Genzyme teams up with Voyager Therapeutics to develop AAV gene therapies for CNS disorders


Will make an upfront payment of US$100m to Voyager

Sanofi's Genzyme and Voyager Therapeutics, a Cambridge, MA, US-based gene therapy company, are to collaborate in the development of novel gene therapies for severe CNS disorders.

The collaboration will leverage Genzyme's leadership in the field of adeno-associated virus (AAV) gene therapy and Voyager's AAV product engine to develop breakthrough therapies for these conditions.

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

The alliance will encompass multiple gene therapy programmes for Parkinson's disease, Friedreich's ataxia and Huntington's disease, as well as other CNS disorders. The collaboration portfolio created will combine programmes and intellectual property from both companies.

Voyager will drive research and development activities and Genzyme will have the option to license several programmes following completion of an initial proof-of-concept human clinical trial.

Voyager will retain US rights to its lead product programmes in Parkinson's disease (VY-AADC01) and Friedreich's ataxia (VY-FXN01). The company will split US profits with Genzyme for the Huntington's disease programme (VY-HTT01).

Voyager's lead amyotrophic lateral sclerosis (ALS) programme (VY-SOD101) is not part of the collaboration and the company retains worldwide rights.

David Meeker, President and CEO of Genzyme, said: '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.

Sign up for your free email newsletter

'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.'