathos saltator). “We chose this species because they have a peculiar feature that makes it easy to transform workers into queens,” says Claude Desplan, a Silver Professor at NYU and one of the senior authors of the second study. If the queen dies, the young worker ants will begin dueling for dominance. Eventually, one of them becomes a “pseudoqueen”also called a gamergateand is allowed to lay eggs.

“In the lab, we can inject any worker embryo to change its genetic makeup,” Desplan says. “We then convert the worker to a pseudoqueen, which can lay eggs, propagate the new genes, and spawn a new colony.”

Desplan, co-senior author Danny Reinberg, a Howard Hughes Medical Institute investigator at NYU Langone, and Shelley Berger, the Daniel S. Och University Professor in the departments of Cell and Developmental Biology and Biology at Penn, began studying these ants several years ago as a way to learn about epigenetics, which refers to changes in gene expression rather than changes in the genetic code itself. “The queens and the worker ants are genetically identical, essentially twin sisters, but they develop very differently,” Desplan says. “That makes them a good system for studying epigenetic control of development.”

The gene that both research teams knocked out with CRISPR is called orco (odorant receptor coreceptor). Ants have 350 genes for odorant receptors, a prohibitively large number to manage individually. But due to the unique biology of how the receptors worka great stroke of luck, in this casethe investigators were able to block the function of all 350 with a single knockout. “Every one of these receptors needs to team up with the Orco coreceptor in order to be effective,” says Waring Trible, a student in Kronauer’s lab and the first author of the Rockefeller study.Once the gene was knocked out, the ants were effectively blind to the pheromone signals they normally use to communicate. Without those chemical cues, they become asocial, wandering out of the nest and failing to hunt for food.

More surprisingly, knocking out orco also affected the brain anatomy in the adult animals of both species. In the same way that humans have specialized processing centers in the brain for things like language and facial recognition, ants have centers that are responsible for perceiving and processing olfactory cues that are expanded compared to other insects. But in these ants, the substructures of these sensory centers, called the antennal lobe glomeruli, were largely missing.

“There are many things we still don’t know about why this is the case,” Kronauer says. “We don’t know if the neurons die back in the adults because they’re not being used, or if they never develop in the first place. This is something we need to follow up on. And eventually, we’d like to learn to what extent the phenomenon in ants is similar to what’s going on in mammals, where brain development does depend to a large extent on sensory input.”

“Better understanding, biochemically speaking, how behavior is shaped could reveal insights into disorders in which changes in social communication are a hallmark, such as schizophrenia or depression,” Berger says.

In a third related study from the University of Pennsylvania, researchers led by Roberto Bonasio altered ant behavior usingthe brain chemical corazonin. When corazonin is injected into ants transitioning to become a pseudo-queen, it suppresses expression of thebrain protein vitellogenin. This change stimulated worker-like hunting behaviors, while inhibiting pseudo-queen behaviors, such as dueling and egg deposition.

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Further, when the team analyzed proteins the ant brain makes during the transition to becoming a pseudo-queen, they found that corazonin is similar to a reproductive hormone in vertebrates. More importantly, they also discovered that release of corazonin gets turned off as workers became pseudo-queens. Corazonin is also preferentially expressed in workers and foragers from other social insect species. In addition to corazonin, several other genes were expressed in a worker-specific or queen-specific way.

“Social insects such as ants are outstanding models to study how gene regulation affects behavior,” says Bonasia, an assistant professor of Cell and Developmental Biology. “This is because they live in colonies comprised of individuals with the same genomes but vastly different sets of behaviors.”

Explore further: ‘Princess pheromone’ tells ants which larvae are destined to be queens

More information: 1. Cell, Trible et al: “orco mutagenesis causes loss of antennal lobe glomeruli and impaired social behavior in ants.” http://www.cell.com/cell/fulltext/S0092-8674(17)30772-9 , DOI: 10.1016/j.cell.2017.07.001

2. Cell, Yan et al: “An engineered orco mutation produces aberrant social behavior and defective neural development in ants” http://www.cell.com/cell/fulltext/S0092-8674(17)30770-5 , DOI: 10.1016/j.cell.2017.06.051

3. Cell, Gospocic et al.: “The neuropeptide corazonin controls social behavior and caste identity in ants” http://www.cell.com/cell/fulltext/S0092-8674(17)30821-8 , DOI: 10.1016/j.cell.2017.07.014

Journal reference: Cell

Provided by: Cell Press

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NYU School of Medicine researcher Dr. Danny Reinberg was awarded a Howard Hughes Institute of Medicine Collaborative Innovation Award for new research on ant epigenetics- helping to unravel the impact lifestyle and environment …

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Scientists at the Universities of Oslo and Liverpool have uncovered the secret behind a goldfish’s remarkable ability to produce alcohol as a way of surviving harsh winters beneath frozen lakes.

The gene-editing technology called CRISPR has revolutionized the way that the function of genes is studied. So far, CRISPR has been widely used to precisely modify single-celled organisms and, more importantly, specific types …

While hundreds of circular RNAs (circRNAs) are abundant in mammalian brains, one big question has remained unanswered: What are they actually good for? In the current issue of Science, Nikolaus Rajewsky and his team at the …

In the cells of palm trees, humans, and some single-celled microorganisms, DNA gets bent the same way. Now, by studying the 3-D structure of proteins bound to DNA in microbes called Archaea, University of Colorado Boulder …

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Does this mean that we are only a few years away from being able to cure homosexuality?

Do you want mutant ants? Because that’s how you get mutant ants!

Why NOT Remove Species Barriers Between Dogs (we have hundreds of Dog Breeds) and Foxes/Wolves at Gamete Level? Why NOT ‘Mate’m’ also at Gamete Level AND Bring out A HYBRID ? (Canid hybrids are the result of interbreeding between different species of the canine (dog) family (genus Canis)Also, fox terrier, Norwegian lundehund, and Spitz blood were combined to create the Sulimov dog.)

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MoU signed to commercialise gene therapy in India – Odisha Sun Times

Posted: at 5:45 pm

New Delhi: In a bid to advance research and commercialise regenerative medicine and gene therapy in India, the Association of Biotechnology Led Enterprise (ABLE) a consortium of biotechnology companies on Friday signed a Memorandum of Understanding (MoU) with a Japan-based trade association.

The collaboration between ABLE and Forum for Innovative Regenerative Medicine (FIRM) will focus on advancing the individual and common missions by sharing information including technology, policy and laws partnerships and opportunities such as co-sponsoring meetings and other cooperation based on common concern.

It will also help advance and promote commercialisation of life saving products in regenerative medicine within India, Japan and other countries.

We are proud to be a partner in this revolutionary research and industry collaborations. The partnership is a step forward to enhance the learning and training on cell and gene treatment leading to enhancement of the cell and gene therapies which help to address major unmet medical needs in India, P. Manohar, Head (Committee for regenerative medicine group) at ABLE, said in a statement on Friday.

Our association with ABLE is an opportunity to work towards the advancement of the field (of regenerative medicine and cell and gene therapies) and tap on the potential to transform human healthcare. Through the partnership, we can share learnings and insights to contribute towards curing major human illness, added Yuzo Toda, Chairman at FIRM. (IANS)

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Human Germline Genome Editing Genetics bodies weigh in on debate with position paper – JD Supra (press release)

Posted: at 5:45 pm

In an article published in American Journal of Human Genetics on 3 August 2017, an international group of 11 organisations with genetics expertise has issued a joint position statement, setting out 3 key positions on the question of human germline genome editing:

(1)At this time, given the nature and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy.

(2)Currently, 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 of gene editing. There should be no prohibition on making public funds available to support this research.

(3)Future clinical application of human germline genome editing should not proceed unless, at a minimum, there is (a) a compelling medical rationale, (b) an evidence base that supports its clinical use, (c) an ethical justification, and (d) a transparent public process to solicit and incorporate stakeholder input.

This serendipitously timed statement comes on the heels of Shoukhrat Mitalipov and colleagues at Oregon Health and Science Universitys publication of an article in Nature reporting the successful use of CRISPR/Cas9 in human embryos to correct a mutation in a gene called MYBPC3 that causes a potentially fatal heart condition known as hypertrophic cardiomyopathy. The publication of this article has drawn the attention of the wider mainstream media and reignited the public debate as to the desirability, feasibility and ethics of editing the human genome in an inheritable way.

Gene editing – putting the paper in context

Whilst debates about the ethics of gene editing (both somatic and germline) go back decades, human germline genome editing has never before been realistically possible from a technical standpoint. That has changed with the advent of the CRISPR/Cas9 system, whose efficiency and ease of use has not only opened up the field of gene editing to a far larger number of companies and laboratories than previously, but has brought the editing of specific genes in a human embryo out of the realms of fantasy into reality. The potential for such technology to improve quality of life and prevent suffering caused by debilitating genetically inherited diseases has captured the imagination of many, particularly people living with currently intractable genetic conditions, their friends and family. However, the power of the technology has also conjured up the familiar spectres of playing God, the uncertainty of long term effects on individuals (and what it means to be human itself), marginalisation of the disabled or genetically inferior and the potential for inequality to manifest itself genetically as well as socioeconomically.

Germline cell editing poses significantly more concerning ethical and regulatory issues than somatic cell editing. The latter will only result in uninheritable changes to the genome of a population of cells in the particular individual treated, whilst the former involves genetic changes that will be passed down, for better or worse, to the individuals offspring.

In early 2015, the first study demonstrating that CRISPR/Cas9 could be used to modify genes in early-stage human embryos was published. Although the embryos employed for those experiments were not capable of developing to term, the work clearly demonstrated that genome editing with CRISPR/Cas9 in human embryos can readily be performed. That report stimulated many scientists and organisations to clarify their stance on the use of genome-editing methods. The United Kingdom and Sweden have both approved experiments for editing DNA of a human embryo but not those that involve implanting embryos. In the UK, Human Fertilisation and Embryology Authority (HFEA) has approved an application by developmental biologist Kathy Niakan, at the Francis Crick Institute in London, to use CRISPR/Cas9 in healthy human embryos. Currently, such experiments cannot be done with federal funding in the United States given a congressional prohibition on using taxpayer funds for research that destroys human embryos. Congress has also banned the U.S. Food and Drug Administration from considering a clinical trial of embryo editing. As would be expected, the safety requirements for any human clinical genome-editing application are extremely stringent.

However, earlier this year, US-based National Academy of Sciences (NAS) and the National Academy of Medicine (NAM), published a report that concluded using genome-editing technology, such as CRISPR/Cas9, to make alterations to the germline would be acceptable if the intention was to treat or prevent serious genetic disease or disorders, and the procedure was proven to be safe ( significant and, to an extent, subjective hurdles to be overcome).

The ASHG position paper

The position paper was the product of a working group established by the American Society of Human Genetics (ASHG), including representatives from the UK Association of Genetic Nurses and Counsellors, Canadian Association of Genetic Counsellors, International Genetic Epidemiology Society, and US National Society of Genetic Counsellors. These groups, as well as the American Society for Reproductive Medicine, Asia Pacific Society of Human Genetics, British Society for Genetic Medicine, Human Genetics Society of Australasia, Professional Society of Genetic Counsellors in Asia, and Southern African Society for Human Genetics, endorsed the final statement. The group, composed of a combination of research and clinical scientists, bioethicists, health services researchers, lawyers and genetic counsellors, worked together to integrate the scientific status of and socio-ethical views towards human germline genome editing.

As part of this process, the working group reviewed and summarised nine existing policy statements on gene editing and embryo research and interventions from national and international bodies, including The International Society for Stem Cell Research (2015) Statement on Human Germline Genome Modification, The Hinxton Group (2015) Statement on Genome Editing Technologies and the statement released following the International Summit on Human Gene Editing (2015) co-hosted by the National Academy of Sciences, National Academy of Medicine, Chinese Academy of Sciences and The Royal Society (UK). It was observed that differences in these policies include the very definition of what constitutes a human embryo or a reproductive cell, the nature of the policy tool adopted to promote the positions outlined, and the oversight/enforcement mechanisms for the policy. However, by and large, the majority of available statements and recommendations restrict applications from attempting to initiate a pregnancy with an embryo or reproductive cell whose germline has been altered. At the same time, many advocate for the continuation of basic research (and even preclinical research in some cases) in the area. One notable exception is the US National Institutes of Health, which refuses to fund the use of any gene-editing technologies in human embryos. Accordingly, due to the lack of public funding in the US, work such as that done by Mitalipovs group must be privately funded.

The working group considered that ethical issues around germline genome editing largely fall into two broad categories those arising from its potential failure and those arising from its success. Failure exposes individuals to a variety of health consequences, both known and unknown, while success could lead to societal concerns about eugenics, social justice and equal access to medical technologies.

The 11 organisations acknowledged numerous ethical issues arising from human germline genome editing, including:

exposing individuals to potentially harmful health consequences, since the magnitude of the potential risks of off-target or unintended consequences are yet to be determined;

the risk that if highly restrictive policies are placed on the conduct and public funding of basic research in the field, this could push research out of the public eye and public interest, underground to private funders or overseas, to organisations and territories where it would be subject to less regulation, transparency and oversight. This could result in research not being subject to ethical and peer oversight, such as ethics board approval, data sharing, peer review and dissemination of research resources;1

the de facto inability of future individuals who are the result of genetic editing, to consent to that editing;

concerns around the boundaries of eugenic use of gene editing technology, which the groups acknowledged could be used to reinforce prejudice and narrow definitions of normalcy in our societies; and

ensuring the gene editing technologies do not worsen existing or create new inequalities within and between societies, noting: Unequal access and cultural differences affecting uptake could create large differences in the relative incidence of a given condition by region, ethnic group, or socioeconomic status. Genetic disease, once a universal common denominator, could instead become an artifact of class, geographic location, and culture. A dangerous consequence of such inequality could be that reduced incidence and reduced sense of shared risk could affect the resources available to individuals and families dealing with genetic conditions.

Having touched on each of these issues, the group then outlined its consensus positions:

1. At this time, given the nature and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy.

It was noted that there is not yet a high quality evidence base to support the use of germline genome editing, with unknown risk of health consequences and ethical issues still to be explored and resolved by society.

The group observed that two major categories of safety concerns are (i) the effect of unwanted or off-target mutations, and (ii) the potential unintended effects of the desired on-target base changes (edits) being made. It noted that it is reasonable to presume that any human genome-editing therapeutic application will require stringent monitoring of off-target mutation rates, but there remains no consensus on which methods would be optimal for this, or what a desirable maximum off-target mutation rate would be when these techniques are translated clinically. The working-group thus outlined its views on the minimum necessary developments that would be required (at least from a safety perspective) before germline genome editing could be used clinically:

definitions of broadly acceptable methodologies and minimum standards for measuring off-target mutagenesis;

consensus regarding the likely impact of, and maximum acceptable thresholds for, off-target mutations; and

consensus regarding the types of acceptable genome edits with regard to their potential for unintended consequences.

2. Currently, 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 of gene editing. There should be no prohibition on making public funds available to support this research.

The group agreed that conducting basic scientific [techniques?] involving editing of human embryos and gametes can be performed ethically via compliance with applicable laws and policies, and that any study involving in vitro genome editing on human embryos and gametes should be conducted under rigorous and independent governance mechanisms, including approval by ethics review boards and meeting any other policy or regulatory requirements. Public funding for such research was seen as important in ensuring that such research is not driven overseas or underground, where it would be subject to less regulation, oversight and transparency.

3. Future clinical application of human germline genome editing should not proceed unless, at a minimum, there is (a) a compelling medical rationale, (b) an evidence base that supports its clinical use, (c) an ethical justification, and (d) a transparent public process to solicit and incorporate stakeholder input.

Even if the technical data from preclinical research reaches a stage where it supports clinical translation of human germline genome editing, the working group stresses that many more things need to happen before translational research in human germline genome editing is considered. The criteria identified by the group in this position cut across medical, ethical, economic and public participation issues and represent the setting of an appropriately high and comprehensive standard to be met before human germline genome editing may be applied clinically. The group acknowledges the challenges of public engagement with such technical subject matter but encourages new approaches to public engagement and engagement of broader stakeholder groups in the public discussion.

The ethical implications of altering the human germline has been the subject of intense discussion in recent years, with calls for such work to be put on hold until the process of genome editing is better understood. ASHG supports somatic genome editing and preclinical (in vitro human and animal) germline genome research but feels strongly that it is premature to consider human germline genome editing in any translational manner at this time.

The working group concludes that Many scientific, medical, and ethical questions remain around the potential for human germline genome editing. ASHG supports somatic genome editing and preclinical (in vitro human and animal) germline genome research but feels strongly that it is premature to consider human germline genome editing in any translational manner at this time. We encourage ethical and social consideration in tandem with basic science research in the upcoming years.

This appears a reasonable position largely in line with the recommendations from the major national and international groups surveyed by the working group. It balances the need to encourage further basic research and validation with strong awareness of the ethical and societal implications of human germline genome editing, setting a high bar before such technology should be translated to the clinic. No doubt, however, the debate will continue, particularly in respect of public funding for such work. Whether the US will maintain their stance against public funding, in the face of international competition, and potential loss of talent and investment, remains to be seen.

Footnotes

1. In this connection it should be noted that China is a good example of a jurisdiction where there is very strong government investment in biotech, including CRISPR, and less regulatory standards than in the West. This combination of factors seems to be fuelling the pace of research there (many CRISPR firsts have come in China e.g. first CRISPR clinical trial in humans; first CRISPR editing of human embryos), but potentially at the risk of less rigorous, well controlled science being conducted (e.g. the recent retraction of the NgAgo paper).

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Human Germline Genome Editing Genetics bodies weigh in on debate with position paper – JD Supra (press release)

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Precision Medicine Market will grow at significant CAGR of 10.5% by 2023 – Markets Insider

Posted: August 10, 2017 at 5:45 am

OCEAN VIEW, Del., Aug. 9, 2017 /PRNewswire-iReach/ — The Industry Growth report on “Precision Medicine Market” by Global Market Insights, Inc. says Precision Medicine Market was USD 39 billion in 2015, and is anticipated to cross USD $87.7 billion by 2023, propelled by Increasing demand for personalized medicine specifically in cancer treatments and advancements in new healthcare technologies.

It is innovative procedure for treating and preventing chronic ailments depending upon changes in individual genes and other lifestyle features. Innovative approach helps doctors properly assess ailment risk and predict optimal treatment. Growing occurrence of cancer and increase in cancer prone geriatric population across the globe is predicted to boost industry expansion.

Threats related with sharing of patient’s genetic information can hinder industry growth. Insurance firms can use patient data and raise their premium for people who are at a risk of acquiring inherited diseases. Further, decline in rate of FDA (U.S. Food and Drug Administration) drug approval has minimized the rate of production of new medicines despite heavy investments. This aspect can hinder global precision medicine market expansion.

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The industry is segmented into different technologies like gene sequencing, companion diagnostics, big data analytics, bioinformatics and drug discovery.

Gene sequencing segment size was more than $8.1 billion for 2015. Current FDA guidelines on next -generation sequencing dependent tests takes into consideration individual differences in genes of various persons, environments and life patterns while creating new type of healthcare.

Companion diagnostics segment has acquired importance owing to rising concerns about rates of drug failures. Further, the segment is expanding at rapid pace owing to rise in financial support and approvals by government.

Heavy throughput omics techniques applied in biological and basic research are predicted to propel bioinformatics segment growth. Out of all omics techniques next-generation technique is predicted to create key impact on the segment growth.

Drug discovery technique contributed more than $9 billion for 2015 and is predicted to register CAGR of 8.31% during forecast timeframe. Further, biomarker directed treatments with medicine targeting epidermal growth factor receptor (EGFR),c-ros oncogene 1 receptor tyrosine kinase (ROS1) and anaplastic lymphoma kinase (ALK) have speeded up the production of new medicines.

Browse key industry insights spread across 94 pages with 85 market data tables & 62 figures & charts from the report, “Precision Medicine Market” in detail along with the table of contents:

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Application Trends

Global industry is segmented into various applications like respiratory application, oncology application, Immunology application and central nervous system (CNS) application.

Oncology application contributed more than 30.1% of precision medicine market share for 2015 and is predicted to record CAGR of 10.91% during forecast timeframe.

CNS application contributed more than $9.1 billion for 2015. Neuroscience therapeutics has been utilizing the approach for long duration.

Regional Trends

Global industry was segmented into key geographical regions like North America, MEA, Europe, APAC and Latin America.

U.S. precision medicine market share was about 65.1% of revenue of North America. Factors like large allocation of budget by U.S. president to agencies like FDA(U.S. food and drug administration) , NIH (National Institute of Health) and NCI (National Cancer Institute) along with favorable government rules have contributed to the regional industry growth.

Germany precision medicine market share was more than $2.5 billion for 2015 and is predicted to contribute significantly to the growth of European industry. Reason for industry growth in the region can be credited to the fact that many institutions have acquired biomarker analysis certification required for colorectal cancer detection tests.

Further, medicine producing and diagnostic firms are making tremendous efforts for enhancing industry growth in Europe. Favorable compensation policies are predicted to promote industry growth in France.

China contributed more than 25.1% to APAC precision medicine market share for 2015 and is predicted to remain key region in future. Favorable government initiatives and high contributions from academic labs has assisted in the regional industry growth.

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Key industry players profiled in the report include Roche Holdings AG, Qiagen, Pfizer, Medtronic, Source Precision Medicine Incorporation, Silicon Biosystems, Tepnel Pharma Services, Covance, Biocrates Life Sciences AG, Novartis, Nanostring Technologies, Laboratory Corporation of America Holdings, Quest Diagnostics, Teva Pharmaceuticals, Intomics, Ferrer InCode, Eagle Genomics Limited and Quest Diagnostics.

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Pressure Relief Devices Market size is projected to experience significant growth from 2017 to 2024. Pressure relief devices market size should witness significant growth due to rising incidences of pressure ulcers across the globe.

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Pancreatic Cancer Treatment Market Size 2017-2024

Pancreatic Cancer Treatment Market size is projected to experience significant growth from 2017 to 2024. Pancreatic cancer treatment market size should witness significant growth due to increasing healthcare expenditure, technological advancements and drug innovations coupled with vast number of unmet needs associated with the disease.

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Precision Medicine Market will grow at significant CAGR of 10.5% by 2023 – Markets Insider

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Gene Editing Might Mean My Brother Would’ve Never Existed – TIME

Posted: at 5:45 am

CRISPR-CAS9 gene editing complex from Streptococcus pyogenes.Molekuul/Science Photo Library/Getty Images

Reynolds, Ph.D., is Rice Family Postdoctoral Fellow in Bioethics and the Humanities at The Hastings Center.

On August 2nd, scientists achieved a milestone on the path to human genetic engineering. For the first time in the United States, scientists successfully edited the genes of a human embryo . A transpacific team of researchers used CRISPR-Cas9 to correct a mutation that leads to an often devastating heart condition. Responses to this feat followed well-trodden trails. Hype over designer babies. Hope over new tools to cure and curb disease. Some spin, some substance and a good dose of science-speak. But for me, this breakthrough is not just about science or medicine or the future of humankind. Its about faith and family, love and loss. Most of all, its about the life and memory of my brother.

Jason was born with muscle-eye-brain disease. In his case, this included muscular dystrophy, cerebral palsy , severe nearsightedness, hydrocephalus and intellectual disability. He lived past his first year thanks to marvels of modern medicine. A shunt surgery to drain excess cerebrospinal fluid building up around his brain took six attempts, but the seventh succeeded. Aside from those surgeries complications and intermittent illnesses due to a less-than-robust immune system, Jason was healthy. Healthy and happy very happy. His smile could light up a room. Yet, that didnt stop people from thinking that his disability made him worse off. My family and those in our religious community prayed for Jason. Strangers regularly came up to test their fervor. Prayer circles frequently had his name on their lists. We wanted him to be healed. But I now wonder: What, precisely, were we praying for?

Jasons disabilities fundamentally shaped his experience of the world. If praying for his healing meant praying for him to be normal, we were praying for Jason to become someone else entirely. We were praying for a paradox. If I could travel back in time, Id walk up to young, devout Joel and ask: How will Jason still be Jason if God flips a switch and makes him walk and talk and think like you? The answer to that question is hard. Yes, some just prayed for his seizures to stop. Some for his continued well-being. But is that true of most? Is that what I was praying for?

The ableist conflation of disability with disease and suffering is age-old. Just peruse the history of medicine. Decades of eugenic practices. Sanctioned torture of people with intellectual disability. The mutilation of otherwise healthy bodies in the name of functional or aesthetic normality. These stories demonstrate over and over again how easily biomedical research and practice can mask atrocity with benevolence and injustice with progress. Which leads me to ask: What, precisely, are we editing for?

Although muscle-eye-brain disease does not result from a single genetic variant, researchers agree that a single gene, named POMGNT1, plays a large role. Perhaps scientists will soon find a way to correct mutations in that and related genes. Perhaps people will no longer be born with it. But that means there would never be someone like Jason. Those prayers I mentioned above? Science will have retroactively answered them. That thought brings me to tears.

I wish we could cure cancer , relieve undue pain and heal each break and bruise. But I also wish for a world with Jason and people like him in it. I want a world accessible and habitable for people full stop not just the people we design. I worry that in our haste to make people healthy, we are in fact making people we want. We, who say we pray for healing, but in fact pray for others to be like us. We, who say were for reducing disease and promoting health, but support policies and practices aimed instead at being normal. We, who are often still unable to distinguish between positive, world-creating forms of disability and negative, world-destroying forms between Deafness , short stature or certain types of neurodiversity and chronic pain, Tay-Sachs or Alzheimers . It is with great responsibility that we as a society balance along the tightrope of biomedical progress. I long for us to find that balance. Ive certainly not found it for myself. Lest I forget how often weve lost it and how easy it is to fall, I hold dearly onto the living memory of Jason. I no longer pray for paradoxes, but for parity for the promise of a world engineered not for normality, but equality.

But that world will never come if we edit it away.

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Gene Editing Might Mean My Brother Would’ve Never Existed – TIME

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Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American

Posted: August 9, 2017 at 4:45 am

By Ben Hirschler

LONDON (Reuters) – The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe.

Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States.

Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases.

After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years.

In the past five years, European regulators have approved two gene therapies – the first of their kind in the world, outside China – but only three patients have so far been treated commercially.

UniQure’s Glybera, for a very rare blood disorder, is now being taken off the market given lack of demand.

The future of GlaxoSmithKline’s Strimvelis for ADA-SCID – or “bubble boy” disease, where sufferers are highly vulnerable to infections – is uncertain after the company decided to review and possibly sell its rare diseases unit.

Glybera, costing around $1 million per patient, has been used just once since approval in 2012. Strimvelis, at about $700,000, has seen two sales since its approval in May 2016, with two more patients due to be treated later this year.

“It’s disappointing that so few patients have received gene therapy in Europe,” said KPMG chief medical adviser Hilary Thomas. “It shows the business challenges and the problems faced by publicly-funded healthcare systems in dealing with a very expensive one-off treatment.”

These first two therapies are for exceptionally rare conditions – GSK estimates there are only 15 new cases of ADA-SCID in Europe each year – but both drugs are expected to pave the way for bigger products.

The idea of using engineered viruses to deliver healthy genes has fuelled experiments since the 1990s. Progress was derailed by a patient death and cancer cases, but now scientists have learnt how to make viral delivery safer and more efficient.

Spark Therapeutics hopes to win U.S. approval in January 2018 for a gene therapy to cure a rare inherited form of blindness, while Novartis could get a U.S. go-ahead as early as next month for its gene-modified cell therapy against leukaemia – a variation on standard gene therapy.

At the same time, academic research is advancing by leaps and bounds, with last week’s successful use of CRISPR-Cas9 gene editing to correct a defect in a human embryo pointing to more innovative therapies down the line.

Spark Chief Executive Jeffrey Marrazzo thinks there are specific reasons why Europe’s first gene therapies have sold poorly, reflecting complex reimbursement systems, Glybera’s patchy clinical trials record and the fact Strimvelis is given at only one clinic in Italy.

He expects Spark will do better. It plans to have treatment centers in each country to address a type of blindness affecting about 6,000 people around the world.

Marrazzo admits, however, there are many questions about how his firm should be rewarded for the $400 million it has spent developing the drug, given that healthcare systems are geared to paying for drugs monthly rather than facing a huge upfront bill.

A one-time cure, even at $1 million, could still save money over the long term by reducing the need for expensive care, in much the same way that a kidney transplant can save hundreds of thousands of dollars in dialysis costs.

But gene therapy companies – which also include Bluebird Bio, BioMarin, Sangamo and GenSight – may need new business models.

One option would be a pay-for-performance system, where governments or insurers would make payments to companies that could be halted if the drug stopped working.

“In an area like haemophilia I think that approach is going to make a ton of sense, since the budget impact there starts to get more significant,” Marrazzo said.

Haemophilia, a hereditary condition affecting more than 100,000 people in markets where specialty drugmakers typically operate, promises to be the first really big commercial opportunity. It offers to free patients from regular infusions of blood-clotting factors that can cost up to $400,000 a year.

Significantly, despite its move away from ultra-rare diseases, GSK is still looking to use its gene therapy platform to develop treatments for more common diseases, including cancer and beta-thalassaemia, another inherited blood disorder.

Rivals such as Pfizer and Sanofi are also investing, and overall financing for gene and gene-modified cell therapies reached $1 billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.

Shire CEO Flemming Ornskov – who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder – sees both the opportunities and the difficulties of gene therapy.

“Is it something that I think will take market share mid- to long-term if the data continues to be encouraging? Yes. But I think everybody will have to figure out a business model.”

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Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American

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