Acceleron is dedicated to the discovery and development of innovative, life-changing medicines for patients with a wide range of serious and rare diseases. Our research focuses on harnessing the power of the TGF-beta protein superfamily, the target-rich area of biology behind the body’s remarkable capacity for cellular growth and repair. Our approach to drug discovery is driven by deep scientific expertise—and an urgency to deliver effective treatments to thousands of patients who have few or no therapeutic options. The Company’s lead product candidate, luspatercept, is a first-in-class erythroid (red blood cell) maturation agent (EMA) being developed to treat patients who have serious blood disorders marked by a late-stage defect in the maturation of red blood cells.
Alongside our partner Celgene—a leader in hematology—we are advancing multiple Phase 2 and Phase 3 trials of luspatercept in five target indications across myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing a neuromuscular program with a locally-acting “Myostatin+” agent, ACE-083.
We recently initiated a Phase 2 trial of sotatercept in pulmonary arterial hypertension (PAH). Acceleron has ongoing preclinical research efforts targeting additional indications in these three disease areas in which there is high unmet medical need.