Xconomy New York — A potential gene therapy for Parkinson’s disease has just gotten a big boost thanks to one of the larger Series A investment rounds in years for a New York biotech. The funding, for Prevail Therapeutics, provides another jolt for the city’s life science startup scene. The startup, seeded in New York last year by OrbiMed Advisors and the Silverstein Foundation—a nonprofit organization formed by OrbiMed partner Jonathan Silverstein—has closed a $75 million Series A round. The cash will help speed an experimental therapy called PR001 towards clinical testing. The treatment is a gene therapy meant to treat patients with a rare, aggressive, form of Parkinson’s that’s associated with a mutation in the gene GBA1. About 10 percent of Parkinson’s patients have this form, according to the Michael J. Fox Foundation. Prevail is run by Columbia University neurology professor and Parkinson’s researcher Asa Abeliovich (pictured), whose work has been focused on the genetics of the debilitating disease. He wouldn’t say when he expects PR001 to begin human trials. “We’re looking forward to treating patients with unmet medical needs as soon as possible,” he says. PR001 would introduce a working copy of the gene in patients in hopes of altering the progression of the disease. Prevail aims to develop other gene therapies for subsets of patients with other genetic forms of neurodegenerative diseases as well, though Abeliovich won’t specify which ones yet. The startup has a deal in place with RegenxBio (NASDAQ: RGNX) to access a type of adeno-associated virus, a tool commonly used to deliver genes to target tissues in the body. Prevail isn’t the only company going after Parkinson’s patients with a GBA1 mutation; Sanofi and Allergan have programs in development as well. And Voyager Therapeutics (NASDAQ: VYGR) is also advancing a gene therapy for Parkinson’s, though that treatment is meant to help patients respond to their medications again after they wane, not stop or slow the disease’s march. The news, meanwhile, is the latest in a recent string of funding announcements for New York biotech, indicating that momentum has picked up again after a relatively quiet 2017. In February, Roche bought Flatiron Health, a New York life sciences/tech hybrid company that amasses data on cancer patients, for $1.9 billion. Since then, two other New York-born startups, Kallyope and Quentis Therapeutics, each raised large funding rounds: $66 million and $48 million, respectively. Now Prevail, based in the Alexandria Center for Life Science in Manhattan, has topped Kallyope and Quentis with its $75 million. OrbiMed, Pontifax Fund, RA Capital Management, EcoR1 Capital, Omega Funds, BVF Partners, Boxer Capital, Adage Capital Management, and Alexandria Venture Investments provided Prevail’s latest financing. Prevail was the first industry investment for the Silverstein Foundation, which OrbiMed partner Silverstein started after being diagnosed with an aggressive form of Parkinson’s. Here’s more on the startup, gene therapies for Parkinson’s disease, and some of the other recent news surrounding New York biotechs.
Biotech start-up Prevail Therapeutics has raised $75m to develop a gene therapy for Parkinson's disease. The New York-based company will use the money to accelerate clinical testing of an experimental treatment called PR001, which has been developed to treat a rare form of the illness.