SMA – THERAPY: P.270 Myostatin dynamics in health and disease: Pharmacologic effects of SRK-015, a highly selective monoclonal antibody inhibitor of myostatin activation

Abstract

Myostatin, an important negative regulator of muscle mass (particularly that of fast-twitch fibers), is synthesized as an inactive precursor, requiring two proteolytic cleavage steps to release the active growth factor. Understanding myostatin production and activation in health and disease may provide important insight into the potential effects of anti-myostatin therapeutics. Scholar Rock is currently developing and investigating SRK-015, a monoclonal antibody that selectively binds and inhibits the activation of latent myostatin, for the treatment of spinal muscular atrophy (SMA).