ENA-ASO

ENA-ASO (also known as KMM001) is an 18-mer chimeric antisense oligonucleotide incorporating 2'-O,4'-C-ethylene-bridged nucleic acid (ENA) chemistry. Developed by Kobe University and KNC Laboratories, it is designed to target the splicing enhancer sequence within exon 1 of the myostatin (MSTN) gene. By modulating pre-mRNA splicing, ENA-ASO promotes the production of a novel splicing variant, myostatin-b, which acts as an endogenous myostatin inhibitor. This dual mechanism simultaneously reduces mature myostatin levels and increases myostatin-b, promoting skeletal muscle growth and hypertrophy. ENA-ASO is being investigated for the treatment of Duchenne muscular dystrophy (DMD) and other muscle-wasting conditions, such as chronic kidney disease (CKD)-induced sarcopenia and sepsis-induced muscle atrophy.