LiCO-siMSTN

LiCO-siMSTN is a lipid-conjugated small interfering RNA (siRNA) designed to target and knock down myostatin (MSTN) expression. Developed by Ractigen Therapeutics, the therapeutic utilizes the proprietary LiCO (Lipid-Conjugated Oligonucleotide) platform, which conjugates an optimized lipid moiety to the duplex RNA via a benzimidazole-based Smart Diverse Linker (SDL) at the 5' end of the passenger strand. This conjugation facilitates cellular uptake via CD36-mediated endocytosis, enabling systemic delivery to extrahepatic tissues such as skeletal muscle, cardiac muscle, and adipose tissue. In preclinical studies, systemic administration of LiCO-siMSTN in non-human primates achieved robust knockdown of MSTN mRNA in skeletal muscles, sustained suppression of circulating myostatin protein, and progressive functional muscle hypertrophy with a favorable safety profile.