LX1004 Gene Therapy Wins Orphan Drug, Rare Pediatric Disease Status

LX1004 Gene Therapy Wins Orphan Drug, Rare Pediatric Disease Status

301900

LX1004 Gene Therapy Wins Orphan Drug, Rare Pediatric Disease Status

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to LX1004, an investigational gene therapy for CLN2 disease, also known as late infantile Batten disease. “Both designations granted to LX1004 underscore the critical importance and urgency to advance new treatment approaches for CLN2 Batten disease, a fatal genetic disorder affecting the central nervous system (CNS),” R. Nolan Townsend, CEO of Lexeo Therapeutics, the therapy’s developer, said in a…

You must be logged in to read/download the full post.