Voyager Therapeutics, Inc. Announces Selection of Gene Therapy Development Candidate for Friedreich's Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment
February 26, 2024 at 07:00 am EST
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Voyager Therapeutics, Inc. announced that the joint steering committee with its collaborator Neurocrine Biosciences has selected a lead development candidate in the Friedreich?s ataxia (FA) program. The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager?s TRACER? capsid discovery platform.
The companies expect the program to advance into first-in-human clinical trials in 2025. The FA program is being developed under the 2019 strategic collaboration agreement between Voyager and Neurocrine Biosciences for research, development, and commercialization of certain AAV gene therapy products for programs targeting Friedreich?s ataxia and two other undisclosed targets. Under the terms of the 2019 collaboration agreement, Voyager is eligible to receive up to $1.3 billion in potential development and commercial milestone payments, tiered royalties on net sales, and program funding, and Voyager could exercise an option for 60/40 cost- and profit-sharing (Neurocrine/Voyager) in the U.S. for the FA program following the determination by the joint steering committee of proof of mechanism based on established milestones and metrics.
Voyager Therapeutics, Inc. is a biotechnology company focused on advancing neurogenetic medicines. The Companyâs pipeline includes programs for Alzheimerâs disease, amyotrophic lateral sclerosis (ALS), Parkinsonâs disease, and multiple other diseases of the central nervous system. Many of its programs are derived from its TRACER AAV capsid discovery platform, which is used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Its pipeline of programs, all of which are in preclinical development, include Anti-Tau Antibody (VY-TAU01), SOD1 Silencing Gene Therapy Program, Tau Silencing Gene Therapy Program, Vectorized Anti-Amyloid Antibody Early Research Program, Friedreichâs Ataxia Program: VY-FXN01, GBA1 Gene Replacement Program, HD Program, and others. VY-TAU01 is for the treatment of Alzheimerâs disease. SOD1 Silencing Gene Therapy Program is for the treatment of ALS.
Voyager Therapeutics, Inc. Announces Selection of Gene Therapy Development Candidate for Friedreich's Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment