The collaboration will leverage Genzyme’s long-standing commitment and scientific leadership in the field of adeno-associated virus (AAV) gene therapy and Voyager’s industry-leading AAV product engine to develop breakthrough therapies for patients suffering from severe CNS disorders.
The alliance will encompass multiple gene therapy programs, including programs for Parkinson’s disease, Friedreich’s ataxia and Huntington’s disease, as well as other CNS disorders.
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Each program targets a severe, debilitating disease and has the potential to deliver transformational therapeutic benefit for patients.
The collaboration portfolio created will combine programs and intellectual property from both companies.
Voyager will drive research and development activities for all programs, working with Genzyme in a highly collaborative way.
Genzyme will have the option to license several programs following completion of an initial proof-of-concept human clinical trial.
However, Voyager will retain all U.S. rights to its lead product programs in Parkinson’s disease (VY-AADC01) and Friedreich’s ataxia (VY-FXN01).
Voyager will split U.S. profits with Genzyme for the Huntington’s disease program (VY-HTT01).
In addition, Voyager’s lead amyotrophic lateral sclerosis (ALS) program (VY-SOD101) is not part of the collaboration and Voyager retains worldwide rights to this program.
Genzyme will make an upfront commitment of $100 million to Voyager, including $65 million in cash, a $30 million equity investment in Voyager and additional in-kind contributions.
Voyager is eligible to receive future potential development and sales milestone payments of up to $745 million, as well as tiered royalties on product sales.
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