Sanofi embarks on an $845M gene therapy R&D odyssey with Voyager

Voyager Therapeutics CEO Steven Paul

By John Carroll and Damian Garde

Sanofi ($SNY) subsidiary Genzyme is tying up with gene therapy upstart Voyager Therapeutics, gambling $100 million upfront on the hot field and promising up to $745 million in milestones for their partnership on a full slate of development programs.

The deal follows what has become a classic pattern for Sanofi, in which the pharma giant is aligning itself with an up-and-coming biotech, leaving the entrepreneurs largely in charge of innovation. Voyager, a 2014 Fierce 15 company, will take the lead on programs for Parkinson's disease, Friedreich's ataxia and Huntington's disease, as well as other CNS disorders.

Voyager is getting $65 million in cash along with $30 million for an equity stake and "additional in-kind contributions" in the deal. Genzyme gets an option to license several programs following completion of an initial proof-of-concept human clinical trial. But Voyager is retaining all U.S. rights to its lead product programs in Parkinson's disease (VY-AADC01) and Friedreich's ataxia (VY-FXN01). Voyager will split U.S. profits with Genzyme for the Huntington's disease program (VY-HTT01). In addition, Voyager's lead amyotrophic lateral sclerosis (ALS) program (VY-SOD101) is not part of the collaboration and Voyager retains worldwide rights to this program.

Helmed by Eli Lilly ($LLY) R&D vet Steven Paul, Voyager got started with a bankroll from Third Rock Ventures. And it quickly set its sights on existing adeno-associated virus (AAV) technology to get started on clinical programs. The biotech emerged as one of the top companies in a new wave of companies making gene therapy one of the hottest fields in the industry. The bigger companies haven't ignored the trend. In just the past few months Pfizer ($PFE) lined up its own collaboration in gene therapy as Biogen Idec ($BIIB) ramped up an internal program and a partnership of its own.

Voyager's startup plans involved hitching therapeutic payloads to an AAV, allowing it to safely and precisely deliver either a replacement healthy gene or a knock-down blow to a mutated one. But it's also at work on new viral delivery vehicles that could mark a major improvement in the field.

Now, with Genzyme in the fold, Voyager plans to boost its staff of 35 by about 50% over the coming year, Paul said, working to push its Parkinson's program through Phase I and get its early-stage candidates into preclinical development. The two research teams are forming steering committees for each joint program, Paul said, and "it's all about getting down and getting the work done now."

Voyager got off the ground with a Phase Ib study on a therapy for Parkinson's disease that could make a world of difference for patients who don't respond to standard treatment. The old drugs levodopa and carbidopa have long proven themselves effective in treating PD symptoms, but the generic treatments don't work for everyone. Voyager's candidate has the promise of not only reversing nonresponse but also lowering the doses patients need to see results, Paul told FierceBiotech earlier.

Genzyme, which has been working in gene therapy for more than two decades, has had a front-row seat for the field's trip from next big thing to scientific pariah and into its current renaissance, said Mark Barrett, the company's vice president of business development.

"We really carried the torch for years when some other companies left the space," Barrett said. And the Voyager deal "certainly isn't a one-off," he added, as the rare disease specialists at Genzyme are looking at gene therapy as another avenue for treating disease, sitting alongside their core competency of enzyme replacement.

- here's the release

Special Report: FierceBiotech's 2014 Fierce 15 - Voyager Therapeutics

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