Voyager Therapeutics said it will develop its lead candidate VY-AADC01 gene therapy for advanced Parkinson’s disease on its own, retaining full global rights, after Genzyme, a Sanofi company, opted against exercising its exclusive development and commercial rights to the program outside the U.S.

Voyager insisted that the decision did not signal an end to the companies’ up-to-$845 million collaboration, launched in 2015. Voyager and Genzyme agreed to develop and commercialize new adeno-associated virus (AAV) gene therapies for central nervous system (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. 

“Sanofi Genzyme remains an important collaborator for Voyager, and we remain fully committed to progressing our Huntington’s disease and Friedreich’s ataxia programs under the ongoing collaboration,” Voyager president and CEO Steven Paul, M.D., said yesterday in a statement.

Genzyme retains outside-U.S. opt-in rights to the Friedreich’s Ataxia program and outside-U.S. opt-in and U.S. co-promotion rights to the Huntington’s disease program.

“We are very pleased to gain full worldwide rights to our Parkinson’s disease program that recently demonstrated robust and durable clinical effects from the ongoing Phase Ib trial,” Dr. Paul added.

On September 6, Voyager trumpeted positive results from its ongoing Phase Ib trial of VY-AADC01 in advanced Parkinson’s disease. The results, according to Voyager, showed durable, dose-dependent and time-dependent improvements across multiple measures of patients’ motor function after a one-time administration of the gene therapy.  

“For patients in Cohort 2 at 12 months, this meant an average increase during the day of four hours of on-time without dyskinesia, which is a very meaningful change,” Voyager CMO Bernard Ravina, M.D., M.S., stated at the time.

Dr. Paul said Voyager plans to start a global, pivotal Phase II/III program “later this year,” and announce next steps for its Parkinson’s program at the company’s upcoming third-quarter conference call, set for Thursday at 4:30 pm ET.

All of Voyager’s other gene therapy programs are in preclinical phases. They include the Huntington’s disease program (VY-HTT01) and Friedreich’s ataxia program (VY-FXN01), as well as programs for monogenic amyotrophic lateral sclerosis (VY-SOD101), frontotemporal dementia/Alzheimer’s disease (VY-TAU01), and severe chronic pain (VY-NAV01).








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