Up until some months ago, Jean-Philippe Combal had been the COO at Paris-based GenSight Biologics, focused on a closely-watched late-stage study of an experimental gene therapy for Leber’s Hereditary Optic Neuropathy (or LHON), a rare mitochondrial disease that destroys vision. But in a quick leap, he’s now jumped ship, leaving CEO Bernard Gilly’s crew and launching a new wave therapy startup of his own that has attracted about $40 million in startup cash.
Vivet Therapeutics is not a competing venture. Combal is jumping into liver and metabolic diseases, building a platform tech to go after a new set of rare diseases. First up for a clinical program is Wilson Disease, an orphan disease triggered by an errant gene that scrambles the body’s ability to regulate levels of copper in the liver.
Working with Jens Kurth, ex-Anokion and Novartis along with Gloria Gonzalez Aseguinolaza from CIMA at the University of Navarra, they have a lead gene therapy dubbed VTX801 ready to go into the clinic next year. The money should get them through first steps in early-stage R&D work with enough cash to pay for a couple of more INDs as they start pipeline construction.
Novartis Venture Fund and Columbus Venture Partners led the round, with a syndicate that includes Roche Venture Fund, HealthCap, Kurma Partners and Ysios Capital.
The nascent gene therapy field has learned a lot from the first two gene therapies to arrive in Europe. Glybera from uniQure is now being yanked after one use, and Strimvelis from GSK is finding considerable resistance from payers as well.
But Combal is looking on the bright side, gaining lessons from the real world experience of the pioneers to see how to build a gene therapy company that can thrive, avoiding the pitfalls that awaited the leaders.
“I think the top companies today (which includes US leader Spark Therapeutics) offer a good example of what is possible and not possible,” Combal, also a Fovea and Sanofi veteran, tells me. To be a success in this field, companies are going to need strong data from at least two years of experience in trial patients.
“We need to get strong outcomes,” he adds, in order to get an approval with the kind of demonstration of efficacy that can win over payers and agencies. “Strimvelis was very helpful, showing that big companies can go into gene therapy.” And he believes the hemophilia studies underway now can deliver the kind of strong data needed to start building markets.
Combal is happy to let the pioneers break these paths. He plans to be close behind, learning at every step and incorporating those lessons into the business plan.
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 24,000+ biopharma pros who read Endpoints News by email every day.Free Subscription