Harnessing the power of gene therapy to target rare genetic disorders
CEO: Jean Philippe Combal
Clinical focus: Rare diseases, including Wilson disease, progressive familial intrahepatic cholestasis types 2 and 3, and citrullinemia type 1.
The scoop: This European biotech believes it can use the latent and largely untapped potential of gene therapy as a curative approach for Wilson disease, as it also looks across the Atlantic for its future.
What makes Vivet Fierce: Getting off a €37.5 million ($41 million) series A raise last year was no mean feat for a European biotech, given that the biopharma industry across the continent typically fails to enjoy the same rich funding streams those in the U.S. do.
The VC arms of Roche and Novartis were early backers for a team of execs from Novartis itself, as well as Sanofi and Gensight. This includes CEO Jean Philippe Combal, Pharm.D., Ph.D., who worked a stint at Fovea (which in 2009 was bought by Sanofi) and in 2014 moved over to GenSight to work on its gene replacement therapy for Leber’s hereditary optic neuropathy, a rare mitochondrial disease that leads to irreversible and often brutal sight loss in teens and young adults.
He’s still in the gene therapy game, but now at upstart Vivet, and says he much prefers the working environment of a biotech, rather than his time at a Big Pharma, where “you are a team of 100 in a global business of hundreds of thousands, and it’s easy to get lost.”
His new biotech’s leading med, VTX801, is slated to enter the clinic next year and will initially target Wilson disease, a rare disorder that can lead to organ damage, neurologic symptoms and potentially death. Around 10,000 patients in the U.S. and 15,000 patients in the EU are estimated to have the disease.
The therapy uses a modified AAV vector to move a truncated functional version of the ATP7B gene into liver cells carrying the defective gene, which should treat the underlying cause of the disease.
Vivet’s pipeline comes out of tech developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada, a not-for-profit foundation at the Centro de Investigación Medica Aplicada; the University of Navarra based in Pamplona, Spain; and the Massachusetts Eye and Ear Infirmary in Boston.
Combal says what he wants to do is analogous to current gene therapy work in hemophilia, which aims to decrease or eradicate the need for treatment, and eventually produce a curative therapy: “The aim in Wilson disease is that we are anticipating for the gene therapy that we will achieve a full restoration of the copper metabolism, reduce liver damage and improve liver function, and therefore reversing the problems inherent in Wilson’s.”
While there have been disappointments and setbacks in the gene therapy field—uniQure pulled Glybera in Europe because of low demand, and GSK is evaluating options for its gene therapy business Strimvelis—the fact that these companies had cared at all about rare disease and gene therapy is only a plus for the industry.
“Strimvelis of course was ex vivo and you can’t export that to patients, so they had to go on site, which made treatment difficult." Only one patient was believed to have been treated in the year since its EU approval.
“But for me," Combal says, "GSK doing this at all [developing Strimvelis] was a very good sign; they were willing to show that … they were able to innovate in ultraorphan disease and put money into R&D for this area. I’m sure they will continue to be innovative with Strimvelis and its future, whether that’s through a new company or not, but I don’t think they will drop off significantly in researching in this area.”
We spoke on the day Gilead bought CAR-T biotech Kite Pharma for around $12 billion, and for Combal, that was significant for him and the industry: “That’s a big deal; $12 billion is a lot, I mean it's peanuts for Gilead, but a lot in terms of paying out for a new form of technology that a few years ago didn’t seem that it could garner such attention.”
Vivet hopes it can be on the same track, and is targeting trials and future funding rounds in the U.S., not just Europe, in the coming years.
Investors: Novartis Venture Fund and Columbus Venture Partners, Roche Venture Fund, HealthCap, Kurma Partners and Ysios Capital.