The company's lead candidate for Charcot Marie Tooth Type 1A (CMT1A) has received Orphan Drug Designation and Rare Pediatric Drug Designation, and is advancing toward IND-enabling studies as of Q2 2024. The company is also developing a preclinical vectorized RNAi asset for the rare neuromuscular disease Facioscapulohumeral Muscular Dystrophy (FSHD).