A young woman had recently received the same dose. Soon after Jesse’s death, Wilson and Yamada had a long discussion about what to do next. Although clinical trials fail all the time, and in fact, other people would die during experimental gene therapy trials, Jesse’s death tapped into fears about the risks of genetic alteration. 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. Yet Jesse Gelsinger—a man he never met—is an inescapable part of Wilson’s legacy. They’d begin by further developing gene therapies for five rare neurological diseases. And after years of operating in relative isolation, Wilson started hearing from drug companies. You could lose a child.’ ” Wilson backed off. “Jim has been very worried about these high doses,” Mendell says. 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. According to his father, Jesse said the worst-case scenario was that he would die “and maybe help doctors figure out a way to save sick babies.”. Enjoy these benefits no matter which membership you pick. Now everyone is happy to work with Jim and gives him a lot of money. “The Wilson lab and gene therapy are almost unprecedented examples of how that potential can translate into a lot of activity.”. By Penn’s latest count, some 42 companies are using AAVs that fall under Wilson’s patents, covering nearly 100 drug development programs. 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. Immediately after the injection, the infant seemed to be doing fine. “Oh my God, you got to be kidding me,” Wilson recalls thinking. When the results held up, Wilson called the team together on a Saturday in spring 2002 to review the results. And Zolgensma is just the beginning. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan over 30 years ago. “It’s all here,” he told Wilson. Gene Therapy Pioneer James Wilson leading studies of adeno-associated viral vectors to potentially prevent COVID-19. Vandenberghe wondered if a virus … He was born with a rare genetic mutation that left him with low levels of an enzyme called ornithine transcarbamylase (OTC), which is needed to properly metabolize nitrogen—a crucial building block of proteins. Am 14. “The community got a black eye and the funding dried up because Wall Street couldn’t distinguish between adenovirus and adeno-associated virus,” says Richard Jude Samulski, director of the University of North Carolina’s Gene Therapy Center. James Wilson had redefined his career once by shifting from clinical pioneer to toolmaker. He runs his gene therapy center like you'd run a very efficient company. “The point is to make us walk in the room and remember,” Kakkis says. The company wanted to kick-start its own pipeline of clinical programs instead of just supplying AAVs to others. Key words: adeno-associated virus (AAV), gene therapy, gene transfer, gene editing, gene delivery, route of administration. In 2017, Squinto scoped it out on behalf of OrbiMed Advisors, a health-focused private equity firm. These viruses were supposed to be safer, though; they were AAV9. At the restaurant, his father was explaining the stunning results of a clinical trial underway at UCL that used AAV8. To date neither Dr. Wilson nor the University of Pennsylvania have accepted any responsibility for Jesse’s death. Once Penn finished testing the therapies in monkeys, the biotech firm would take the data and petition the US Food and Drug Administration for permission to begin clinical trials in humans. I can't tell you how disappointed I was to find out that nobody credited me with the discovery of all of the 100+ novel AAV vectors, which neither my supervisor, Dr. Gao, nor the head of the lab, Dr. Wilson, could have done without me because I was the only person in the institute who had the scientific expertise and educational background to undertake such an ambitious project using PCR, which was notorious for randomly mutating the DNA, and succeed. Publications. Wilson says he thought a lot about whether continuing to work on the disease would create conflicts or distractions from the goal of curing patients. When Matt was 16, he began working in his dad’s lab. “Jim was a high-profile golden boy,” says Tachi Yamada, one of his mentors at the University of Michigan. Gene Therapy Pioneer James Wilson leading studies of adeno-associated viral vectors to potentially prevent COVID-19. And his new company, Scout Bio, is developing gene therapies to treat anemia, atopic dermatitis, and chronic pain in pets. “I hate that kind of thing.”. “Now everyone is happy to work with Jim and gives him a lot of money.”. The credit belongs to the man who is actually in the arena, whose face is marred by dust and sweat and blood.”. That fact would later make them gene therapy’s greatest asset. To Yamada, the focus on Wilson’s conflict of interest because of Genovo was overblown. As more groups began using Wilson’s viruses, it grew clear that they would want commercial licenses to develop bona fide gene therapies. “I was really intent on getting these things in the hands of everyone.” He argued that academics had an obligation to share their source materials and that anyone who wanted to use the AAVs could reverse engineer them anyway. He had four kids himself. James Wilson of the University of Pennsylvania reported this week that monkeys and pigs given super-high doses of gene therapy died or suffered disturbing behavioral changes. 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. “This is really going to lead to a simplification of the drug development process,” Wilson says. In January of 2018, gene therapy pioneer James Wilson resigned from the company’s scientific advisory board, according to a company filing, over “emerging concerns about the possible … The pace is a source of both excitement and anxiety for anyone who’s weathered gene therapy’s peaks and troughs over the past 3 decades. “Every other week there was another shoe to drop,” Wilson recalls. 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 … 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. Gene therapy pioneer James Wilson resigned from the scientific advisory board at Solid Biosciences LLC (Cambridge, Mass. Wilson has expanded his armamentarium to include messenger RNA therapy, which is like a temporary version of gene therapy, as well as gene editing, which can make precise changes to genes instead of replacing them wholesale. GSK held the rights to the viruses, and the drug company wasn’t interested in sharing. Wilson and others stuffed them into hollowed-out adenoviruses—which are naturally adept at infiltrating cells to propagate their own genes. In the previous 35 years, scientists had discovered only six variants of AAVs, and although they seemed safe, none of them were particularly good at delivering genes. “It is so amazing how his memory has held on and is so alive still,” Gelsinger says. The challenge is actually getting the therapeutic genes into the body. “I never felt that I would walk away from science,” Wilson says. Others worried about the perception of GSK working with a scientist disbarredby the FDA. 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. The resurgence of gene therapy has reawakened Paul Gelsinger’s concerns about its safety too. Again, GSK wouldn’t budge. In February 2018, Wilson published a small study showing that high doses of a virus similar to AAV9 caused such severe toxicity in monkeys and piglets that the animals had to be euthanized. Wilson saw gene therapy as a less extreme option than some contemporary treatments. V. RESURGENCE. It became a sticking point in the media. “We had to pay a Wilson tax on the field.”, Wilson’s old mentor Yamada watched the fallout from the OTC deficiency trial with dismay. His liver produced low levels of the enzyme. All Rights Reserved. Wilson recently moved his discovery team to a commercial building down the street. Behandlung von Erbkrankheiten Therapie von SCID. Later that year, the Gelsinger family sued Penn—Gelsinger had come to believe Wilson’s ties to Genovo and his rush to be first in curing a genetic disease had caused the researcher to take an unnecessary risk with his son’s life. Yamada was ready to overrule everyone, if needed, but it didn’t come to that. For a brief moment, Wilson was able to get his mind off the viruses, but it didn’t last long. By the fall of 1999, the OTC deficiency study was one of seven underway at the Institute for Human Gene Therapy in Philadelphia. He told some lawyer friends, Allan Fox and Daniel Kiser, about his dilemma, and they came up to Penn in November 2007, along with their protégé, Ken Mills, to help Wilson figure out how he could get the rights to his viruses back. Wilson graduated from Albion College (B.A., Chemistry) and the University of Michigan (MD, PhD). Dr. Wilson has dedicated his career to gene therapy and much of his research has focused on the development of AAV vectors as vehicles for gene delivery. 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. “I have nightmares all the time,” says Gao, who is now director of a gene therapy center at the University of Massachusetts. I hope that the future text books on genome therapy will correct this injustice and your and others now silent names will be written in! 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. Over dinner at a Mexican restaurant in 2011, his dad, James, was talking about a hemophilia gene therapy. Wilson was furious. Their first study isolated only fragments of AAV capsid sequences—the genetic code for the outer shells of the viruses—so they still had to isolate the full sequences, turn them into usable viruses, and test them in animals. Jesse’s health deteriorated quickly. Password and Confirm password must match. In September, Jesse flew to Philadelphia to begin the trial. Guest speakers for the event included Penn Medicine gene therapy pioneer and Passage Bio co-founder Dr. James Wilson. 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. “And I am willing to pay for it.”. In June, he would turn 18, making him eligible for an experimental gene therapy trial for OTC deficiency that he’d first heard about in September 1998. 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. “Gene therapy was still under the shadow of Jesse Gelsinger,” Gao says. Over the next few months, the duo negotiated an intimate arrangement: they would form a new company, and Wilson’s lab at Penn would function as its R&D arm. Appreciate a generally well written account of the history of this chapter in the development of Gene therapy. Wilson was eager to get the new viruses into the hands of other researchers who could independently show that they were up to snuff, but he met a roadblock. Your account has been created successfully, and a confirmation email is on the way. 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. Yamada thought Wilson was gene therapy’s best hope, and he was already drafting a plan to fund Wilson’s lab. 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. 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. 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. It was actually on Sept. 17, 2015, as stated in the story. “It was not acceptable that we would limit distribution of these,” he recalls. Worse, Wilson also caught wind that his GSK funding would be winding down. Later that afternoon, Wilson walked into Gao’s office and drilled him on his methods and interpretations. Sign up for C&EN's must-read weekly newsletter, Squinto was astounded. To send an e-mail to multiple recipients, separate e-mail addresses with a comma, semicolon, or both. Both Penn and Wilson’s team were supportive, and as his operations grew, Wilson developed a mantra that he repeats to himself several times each day: “innovate, execute, and diversify.” His team started using AAV8 and AAV9 to create experimental gene therapies for a bevy of rare diseases. After Jesse’s death, the connection fostered allegations that Wilson had acted recklessly for profit. It was the first public tragedy of a highly hyped field. We use the information you provide to make your reading experience better, and we will never sell your data to third party members. Investors can’t seem to make enough bets on potential gene therapy profits. Some were even able to sit, speak, and walk. Jesse was the 18th patient in the study, and the last. Repurposing a proven gene therapy approach to treat, prevent COVID-19 In a Q&A, Penn Medicine’s James M. Wilson discusses using adeno-associated viral vectors to transport a lab-made antibody … “I was cheap labor,” he quips. Graduate Group Affiliations Cell and Molecular Biology; Contact information. Business news and analysis sent straight to your inbox every Tuesday morning. Our Benefits and Perks . That same year, a bright student named Luk Vandenberghe joined Wilson’s lab—although other scientists warned him against it. PassageBio, started in 2018, 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. “At Penn Medicine, we are the most for-profit nonprofit in the world,” Kevin Mahoney, then CEO-elect, told the room. In July 2001, a year after the GSK money first arrived, Gao began fishing for new AAVs using a technique called polymerase chain reaction to pull fragments of the viral DNA from monkey tissue. Wilson has been thinking about his legacy and what’s kept him committed to gene therapy all these years. 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. Wilson is an advisor to REGENXBIO, Dimension Therapeutics, and Solid Gene Therapy, and is a founder of, holds equity in, and receives sponsored research support from … Four days after being treated, Gelsinger died. They went out for a hike in the desert, and Gelsinger recalls that Wilson was going on about his institute, how morale was low after the tragedy, that he was losing people. Wilson admits that scientists still don’t totally understand why AAV becomes toxic at a certain dose, but it is clear that without more effective gene-delivery vessels, high doses may be the only way to correct enough cells for some conditions, particularly muscle diseases. J.M. Discount will be applied automatically at checkout. Dr. James Wilson, the director of the gene therapy program at the University of Pennsylvania’s medical school. About. Many felt that gene therapy just wasn’t ready for prime time. Great article, but for clarity regarding Wilson not anticipating an innate immune response in the OTC trial, consider this: CTL assays were a required part of the original OTC clinical trial & were not done on any of the participants. Dr. Wilson was a Co-Investigator on the OTC NIH … “When you are a scientist working here, you feel like nothing is impossible,” she 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 … (optional). Investor money dried up, and start-ups shuttered. Stephen Squinto vividly remembers his first visit to James Wilson’s lab—if it can even be called that. Regulatory authorities swept in to investigate the tragedy. I have nightmares all the time. 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). After Jesse regained consciousness in January, he developed a new appreciation for the gravity of his condition. Today, Wilson’s gene therapy center is larger than ever. Join us to get the news you need. He remained Wilson’s only graduate student for several years. Discount will be applied automatically at checkout. Those concerns have guided the strategy of Wilson’s new company, Passage Bio, which is focused on rare genetic neurological conditions. The results of the 15-patient study, published in November 2017, showed that all infants were still alive at 20 months. I was a member of that committee, which recommended minor tinkering. 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. Read More View All News 11/11/2020 Penn Researchers Develop Approach to Prevent Toxicity Tied to Neurological Gene Therapy. But even with new money from a public stock offering “we will need to raise substantial additional capital to complete the development and commercialization of our product candidates,” the company warned investors in its SEC filing. By submitting your information, you are gaining access to C&EN and subscribing to our weekly newsletter. “We basically had to become a hybrid of industry and the academy,” Wilson says. Not you? But many virologists thought studying them was a waste of time since they weren’t associated with any diseases. Compared with some people with OTC deficiency, Jesse had it good. “We lost a generation of patients that we could have helped.”, Some still worry that history could repeat itself. “People were in full-on retreat from having anything to do with the field.”. He completed residency … REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. This site uses cookies to enhance your user experience. Yet Wilson’s lab stands apart for its scale and for his heavy reliance on more experienced scientists that develop dozens of experimental gene therapies in parallel. It didn’t take long for Wilson’s lab to make progress. When Jesse’s body began breaking down within a day of the adenovirus injection, Wilson knew that T cells couldn’t be the culprit, since they take a week or more to mount their attack. “When I first came to Penn, working with a company was bad, evil,” Wilson says. One iteration of the therapy even killed monkeys at high doses, so Wilson asked one of his researchers, Guangping Gao, to develop a crippled adenovirus—one that delivered genes but didn’t trigger the immune system’s T cells to attack the liver. “These are intense efforts that normally take place at drug development companies,” explains Vandenberghe, who started his own lab at Massachusetts Eye and Ear in 2012. Her team is already working on 12 of them. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan over 30 years ago. The editorial, published in Human Gene Therapy, is co-authored by James M. Wilson, MD, Ph.D., director of the Gene Therapy Program at the University of Pennsylvania and former Editor Human Gene … 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. 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. Regenxbio CEO Ken Mills (center) and James Wilson (center left) celebrate the initial public offering of their gene therapy company Regenxbio on Sept. 17, 2015, the 16th anniversary of Jesse Gelsinger's death. 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. 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. Another site housed the many animals—mice, monkeys, cats, and dogs—in which the experimental AAV gene therapies are tested. “You need to find out what caused the problem,” Yamada advised. 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. Penn is proud of its prowess in translating academic work into medicines, and it’s not bashful about the financial benefits, either. James M. Wilson is a biomedical researcher with expertise in gene therapy. The group had already dabbled in using AAVs as alternatives to adenoviruses. In 2013, several programs from his lab—including one for Jesse Gelsinger’s disease, OTC deficiency—formed the basis of a Regenxbio spin-off called Dimension Therapeutics. Wilson remembered he had promised to take his 11-year-old son, Matt, to see a sci-fi flick—one that his mom wouldn’t take him to see—and he was running late. “I basically bullied my way through,” he says. “Things are happening so fast,” Wilson says with a touch of awe. Inside GSK, it was a hard sell. 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. That therapy was approved for sale this May. Science is not a solitary effort - gains and advances are made through the efforts of countless unnamed and unrecognized scientists at various levels on the career ladder whose dedication to their craft, profession, lab and patients needs to be highlighted as well as the individuals at the top. Wilson isn’t alone in his close ties to biotech. That period was a blur of isolating, characterizing, and testing new AAVs, Vandenberghe recalls. And while some people praise him for catalyzing the field’s resurgence, others still blame him for its near demise 20 years ago. That the conversation had veered to viruses was typical: Matt grew up in a house saturated with science. That changed soon after their hike. Suddenly, all the attention was on Wilson and his lab. “From Jim’s standpoint, it looked pretty easy,” Yamada says. 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). 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 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. Ultragenyx got the OTC deficiency program when it acquired Dimension Therapeutics in 2017, although the firm doesn’t advertise that the program’s origins lie in Wilson’s lab. More information: James M. Wilson et al, Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy, Human Gene Therapy (2020).DOI: 10.1089/hum.2020.182 A Penn surgeon walked him through the protocol, and a few days later, on Sept. 13, injected him with a large dose of gene-stuffed viruses: 600 billion per kilogram of his body weight. “Should we morph to support the industry as it tries to get back on its feet?” Wilson recalls asking his group. Mendell suspected that although some AAV9 reached the infant’s neurons, most of the viruses were getting stuck in the liver, where they were causing massive inflammation. Wilson’s son Matt recalls the moment when gene therapy … James M. Wilson has led an effort to develop the field of gene therapy. “And you know what happens to high-profile golden boys. “We expect to establish our own manufacturing facility for long-term commercial market supply,” the company added in its SEC filing. But a few weeks later, the child’s liver enzyme levels skyrocketed. “He’s an ambitious guy,” Gelsinger says. 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. But one thing is clear. Wilson directs the Gene Therapy Program and the Orphan Disease Center, which together staff an army of more than 200 people spread across multiple floors of multiple buildings at the University of Pennsylvania. Videos of the event showed Wilson clapping alongside Mills—the CEO of Regenxbio—who rang the opening bell amid falling confetti. Gene therapy pioneer James Wilson resigned from the scientific advisory board at Solid Biosciences LLC (Cambridge, Mass. Related: Astellas to acquire gene therapy firm Audentes for $3 billion. Safer viruses many felt that I would walk away from science, ” says Yamada! To fund Wilson ’ s medical school liver disease so we can link this account to your inbox every morning. 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