Sanofi, Voyager Therapeutics enter $845-million deal on gene therapies for CNS disorders

Sanofi's Genzyme unit and Voyager Therapeutics entered an agreement potentially worth up to $845 million to develop gene therapies for severe CNS disorders, the companies reported Wednesday. The collaboration will combine Genzyme's experience in the field of adeno-associated virus (AAV) gene therapy and Voyager's AAV product engine to develop therapies for multiple programmes, including Parkinson's disease, Friedreich's ataxia and Huntington's disease.

The drugmakers noted that under the deal, Genzyme will make an upfront payment of $65 million in cash, along with a $30 million equity investment in Voyager, with the latter eligible to receive future milestones of up to $745 million, in addition to tiered royalties on product sales.

Under the agreed terms, Voyager will lead R&D activities for all programmes. Genzyme will hold an option to licence several programmes following completion of an initial proof-of-concept study, while Voyager will retain US rights to its lead product programmes in Parkinson's disease, VY-AADC01, and Friedreich's ataxia, VY-FXN01. The companies will share US profits for the Huntington's disease programme, VY-HTT01, while Voyager’s lead amyotrophic lateral sclerosis programme, VY-SOD101, is not part of the collaboration.

Genzyme CEO David Meeker said "the field of gene therapy has advanced rapidly in the past several years, and the science has never been better," adding "joining together with...Voyager allows us to strengthen and accelerate our efforts to address a set of very challenging neurological diseases."

In 2012, the European Commission approved uniQure's Glybera (alipogene tiparvovec) for patients with lipoprotein lipase deficiency suffering from recurring acute pancreatitis, making the product the first gene therapy approved by regulators in the Western world. Other companies have also been entering the field, with Bayer partnering with Dimension Therapeutics last year to develop a gene therapy for haemophilia A.

To read more Top Story articles, click here.