The candidate is a replacement for the mutated gene FXN (which codes for the protein frataxin) inside a protein shell known as a capsid. This newly selected candidate is part of the Voyager Therapeutics and Neurocrine Biosciences capsid discovery platform TRACER™. TRACER-generated capsids have shown the ability to enter cells and parts of the brain and central nervous system that are traditionally hard to reach. The companies expect the FA program to advance into its first-in-human clinical trials in 2026. Read the press release from Voyager Therapeutics here.
On 7th May 2026, Voyager Therapeutics provided an update a via press release. Neurocrine completed GLP toxicology (pre-clinical safety assessments) with their investigational gene therapy, NBIB-‘223, for FA. They have also received FDA orphan drug designation, a status granted to drugs by the FDA, the US medicines regulator, to treat rare diseases that provides certain incentives. Neurocrine has stated that it intends to initiate a clinical trial with NBIB-‘223 in the second half of 2026, pending successful FDA IND clearance (formal authorisation that the investigational drug can be used in a clinical trial). Read the press release here.
