Author Archives: Margarida Maia

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…