DONATE NOW

  • Image

LX1004' Wins Orphan Drug and Rare Pediatric Disease Status

5 August 2021

CLN2 Gene Therapy ‘LX1004’ Wins Orphan Drug and Rare Pediatric Disease Status
The U.S. 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 press release.
Townsend said Lexeo is pursuing LX1004’s development “with the hope of making it available for patients as soon as possible.”