Voyager Therapeutics, a gene therapy company headquartered in Cambridge, Mass. developing treatments for diseases of the central nervous system (CNS), and Genzyme, a Sanofi company and gene therapy pioneer, have announced a major strategic collaboration to discover, develop and commercialize novel gene therapies for severe CNS disorders. The collaboration will leverage Genzyme’s long-standing commitment and experience in adeno-associated virus (AAV) gene therapy and Voyager’s AAV product engine to develop breakthrough therapies for patients suffering from severe CNS disorders.

The alliance will encompass multiple gene therapy programs, including programs for Parkinson’s disease, Friedreich’s ataxia and Huntington’s disease, as well as other CNS disorders. Each program targets a severe, debilitating disease and has the potential to deliver transformational therapeutic benefit for patients. The collaboration portfolio created will combine programs and intellectual property from both companies.

Voyager will drive R&D activities for all programs, working with Genzyme in a highly collaborative way. Genzyme will have the option to license several programs following completion of an initial proof-of-concept human clinical trial. However, Voyager will retain all U.S. rights to its lead product programs in Parkinson’s (VY-AADC01) and Friedreich’s ataxia (VY-FXN01). Voyager will split U.S. profits with Genzyme for the Huntington’s 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.

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

“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 and CEO 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.”

“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.”