PRISM Rare Disease Spotlight: Voyager Therapeutics Selects Development Candidate for Friedreich’s Ataxia, Triggers $5M Milestone Payment

Voyager Therapeutics (Nasdaq: VYGR) has selected a lead development candidate in its Friedreich’s Ataxia (FA) program. The company expects the program to advance into first-in-human clinical trials in 2025. Selection of the development candidate triggered a $5 million milestone payment to Voyager, which the Company expects to receive in the first quarter of 2024.

Under the terms of Voyager’s 2019 collaboration agreement with Neurocrine Biosciences (Nasdaq: NBIX), 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.

“The nomination of this development candidate in FA marks an important step in our strategic collaboration with Neurocrine, reflecting the power of combining Voyager’s TRACER AAV capsids and payload design capabilities with Neurocrine’s expertise in neuroscience and clinical development,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “While there has been encouraging recent progress in the treatment of FA, it remains a very challenging and eventually fatal disease for which new therapeutic approaches are needed.”


Voyager’s development candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from the company’s TRACER™ capsid discovery platform.

Voyager’s TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system tropism in multiple species, including non-human primates.

Voyager has established multiple collaboration agreements providing access to its next-generation TRACER™ capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.

The company is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.

About Voyager Therapeutics

Voyager Therapeutics, Inc. is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit

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PRISM Rare Disease Spotlight: Voyager Therapeutics Selects Development Candidate for Friedreich’s Ataxia, Triggers $5M Milestone Payment

Catie Corcoran

Biotech Editor