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Voyager Therapeutics has partnered with Sanofi company Genzyme to discover, develop and commercialise adeno-associated virus (AAV) gene therapies for severe central nervous system (CNS) disorders.

Under the deal, Voyager will receive an upfront payment of $100m, including $65m in cash and $30m in equity investment and additional in-kind contributions.

Voyager will also eligible to receive future potential development and sales milestone payments of up to $745m, in addition to tiered royalties on product sales.

"The alliance will carry out multiple gene therapy programmes, including programmes for Parkinson’s, Friedreich’s ataxia and Huntington’s disease."

Voyager president and CEO Steven Paul said: "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."

The alliance will carry out multiple gene therapy programmes, including programmes for Parkinson’s, Friedreich’s ataxia and Huntington’s disease, as well as other CNS disorders.

Voyager will take care of research and development activities for all programmes, while Genzyme will be provided with an option to licence several programmes following completion of an initial proof-of-concept human clinical trial.

As part of the deal, Voyager will hold all US rights to its lead product programmes in Parkinson’s disease (VY-AADC01) and Friedreich’s ataxia (VY-FXN01).

Voyager noted that it will split US profits with Genzyme for the Huntington’s disease programme (VY-HTT01). Its amyotrophic lateral sclerosis (ALS) programme (VY-SOD101) is not part of the collaboration and retains worldwide rights to this programme.

Genzyme president and CEO David Meeker 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."


Image: The entrance to the Genzyme building at 500 Kendall Square in Cambridge, Massachusetts. Photo: courtesy of Tim Pierce.