I am afraid, seriously, that the field is moving too fast. 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 … That period was a blur of isolating, characterizing, and testing new AAVs, Vandenberghe recalls. 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. Many felt that gene therapy just wasn’t ready for prime time. James M. Wilson, researcher in gene therapy at the University of Pennsylvania. 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.”. Xconomy National — [ Updated, 1/31/18, see below] Gene therapy pioneer James Wilson and University of Pennsylvania colleagues sounded an alarm Tuesday morning about the use of gene … Jim’s laboratory has made seminal contributions to the technology of gene transfer and has paved the way for translation of these technologies into the clinic. “The point is to make us walk in the room and remember,” Kakkis says. 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. That therapy was approved for sale this May. He was stripped of his titles, his gene therapy center was disbanded, and he was barred from doing any more clinical trials until 2010. Jesse was the 18th patient in the study, and the last. It was particularly frustrating for a small group of researchers who had been championing the use of a different kind of gene-delivery vehicle: adeno-associated viruses—the AAVs. 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. The credit belongs to the man who is actually in the arena, whose face is marred by dust and sweat and blood.”. 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. 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. Gene therapy pioneer James Wilson is applying his knowledge and experience in the field to a new frontier: the Covid-19 pandemic. Again, GSK wouldn’t budge. C. Dr. Wilson is a professor at the University of Pennsylvania and was Director of the Institute for Human Gene Therapy at the University of Pennsylvania. “And I am willing to pay for it.”. “Should we morph to support the industry as it tries to get back on its feet?” Wilson recalls asking his group. 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. 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. Department: Medicine. At the restaurant, his father was explaining the stunning results of a clinical trial underway at UCL that used AAV8. https://cen.acs.org/.../Gene-therapy-pioneer-James-Wilson/97/web/2019/02 That data woke up investors to gene therapy. In July 2000, the GSK cash arrived, allowing Wilson’s lab to begin developing a new generation of safer gene therapies in full force. 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. 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 “I have nightmares all the time,” says Gao, who is now director of a gene therapy center at the University of Massachusetts. Furthermore, it was almost preposterous that no one had discovered these allegedly new viruses before. The university later paid a half-million-dollar fine, and a private settlement to the patient’s family. One little slip and everybody jumps on them.”. 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. Before he knew it, 5:00 p.m. had rolled around. Discount will be applied automatically at checkout. 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. 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. Related: FDA approves second gene therapy, Zolgensma, to treat spinal muscular atrophy in infants. Jesse was suffering from yet another of his medical episodes. He says it has about 80 open positions, including veterinarians, IND application writers, and even an intellectual property scientist. ), according to an SEC filing. “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. Twenty years ago, a young man named Jesse Gelsinger was injected with a large dose of gene-shuttling viruses designed in Wilson’s lab. 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. He laid out a plan to convert what remained of his lab into a discovery shop, but he’d need money. Wilson asked another scientist to repeat the experiment. “When you are a scientist working here, you feel like nothing is impossible,” she says. And almost everyone Squinto met was a staff scientist. Dr. Jim Wilson has been pioneering gene therapy research for decades, setting the stage for medicine’s next revolution. “We basically had to become a hybrid of industry and the academy,” Wilson says. His liver produced low levels of the enzyme. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan over 30 years ago. The largest center of its kind, it was housed at Penn and led by Wilson. It wasn’t his first hospitalization, but this time was especially scary. Although that lawsuit was resolved out of court in just 6 weeks, investigations by Penn, the FDA, and the Department of Justice lingered for years. 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. “He’s an ambitious guy,” Gelsinger says. They’d begin by further developing gene therapies for five rare neurological diseases. After Jesse regained consciousness in January, he developed a new appreciation for the gravity of his condition. 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. Later that afternoon, Wilson walked into Gao’s office and drilled him on his methods and interpretations. After years of worry and doubt, gene therapy -- in effect, using genes as medicine -- is exploding across the white-hot biotech scene. “He runs his gene therapy center like you’d run a very efficient company,” he thought. 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. 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. The road to hopeful start-ups such as Passage Bio has included blind alleys and reversals. Dr. James Wilson, the director of the gene therapy program at the University of Pennsylvania’s medical school. Business news and analysis sent straight to your inbox every Tuesday morning. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Suddenly, all the attention was on Wilson and his lab. “It was a major redo, and we were hesitant.” It would mean expanding the staff to include toxicologists, study directors, alliance managers, and other well-paid professionals required to turn scientific ideas into experimental therapies ready to test in humans. Chemical & Engineering News will not share your email address with any other person or company. Password and Confirm password must match. 2020 Jul;31(13-14):695-696. doi: 10.1089/hum.2020.182. To send an e-mail to multiple recipients, separate e-mail addresses with a comma, semicolon, or both. 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. 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. Copyright © 2020 American Chemical Society. “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. ), according to an SEC … After raising additional cash from other investors, Passage Bio officially launched in February with $115.5 million. Wilson stopped going to conferences—he was no longer invited to speak—and for years he avoided the press. California residents do not sell my data request. 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. 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. People had lost faith not only in Wilson’s lab but also in the field as a whole. When Gao first presented the data at a lab meeting in mid-December, Wilson was skeptical. 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. “It is so amazing how his memory has held on and is so alive still,” Gelsinger says. Videos of the event showed Wilson clapping alongside Mills—the CEO of Regenxbio—who rang the opening bell amid falling confetti. But a few weeks later, the child’s liver enzyme levels skyrocketed. Choose the membership that is right for you. Now that his long ban from running clinical trials had ended, he saw an opportunity to evolve again. “We expect to establish our own manufacturing facility for long-term commercial market supply,” the company added in its SEC filing. Wilson has been thinking about his legacy and what’s kept him committed to gene therapy all these years. “Jim has been very worried about these high doses,” Mendell says. It was the first public tragedy of a highly hyped field. Over the next few years, Penn’s viral vector center became the Amazon of AAV. “Gene therapy was still under the shadow of Jesse Gelsinger,” Gao says. A plaque displaying his name and picture hang by the door. “I basically bullied my way through,” he says. By continuing to use this site you are agreeing to our COOKIE POLICY. Money, hard to raise not long ago, is flooding into academic labs and companies developing gene therapies. The excitement expressed about academic-industry partnerships that night is a dramatic shift from the mood at the turn of the century. “Every other week there was another shoe to drop,” Wilson recalls. Immediately after the injection, the infant seemed to be doing fine. This site uses cookies to enhance your user experience. “We are not going to be a passive participant in the process of translating the potential of our science into goods, products, services,” says John Swartley, director of the Penn Center for Innovation. They made it to the movie, Resident Evil, in the nick of time. Jesse’s story is the subject of textbook chapters on bioethics and a case study in law journals. 125 S. 31st Street, Suite 1200 TRL Philadelphia, PA 19104-3403. Related: Astellas to acquire gene therapy firm Audentes for $3 billion. About. 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