mRNA-LNP myostatin propeptide

mRNA-LNP myostatin propeptide is an investigational mRNA-based therapy delivered via lipid nanoparticles (LNPs) that encodes a cleavage-resistant myostatin propeptide [1.49P]. Developed by researchers at the University of Hawaii at Manoa, this therapy is designed for intramuscular delivery to selectively inhibit myostatin, a negative regulator of skeletal muscle growth, without off-target inhibition of other TGF-β family members. The cleavage-resistant propeptide binds to co-secreted mature myostatin, maintaining it in an inactive latent complex [1.49P]. In preclinical mouse models, local delivery of the mRNA-LNP myostatin propeptide has demonstrated transient, dose-dependent expression and site-specific skeletal muscle hypertrophy, offering a potential therapeutic approach for muscle wasting disorders [1.49P].