Startup AAVLife sets sights on ataxia with $12M and a novel gene therapy

Parisian biotech AAVLife has come out of stealth mode with a Versant Ventures-led $12 million A round, looking to advance a gene-deleting therapy that promises to treat the rare and deadly Friedreich's ataxia.

The disease, which gradually robs sufferers of motor function, is a result of a mutation in the frataxin gene that leads to progressive cardiac degradation and, often, death. Targeting only Friedreich's ataxia's effects on cardiac function, AAVLife's solution involves replacing the malfunctioning frataxin gene with a healthy one, delivered using a harmless adeno-associated virus (AAV), which explains the biotech's name.

The method, based on work from the French National Institute of Health and Medical Research (Inserm), has already demonstrated promise in a mouse study published in Nature Medicine, and AAVLife said it's in the midst of further preclinical work with plans to test its therapy on humans next year.

To get there, the biotech has recruited a cast of experts in gene therapy and AAV delivery, including CEO Amber Salzman, a former GlaxoSmithKline ($GSK) R&D exec who played a major role in bringing about the first gene-therapy trial for the rare adrenoleukodystrophy, the top target of IPO success story bluebird bio ($BLUE). Combined with the $12 million round, led by Versant and joined by the Inserm Transfert Initiative, AAVLife now has the cash and personnel it needs to potentially make a difference for the roughly 20,000 Friedreich's ataxia sufferers in the U.S. and Europe, Salzman said.

"Friedreich's ataxia may be a rare disorder, but it is an all too common feature of everyday life for patients and their families," she said in a statement. "We are moving with appropriate care but also urgency."

Meanwhile, maturing science and willing investors have made it a good time to be a gene therapy startup. In February, Third Rock Ventures staked $45 million on Cambridge, MA's Voyager Therapeutics with a plan to use AAV-delivered genes to treat central nervous system diseases, and the Wellcome Trust put up $20 million in January to support NightstaRx and its gene therapies for degenerative eye conditions.

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