Genzyme, a Sanofi company, and Voyager Therapeutics said today they have launched an up-to-$845 million collaboration to develop and commercialize new adeno-associated virus (AAV) gene therapies for CNS disorders.
The partnership is designed to combine Genzyme’s expertise in AAV gene therapy with Voyager’s AAV product engine, with the goal of commercializing multiple gene therapy programs—including programs for Parkinson’s disease, Friedreich’s ataxia, and Huntington’s disease, as well as other CNS disorders. Genzyme and Voyager said their collaboration portfolio will combine programs and intellectual property from both companies.
Under their gene-therapy alliance, Voyager will oversee research and development activities for all programs, while Genzyme will have the option to license several programs following completion of an initial proof-of-concept human clinical trial.
Voyager will retain all U.S. rights to its lead product programs in Parkinson’s disease (VY-AADC01) and Friedreich’s ataxia (VY-FXN01), with Genzyme holding outside-U.S. rights. VY-AADC01 is in an ongoing Phase Ib study by Voyager with collaboration partners at the University of California, San Francisco; all of Voyager’s other gene therapy programs are in preclinical phases.
Voyager will split U.S. profits with Genzyme for the Huntington’s disease program (VY-HTT01). However, Voyager retains worldwide rights to its lead amyotrophic lateral sclerosis (ALS) program (VY-SOD101), which is not part of the collaboration.
Genzyme has agreed to pay Voyager $100 million upfront—to consist of $65 million in cash, a $30 million equity investment in Voyager, and additional unspecified in-kind contributions. Voyager will also be eligible for future potential development and sales milestone payments of up to $745 million, as well as tiered royalties on product sales.
“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,” Steven Paul, M.D., Voyager’s president and CEO since September 2014, said in a statement.
Headquartered in Cambridge, Mass., Voyager is a private company launched in 2014 with $45 million in Series A venture capital financing from Third Rock Ventures.