GA-001 (Genosera)

GA-001 is a preclinical gene therapy candidate being developed by Genosera for the treatment of GNE myopathy, a rare, progressive, adult-onset recessive genetic muscle disorder. The disease is caused by loss-of-function mutations in the *GNE* gene, which encodes the bifunctional enzyme UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase, a rate-limiting step in the sialic acid biosynthetic pathway. GA-001 aims to deliver a functional copy of the *GNE* gene to restore enzyme activity and sialic acid production, potentially halting or reversing muscle degeneration. Genosera plans to hold a pre-IND meeting with the FDA in 2026, with clinical trials anticipated to begin in 2027.