FA is a candidate for gene therapy because it is caused by a mutation in a single gene, the frataxin gene, and this mutation is in a non-coding region of the gene. Individuals with FA do not make sufficient frataxin protein. The goal of gene therapy is to supplement the native gene’s frataxin protein production with a frataxin gene that does not contain any mutation and that produces additional frataxin. Neurocrine has partnered with Voyager to use Voyager’s modified AAV capsid to deliver the frataxin gene to all tissues in the body, including brain.
The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.
Voyager has a platform called TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) that is used to discovery AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism.
January 2019: Neurocrine Biosciences, Inc. and Voyager Therapeutics, Inc. announced the formation of a strategic collaboration focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia.
Continued discovery research has advanced a novel capsid and gene therapy for FA that is currently in late stage research.
February 2024: 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.
This brief video serves as an introduction to gene therapy for FA. The aim of this video and accompanying educational resources is to provide information to help individuals and families make informed decisions regarding participation in clinical trials exploring investigational drugs or gene therapy.
