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.