Swiss majors Roche and Novartis back gene therapy upstart

The cash boost will go toward early work on a group of rare and inherited metabolic diseases, including Wilson disease.

Newly born European biotech Vivet Therapeutics has started life with €37.5 million ($41 million) and an executive team made up of Novartis, Sanofi and Gensight veterans, as the company looks to get to work on its gene therapies for a host of rare diseases.

Novartis Venture Fund and Columbus Venture Partners led the round, with Roche Venture Fund, HealthCap, Kurma Partners and Ysios Capital also taking part.

The cash boost will go toward early work on a group of rare and inherited metabolic diseases, including Wilson disease, progressive familial intrahepatic cholestasis types 2 and 3, and citrullinemia type I.

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Vivet's lead gene therapy program, VTX801, is first targeting Wilson disease, a rare genetic disorder caused by a defective gene in liver cells encoding the ATP7B protein, which 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 biotech’s therapy uses a modified AAV vector to move a truncated functional version of the ATP7B gene into the liver cells carrying the defective gene, which should treat the underlying cause of the disease.

This is designed to restore copper metabolism, reduce liver damage and improve liver function, reversing the problems inherent in Wilson’s. Human trials for the med in this setting are slated to start next year.

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 Massachusetts Eye and Ear Infirmary in Boston.

Vivet Therapeutics was born last year in Paris, with a wholly owned subsidiary in Spain, and founded by Jens Kurth (formerly of Anokion and Novartis) as well as Jean Philippe Combal (who has served stints at Gensight Biologics and Sanofi).

Combal, its co-founder and CEO, said: “Vivet is delighted to have attracted such a substantial investment from these high-profile life sciences investors. This fundraising reflects our shared excitement about the potential of our lead candidate VTX801 and our technology for generating further novel gene therapies targeting rare inherited metabolic diseases. Early results from preclinical studies with VTX801 are very promising, and we are now well funded to advance this candidate into the clinic, while developing our portfolio and technologies.”

Florent Gros, MD at Novartis Venture Fund, said of its investment: “We have searched extensively for next generation AAV technologies and clinical applications. We are very excited by Vivet Therapeutics’ clinical and commercial prospects; the company has outstanding management, assets and capabilities.”

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