Genzyme and Voyager collaborate on CNS gene therapies


Genzyme and Voyager Therapeutics are to collaborate on developing novel gene therapies for severe CNS disorders in an agreement worth up to $845 million.

Sanofi subsidiary Genzyme will pay Voyager $100 million upfront, with Voyager eligible to receive potential development and sales payments of up to $745 million, plus further royalties.

The firms will use Genzyme's adeno-associated virus (AAV) gene therapy and Voyager's AAV product engine to develop breakthrough therapies for Parkinson's disease, Friedreich's ataxia and Huntington's disease, as well as other CNS disorders.

Genzyme will have the option to license several programmes after initial human trials, but Voyager will retain all US rights to its programmes in Parkinson's disease (VY-AADC01) and Friedreich's ataxia (VY-FXN01). The firms will split US profits for the Huntington's disease programme (VY-HTT01).

Voyager's amyotrophic lateral sclerosis (ALS) programme VY-SOD101 is not part of the collaboration.

The agreement is a boost for Voyager, which was only launched in 2014, with funding from Third Rock Ventures. CEO Steven Paul said, "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."

Big pharma has increasingly been making inroads in gene therapy research, partnering with up-and-coming biotechs – as Pfizer did in December with Spark Therapeutics and Biogen has done with a joint venture with Italy's San Raffaele-Telethon Institute for Gene Therapy (TIGET) in haemophilia in January.

However, the Voyager news comes on the same day that Sanofi has announced 100 R&D job cuts in Massachusetts at the Genzyme R&D Centre and another smaller cancer research centre, in a move that reflects the company's struggles to find success in the oncology division in the five years since it was established. The oncology business will be absorbed into the overall R&D operation.

Sanofi continues to restructure globally, led by chairman Serge Weinberg following the forced exit of CEO Chris Viehbacher last October. It has a strong pipeline and plans to introduce six products in 2015.


Regeneron/Sanofi in pole position for PCSK inhibitor launch

profile mask

Linda Banks

12 February, 2015