ACE-083 is an investigational protein therapeutic that has been designed for local administration to increase muscle mass and strength in specific muscles and muscle groups. Acceleron is developing ACE-083 for diseases in which improved muscle strength may provide a clinical benefit, such as inclusion body myositis and certain forms of muscular dystrophy.
Select proteins in the TGF-β protein superfamily normally function to modulate muscle growth. ACE-083 has been designed to block those proteins and, in doing so, can increase muscle mass and strength. ACE-083 has been engineered to exert this action only within the muscle or muscle group treated. In preclinical animal studies, ACE-083 has shown selective and dose-dependent increases in muscle mass in treated muscles with no effects in untreated muscles.