As a critical regulator of muscle growth and disease progression, GDF-8 is currently a significant target for treating skeletal muscle diseases, neuromuscular diseases, obesity, and cancer. Scholar Rock's SRK-015 (Apitegromab) is a representative drug in this category. SRK-015 is a fully human monoclonal antibody that binds with high specificity to human Pro-GDF-8 or Latent GDF-8 without binding to mature GDF-8 and other closely related growth factors. It inhibits GDF-8 before release, offering high selectivity and minimal side effects. SRK-015 is currently in Phase 3 clinical trials (NCT05156320) for spinal muscular atrophy (SMA) and is the first potential muscle-directed therapy for SMA. Roche's RO7204239 (GYM329, RG6237) targets Latent myostatin and is being tested for SMA (NCT05115110) and facioscapulohumeral muscular dystrophy (NCT05548556).
