LEXEO Therapeutics Receives Rare Pediatric Disease & Orphan Drug Designations for LX1004
Updated: Sep 9
Phase 1 clinical trial of LX1004 complete; pivotal studies planned for 2022
NEW YORK – July 28, 2021 (GLOBE NEWSWIRE) – LEXEO Therapeutics, a fully integrated clinical-stage gene therapy company advancing disease-modifying treatments for genetic conditions, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation and Orphan Drug designation to LX1004 for the treatment of CLN2 Batten disease, a fatal genetic condition of early childhood caused by a mutation in the CLN2 gene, which results in cognitive impairment, blindness, seizures, and loss of motor function and, untreated, leads to death at a young age. LX1004 is an AAV-mediated gene therapy delivering CLN2 to the central nervous system.