Business:
Small Molecules for Musculoskeletal Diseases
Drug notes:
Also Clin2 DMD, Clin1 limb-girdle muscular dystrophy, McArdle disease; EDG-7500 Clin0 hypertrophic cardiomyopathy; EDG-003 Clin0 cardiovascular condition
About:
Edgewise Therapeutics is developing precision medicines to treat rare muscle disorders. Patients with Duchenne (DMD) or Becker (BMD) muscular dystrophy new muscle fiber formation cannot keep up with fiber collapse resulting in loss of muscle function. Using an integrated knowledge of muscle physiology at a whole-body level, Edgewise is discovering innovative solutions that directly target the muscle fibers of patients with rare muscle disorders. Powered by their proprietary drug discovery platform, Edgewise is designing their drugs to reduce muscle stress in fast skeletal muscle fibers which are more prone to injury in DMD and BMD patients. For example, Edgewise’s lead candidate, EDG-5506, selectively targets the motor protein myosin to reduce stress and is now in Phase 2 clinical trials.
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