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.
FA is a candidate for genetic therapy because it is caused by a mutation in a single gene, FXN. Mutations in FXN cause low production of a protein called frataxin, which leads to the symptoms of FA. Genetic therapies are treatment approaches that aim to address a disease at the DNA level. Gene addition is one type of genetic therapy that is designed to deliver working copies of the FXN gene to cells within the body. These delivered FXN genes would then produce frataxin protein.
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 improved targeting to the central nervous system (CNS).
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.
Discovery research led to the selection of an advanced novel capsid and gene therapy for FA.
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, NBIB-‘223, 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.
Neurocrine is conducting IND-enabling studies for NBIB-‘223.
February 2026: During an investor call, Neurocrine reported plans to initiate a Phase I study for NBIB-‘223 in 2026.
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.
