Advertisement Voyager, Genzyme to develop new AAV gene therapies for patients with CNS disorders - Pharmaceutical Business review
Pharmaceutical Business review is using cookies

ContinueLearn More
Close

Voyager, Genzyme to develop new AAV gene therapies for patients with CNS disorders

Voyager Therapeutics and Genzyme have entered into $845m collaboration to develop and commercialize new adeno-associated virus (AAV) gene therapies for patients suffering from severe central nervous system (CNS) disorders.

The collaboration will support the development, expansion and commercialization of Voyager’s new CNS product pipeline.

It will also include several gene therapy programs for Parkinson’s disease (PD), Friedreich’s ataxia and Huntington’s disease, as well as other CNS disorders.

Each program is said to have the potential to deliver transformational therapeutic benefit for patients.

Voyager working in collaboration with Genzyme will drive research and development activities for all programs.

After completion of an initial proof-of-concept human clinical trial, Genzyme will have the option to license several programs.

Voyager president and chief executive officer 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.

"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."

Under the deal, Voyager will retain all US rights to its lead product programs in Parkinson’s disease (VY-AADC01) and Friedreich’s ataxia (VY-FXN01), while it will split US profits with Genzyme for the Huntington’s disease program (VY-HTT01).

Additionally, Voyager’s lead amyotrophic lateral sclerosis (ALS) program (VY-SOD101) is not part of the collaboration and Voyager retains worldwide rights to this program.

The deal will see Genzyme make an upfront commitment of $100m to Voyager, which is also eligible to receive future potential development and sales milestone payments of up to $745m, as well as tiered royalties on product sales.