AAVLife Gets $12M to Bring Gene Therapy to Rare Ataxia

4/15/14Follow @benthefidler

Bluebird Bio is a symbol, in a way, of the renaissance of gene therapy. The Cambridge, MA-based company raised $116 million in an IPO last year, and a number of startups pursuing gene therapy have cropped up since. So perhaps its no surprise that the field’s new entrant, AAVLife, comes from some of the same people that indirectly helped Bluebird get off the ground a few years ago.

Today, AAVLife is emerging from stealth with the help of a $12 million Series A round led by San Francisco-based Versant Ventures and the Inserm Transfert Initiative, a venture arm of the French Institute of Health and Medical Research. The funds will help Paris and New York-based AAVLife build on an animal study just published in Nature in which researchers used gene therapy to curb the cardiological dysfunction associated with Friedreich’s Ataxia, a rare, often fatal, genetic neuromuscular disorder that causes a progressive loss of motor function, among other things. AAVLife has been built to advance that work into clinical trials and hopes to get its first human study up and running in early- to mid-2015, according to CEO Amber Salzman.

To be clear, at this point, AAVLife isn’t trying to cure Friedreich’s outright—just the part of it that affects the heart. But Salzman says the biggest reason people with Friedreich’s end up dying young is heart failure from the damage that accompanies the neurological symptoms. So on one hand, reversing these effects would still leave Friedreich’s sufferers dealing with the neurological symptoms of their disease, which get progressively worse and can often leave patients in a wheelchair. On the other, these patients would theoretically get many more years, “possibly decades” of life, says Versant venture partner Thomas Woiwode.

“The thought from the patient groups and everyone involved has been, if we can make sure that [these patients] live and they don’t die of heart failure in their 20s, that’s a huge benefit. We didn’t want to slow down anything just because we couldn’t cure everything,” Salzman says. “But we are very rapidly looking at what’s the best way to approach getting to the other cells as well.”

AAVLife’s roots can be traced back to Bluebird (NASDAQ: BLUE). About 12 years ago, Salzman’s nephew and later her one-year-old son were diagnosed with x-linked adrenoleukodystrophy, a crippling genetic brain disorder. Salzman, then an R&D executive at GlaxoSmithKline, took action: she dove into research, helped form the Stop ALD Foundation, and began collaborating with pediatric neurology specialist Patrick Aubourg. The two began thinking about a gene therapy approach to treat the disorder, which is triggered by a specific genetic mutation.

AAVLife CEO Amber Salzman

AAVLife CEO Amber Salzman

Through her foundation, Salzman helped coordinate the backing to advance the concept, ultimately leading to a successful gene therapy procedure that Aubourg helped perform on two boys with ALD. The results of that study were published in Science, and Third Rock Ventures and Genzyme took that work forward in the form of Bluebird, now a publicly-traded company with a roughly $500 million market cap.

“When it comes to rare diseases, when you get a passionate and effective foundation behind it, it’s amazing what they can pull off,” she says.

Salzman and Aubourg kept working together over the next few years, even as Salzman took over a startup called Cardiokine and eventually sold it to Cornerstone Therapeutics in 2011. Aubourg was looking into treatments for other rare, neurodegenerative diseases—among them Friedreich’s. The disease is caused by a mutation to the FXN gene, which leads to low production of frataxin, a protein that helps mitochondria—the cell’s power plants—function normally.

Aubourg convinced Salzman to take a project on and form a company with him once she sold Cardiokine. With some pressing from the Friedreich’s Ataxia Research Alliance, Salzman decided to center the project around a therapy for Friedreich’s. She then helped bring together an international team of researchers and gene therapy experts to work on the project—among them Ronald Crystal of New York’s Weill Cornell Medical College and Helene Puccio of the University of Strasbourg in France. With the group in place, AAVLife officially formed in January.

Indeed, while AAVLife is a small startup at this point, it’s also an international effort. The company’s offices are in Paris, where it can tap into Inserm for R&D help, but it’s also leaning on Crystal in New York to … Next Page »

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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  • Melissa Gordon

    Thank you for never giving up on our kids. I can’t begin to tell you how happy this makes FA families. We are giving our kids their future back.

    Melissa Gordon