NBIB-223

NBIB-223 is an investigational adeno-associated virus (AAV)-based gene therapy being developed by Neurocrine Biosciences in collaboration with Voyager Therapeutics for the treatment of Friedreich's Ataxia (FA). FA is a rare, progressive neurodegenerative disorder caused by mutations in the FXN gene, leading to a deficiency of the frataxin protein. NBIB-223 utilizes Voyager's proprietary TRACER™ (Tropism Redirection of AAV Capsids by Evolution of RNA) platform, which employs a modified AAV capsid designed to efficiently cross the blood-brain barrier and deliver functional copies of the FXN gene to the central nervous system and other affected tissues. The therapy aims to restore frataxin protein production to halt or reverse the progression of the disease. As of mid-2026, the program is in the IND-enabling stage, with plans to initiate Phase 1 clinical trials in the second half of 2026.

Other names

NBIB-223NBIB223NBIB 223