Aiming at AAV therapies

CAMBRIDGE, Mass.—Genzyme, a Sanofi company, and gene therapy company Voyager Therapeutics have signed a major strategic collaboration for the discovery, development and commercialization of novel AAV gene therapies for severe central nervous system (CNS) disorders. The collaboration will include a number of gene therapy programs, including those for Parkinson’s disease, Friedreich's ataxia and Huntington’s disease, as well as other CNS disorders. The resulting collaboration portfolio will combine programs and intellectual property from both Genzyme and Voyager Therapeutics.

“We’ve been working in gene therapy for more than 20 years, and our commitment remains strong because we believe in its promise to transform the lives of patients with debilitating diseases,” said David Meeker, president and CEO of Genzyme. “The field of gene therapy has advanced rapidly in the past several years, and the science has never been better. Joining together with a great partner in Voyager allows us to strengthen and accelerate our efforts to address a set of very challenging neurological diseases.”

Per the terms of the collaboration, Genzyme will make an upfront commitment to Voyager of $100 million, including $65 million in cash, a $30-million equity investment in Voyager and additional in-kind contributions. Voyager is also eligible to receive future potential development and sales milestone payments of up to $745 million, in addition to tiered royalties on product sales.

Voyager will take point in leading research and development activities for all programs, and will collaborate closely with Genzyme. For its part, Genzyme will have the option of licensing several programs following the completion of an initial proof-of-concept clinical trial in humans. Voyager will retain all U.S. rights to its lead product programs in Parkinson’s disease (VY-AADC01) and Friedreich's ataxia (VY-FXN01), and will split U.S. profits with Genzyme for its Huntington’s disease program, VY-HTT01.

“Through our combined efforts, Voyager and Genzyme are at the forefront of converting the promise of gene therapy into innovative therapies for CNS disorders that make a meaningful difference in patients’ lives,” said Steven Paul, M.D., president & chief executive officer of Voyager. “Voyager is proud to be collaborating with one of the biotechnology industry pioneers of gene therapy to not only advance development of our lead programs, but also expand our pipeline into new CNS diseases in need of new treatments. This strategic collaboration provides significant funding to drive the development of our expanded product pipeline, while also allowing Voyager to continue to thrive as an independent company.”

SOURCE: Genzyme press release