Am 14. The experimental treatment was created to treat a rare metabolic liver disease. That the conversation had veered to viruses was typical: Matt grew up in a house saturated with science. Sign up for C&EN's must-read weekly newsletter, Squinto was astounded. And Zolgensma is just the beginning. Wilson was furious. “He runs his gene therapy center like you’d run a very efficient company,” he thought. “And you know what happens to high-profile golden boys. That puts Passage Bio in the market with Roche’s Spark Therapeutics and other Philadelphia firms scouting the city and its suburbs for clean-room manufacturing sites or contractors. “At some point we were concerned that even though we generated it, we weren’t going to make the most interesting observations on our own technology.” They were right. Surgeons were already transplanting whole livers into kids with OTC deficiency to restore that missing enzyme. “But Jim’s pretty tough.”, Yamada, meanwhile, had recently left academia to become head of R&D at GlaxoSmithKline. He remained Wilson’s only graduate student for several years. And after years of operating in relative isolation, Wilson started hearing from drug companies. The significance of my contribution explains why I'm listed as a co-inventor in all the patents related to that work even though I never finished my PhD and why I personally analyzed and deposited all of the initial sequences into Genbank. Furthermore, it was almost preposterous that no one had discovered these allegedly new viruses before. Sign in with a different account. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan over 30 years ago. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Again, GSK wouldn’t budge. Behandlung von Erbkrankheiten Therapie von SCID. “We lost a generation of patients that we could have helped.”, Some still worry that history could repeat itself. Passage Bio is a genetic medicines company based in Philadelphia, PA that is developing a deep pipeline of adeno-associated virus (AAV) delivered therapeutics for the treatment of rare monogenic central … When Gao first presented the data at a lab meeting in mid-December, Wilson was skeptical. Dr. James Wilson, director of the gene therapy program at the University of Pennsylvania, is taking a new role at a Maryland biotech company he helped launch. Jim Wilson was only endangering his career, not his life. A handful of academics with an entrepreneurial bent have turned their biomedical labs into start-up incubators, fueled by troops of precocious graduate students and postdocs. Wilson graduated from Albion College (B.A., Chemistry) and the University of Michigan (MD, PhD). Despite that—or perhaps because of it—Wilson’s downsized and outcast lab kept plugging away with a newfound focus on finding safer viruses. It didn’t take long for Wilson’s lab to make progress. At that time, the first experimental inklings of gene therapy suggested it was possible, and scientists were racing to turn the idea into a reality. The caption on the photo of the Regenxbio IPO was updated on Sept. 13, 2019, because it incorrectly stated the date of the IPO as Sept. 15, 2015. Yamada thought Wilson was gene therapy’s best hope, and he was already drafting a plan to fund Wilson’s lab. To top it all off, Amicus also agreed to pay Wilson’s lab $10 million a year for 5 years to expand his work to improve gene therapies and develop the next generation of AAVs. “Is it fair for us to avoid developing a potential curative therapy for OTC deficiency because of the past?” The answer, he says, is no. Dr. Wilson was a Co-Investigator on the OTC NIH … 2020 Jul;31(13-14):695-696. doi: 10.1089/hum.2020.182. Gene therapy pioneer James Wilson’s revelation of two new types of toxicity in animals receiving high systemic doses of adeno-associated viral vectors is unlikely to herald a major disruption of the AAV space, though it remains to be seen whether they influenced his decision to resign as SAB chair of Solid Biosciences Inc. Others worried about the perception of GSK working with a scientist disbarredby the FDA. “I sometimes called it the gulag of the Wilson lab,” he jokes. That changed soon after their hike. “I basically bullied my way through,” he says. Related: FDA approves second gene therapy, Zolgensma, to treat spinal muscular atrophy in infants. “People were in full-on retreat from having anything to do with the field.”. He runs his gene therapy center like you'd run a very efficient company. The firm, which employs 20, is developing treatments for rare single-gene disorders of the central nervous system, including Krabbe disease, GM1 gangliosidosis, and frontotemporal dementia. The first was the decision to permit James Wilson to conduct gene therapy experiments at Penn while maintaining a majority interest in Genovo, a biotech company that stood to make millions if the … The FDA can barely keep up with the growing pipeline, and it anticipates receiving more than 200 applications from groups that want to test new cell and gene therapies next year. Description of Research Dr. Wilson is an innovator and pioneer in the field of gene therapy. James M. Wilson, MD, PhD, director of the Gene Therapy Program at the University of Pennsylvania, and a co-founder of Passage Bio. Depending on whom you ask, Jesse’s death either catalyzed a bold rethinking of gene therapy or simply slowed down the inevitable march of progress that would have occurred with or without Wilson. BREAKING: Everything we know about the COVID-19 coronavirus. The company’s “scientific founders” include doctors Stephen Squinto and Tadataka Yamada, along with Wilson; staff has worked at companies including Biogen, GlaxoSmithKline, Merck & Co., and other cell and gene therapy pioneers. Penn scientists churned out thousands of AAV preps a year that they used internally and distributed to others. Mauricio, thank you for that note! The center fulfilled an ambition that started when Wilson was an MD-PhD student at the University of Michigan Medical School in the early 1980s and witnessed the devastating consequences of rare genetic diseases firsthand. If you have an ACS member number, please enter it here so we can link this account to your membership. 125 S. 31st Street, Suite 1200 TRL Philadelphia, PA 19104-3403. At the restaurant, his father was explaining the stunning results of a clinical trial underway at UCL that used AAV8. These viruses were supposed to be safer, though; they were AAV9. “The Wilson lab and gene therapy are almost unprecedented examples of how that potential can translate into a lot of activity.”. “What has changed significantly is the value that academic institutions place in partnering with industry,” he adds. And at the center of it all is none other than James Wilson. Symptoms began appearing within five days of administering the gene therapy. At the helm of that heartbreaking clinical trial in 1999 was James Wilson, MD, PhD, director of the gene therapy program at the University of Pennsylvania and scientific founder of REGENXBIO, one of several companies developing adeno-associated virus (AAV) viral vectors for gene therapies – a very different virus from the one that killed Jesse. Two years ago, when Paul Gelsinger’s daughter was taking a graduate genetics course at the University of Arizona, her professor used Jesse’s story as an example of science gone awry and a lack of informed consent. In late November, Wilson flew to Arizona to meet Paul Gelsinger. Jesse was in and out of the hospital over the holidays, seizing, vomiting, and not getting any better. is the latest in a two-year wave of dozens of cell and gene therapy start-ups to go public before they have products on the market. Search PubMed for articles. Now that his long ban from running clinical trials had ended, he saw an opportunity to evolve again. I have nightmares all the time. “I have nightmares all the time,” says Gao, who is now director of a gene therapy center at the University of Massachusetts. California residents do not sell my data request. Investor money dried up, and start-ups shuttered. To my knowledge, that was the first time in history that novel viruses had been discovered and more importantly, isolated using the PCR technique, for which we subsequently got the patent. His lab’s many biotech partners include Biogen, Johnson & Johnson, Moderna, and Precision BioSciences, with programs running the gamut from common conditions like Alzheimer’s to rare diseases, including OTC deficiency. In July 2000, the GSK cash arrived, allowing Wilson’s lab to begin developing a new generation of safer gene therapies in full force. Gene therapy pioneer James Wilson resigned from the scientific advisory board at Solid Biosciences LLC (Cambridge, Mass. Gene Therapy Pioneer James Wilson leading studies of adeno-associated viral vectors to potentially prevent COVID-19. Appreciate a generally well written account of the history of this chapter in the development of Gene therapy. University of Pennsylvania scientist and gene therapy pioneer James Wilson has developed a new tactic designed to make these treatments safer for the nervous system. Penn is proud of its prowess in translating academic work into medicines, and it’s not bashful about the financial benefits, either. Investors can’t seem to make enough bets on potential gene therapy profits. “He’s an ambitious guy,” Gelsinger says. James M. Wilson has led an effort to develop the field of gene therapy. Wilson spoke about the medical breakthroughs he expects to … Gene therapy researcher James Wilson, of the University of Pennsylvania in Philadelphia, directed a clinical trial involving gene therapy that led to the death of 18-year-old Jesse Gelsinger in 1999. Stephen Squinto vividly remembers his first visit to James Wilson’s lab—if it can even be called that. Eventually, in March 2002, the Penn researchers convinced Thomas Shenk, a respected virologist at Princeton University, to review the paper for the Proceedings of the National Academy of Sciences of the United States of America, where it was published online that August. “Every other week there was another shoe to drop,” Wilson recalls. The university later paid a half-million-dollar fine, and a private settlement to the patient’s family. Epub 2020 Jul 1. Those concerns have guided the strategy of Wilson’s new company, Passage Bio, which is focused on rare genetic neurological conditions. “I never felt that I would walk away from science,” Wilson says. Last year, when he learned that Ultragenyx Pharmaceutical was running a new clinical trial for OTC deficiency, he emailed the firm, hoping it was being more careful than Wilson was 2 decades earlier. Related: Crowds swarm gene-therapy conference as excitement hits an all-time high. By the fall of 1999, the OTC deficiency study was one of seven underway at the Institute for Human Gene Therapy in Philadelphia. The challenge is actually getting the therapeutic genes into the body. “I think a lot of us sit back at meetings thinking about where the field would be if we hadn’t gone through that episode,” UNC’s Samulski says. Dr. Jim Wilson has been pioneering gene therapy research for decades, setting the stage for medicine’s next revolution. Gene therapy hype is now back in full swing, and the rekindled excitement is dredging up old concerns. “I think lesser personalities would have been destroyed,” says Nelson Wivel, a deputy director in Wilson’s lab at that time. The goal was to deliver a working copy of the teenager’s broken gene, but the viruses threw his immune system into overdrive. His work is being applied to genetic treatments by a start-up company, Passage Bio, which has applied for an initial public stock offering (IPO). Although each experimental gene therapy must be tested anew, it should be relatively straightforward to crank out several more programs that use the same AAV, each just packed with a different gene. “All that was going through my mind was Jesse Gelsinger all over again and the end of AAV,” Mendell recalls. Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Eleven of those firms have obtained licenses from Regenxbio, a firm that Wilson cofounded in 2009 to commercialize his technology. Email: wilsonjm@upenn.edu. A developer of gene therapies for rare monogenic CNS diseases—whose co-founders include pioneer researcher James M. Wilson, MD, PhD—has launched with $115.5 million in Series A financing. It is typical for modern biology (and perhaps in other fields) research, that bosses take all the credit and people who did the actual work at the bench left aside (how unjust!). (optional). The company was founded by industry veterans Stephen Squinto, Ph.D., and Tachi Yamada, M.D., in a partnership with gene therapy pioneer James Wilson, M.D., Ph.D, The company has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program as well as the Penn Orphan Disease Center. Another site housed the many animals—mice, monkeys, cats, and dogs—in which the experimental AAV gene therapies are tested. James M. Wilson, researcher in gene therapy at the University of Pennsylvania. The investor walked through rows and rows of lab benches—standard biology fare, to be sure, but at an immense scale. Over dinner at a Mexican restaurant in 2011, his dad, James, was talking about a hemophilia gene therapy. He was stripped of his titles, his gene therapy center was disbanded, and he was barred from doing any more clinical trials until 2010. When Wilson graduated in 1984, he was itching to get into the lab to work on the problem but was stuck completing his residency first. Title: The redemption of James Wilson, gene therapy pioneer. Key words: adeno-associated virus (AAV), gene therapy, gene transfer, gene editing, gene delivery, route of administration. Guest speakers for the event included Penn Medicine gene therapy pioneer and Passage Bio co-founder Dr. James Wilson. His work is being applied to genetic treatments by a start-up company, Passage Bio, which has applied for an … “It’s all here,” he told Wilson. That therapy was approved for sale this May. The $29.4 million that GSK provided Wilson over the next 9 years was a lifeline, but it came with strings attached: the big pharma firm owned the rights to discoveries made with its money. James Wilson, a molecular biologist at the University of Pennsylvania, and his colleagues found that an AAV9 variant (AAVhu68) used to deliver a human gene to spinal cord motor neurons in rhesus monkeys and piglets resulted in severe toxicity affecting the liver and motor neurons. Their work led to the discovery and dissemination of new AAVs, including one used in the recently approved gene therapy Zolgensma, which saves the lives of infants born with an otherwise fatal neurological disease. “Now there is an academic group trying to compete with that, and frankly, service them.” In his estimation, Wilson’s lab has largely become an “IND warehouse.”. Compared with some people with OTC deficiency, Jesse had it good. The credit belongs to the man who is actually in the arena, whose face is marred by dust and sweat and blood.”. James Wilson had redefined his career once by shifting from clinical pioneer to toolmaker. Videos of the event showed Wilson clapping alongside Mills—the CEO of Regenxbio—who rang the opening bell amid falling confetti. “And that’s what got him in trouble.”. That fact would later make them gene therapy’s greatest asset. Related: Astellas to acquire gene therapy firm Audentes for $3 billion. “The whole field was miscast as profiteers, a truly inappropriate demonizing of very important science,” Yamada says. Join us to get the news you need. Wilson trained only a handful of PhD students at a time. Passage Bio, based on gene therapy technology developed by University of Pennsylvania biotech pioneer James Wilson, says it will use proceeds from the sale to clinically test its initial therapies and bring them closer to market. Read More View All News 11/11/2020 Penn Researchers Develop Approach to Prevent Toxicity Tied to Neurological Gene Therapy. Wilson’s son Matt recalls the moment when gene therapy was on the up and up. Wilson’s son Matt recalls the moment when gene therapy … I'm no scientist, but it is my understanding that those assays are still a good indicator of immune response. It hopes to have all three in clinical testing programs over the next two years, and has “exclusive rights” to a total of 12 treatments under development at Penn, but adds that “it will be several years, if ever," before Passage Bio’s medicines hit the market. The company was founded by industry veterans Stephen Squinto, Ph.D., and Tachi Yamada, M.D., in a partnership with gene therapy pioneer James Wilson, M.D., Ph.D. “But we’re working on so many other liver metabolic diseases,” he says. James M. Wilson Gene therapy researcher James Wilson, of the University of Pennsylvania in Philadelphia, directed a clinical trial involving gene therapy that led to the death of 18 … The road to hopeful start-ups such as Passage Bio has included blind alleys and reversals. "Gene therapy pioneer James Wilson, MD, PhD, professor of Medicine and director of the Gene Therapy Program and the Orphan Disease Center at Penn’s Perelman School of Medicine, and … Today, Wilson’s gene therapy center is larger than ever. Scientific integrity, vigilance, good clinical practice, responsible oversight, and thorough pre-clinical evaluations are critical to the realization of the promise of gene therapy and the continued success of this field. Got james wilson gene therapy company in an induced coma for 2 days while waiting for his body to.... 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