Companies in #Friedreich's Ataxia

Biogen is a leading global biotechnology company that discovers, develops, and delivers innovative therapies for serious neurological and neurodegenerative diseases. Its portfolio includes medicines for multiple sclerosis, spinal muscular atrophy, Alzheimer's disease, and rare diseases, with a growing late-stage neurology pipeline and recent expansion into rare diseases through the Reata Pharmaceuticals acquisition.

Therapeutics company focused on Friedreich's Ataxia. Received IND clearance for GV101 from the FDA in August 2026.

Minoryx Therapeutics is a clinical-stage biotechnology company developing differentiated therapies for X-linked adrenoleukodystrophy (X-ALD) and other orphan CNS diseases. Its lead candidate, leriglitazone, is an oral, brain-penetrant PPAR-γ agonist for cerebral ALD and Friedreich's ataxia.

Voyager Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to leveraging human genetics to develop neurogenetic medicines and gene therapies for neurological diseases. Founded in 2013 and headquartered in Lexington, Massachusetts, Voyager utilizes its proprietary TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform to engineer novel adeno-associated virus (AAV) capsids capable of crossing the blood-brain barrier with enhanced central nervous system tropism and reduced off-target organ distribution. The company is advancing a proprietary and partnered pipeline targeting Alzheimer's disease (including VY7523, an anti-tau monoclonal antibody in Phase 1, and VY1706, a tau-silencing AAV gene therapy entering Phase 1), amyotrophic lateral sclerosis (ALS), Parkinson's disease, and Friedreich's ataxia (NBIB-223, partnered with Neurocrine Biosciences). In November 2026, Voyager disclosed in its Q3 Form 10-Q an implemented workforce reduction and $1.4 million in restructuring charges to reduce operating expenditures and extend cash runway into Q2 2028.