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 strategy is to develop medicines that selectively target multiple ligands (proteins) in the TGF-beta superfamily, which play critical roles in the development of serious diseases. Our deep scientific leadership in this area of biology has enabled us to build a pipeline of innovative agents or therapeutic candidates – including our first FDA-approved medicine. Driven by an urgency to deliver effective treatments to thousands of patients who have few or no therapeutic options, we continue to expand our pipeline with a focus on the maturation of red blood cells, growth of skeletal muscle, restoration of pulmonary vasculature homeostasis, as well as other biology that plays an important role in in serious diseases.
Along with our partner Celgene, we recently announced that the U.S. Food and Drug Administration (FDA) approved REBLOZYL® (luspatercept-aamt) — a recombinant fusion protein that binds several TGF-beta superfamily ligands.
In parallel to initiating the commercialization of REBLOZYL®, we and Celgene continue to expand on the opportunity with this erythroid maturation agent, advancing multiple Phase 2 and Phase 3 trials of luspatercept-aamt in three additional target indications across myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. We are also advancing Acceleron-led neuromuscular and pulmonary disease programs with a locally-acting “Myostatin+” agent called ACE-083 in Charcot-Marie-Tooth disease, as well as sotatercept in pulmonary arterial hypertension (PAH).
Acceleron has ongoing preclinical research efforts targeting additional disease areas in which there is high unmet medical need.