LEXEO Therapeutics Receives Rare Pediatric Disease Designation and Orphan Drug Designation for LX1004 for the Treatment of CLN2 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), said R. Nolan Townsend, Chief Executive Officer of LEXEO Therapeutics.
- 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), said R. Nolan Townsend, Chief Executive Officer of LEXEO Therapeutics.
- The FDA grants Rare Pediatric Disease designation for serious and life-threateningdiseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S.
- CLN2 Batten disease is an autosomal recessive lysosomal storage disease with approximately 1,000 cases worldwide.
- LEXEOs current pipeline consists of adeno-associated virus (AAV)-mediated gene therapies in rare cardiac diseases, CLN2 Batten disease, and APOE4-associated Alzheimers disease.