We are a clinical-stage biotechnology company developing novel medicines to bring the curative power of bone marrow transplant to more patients. Transplant is a well-established and often curative medical procedure, and emerging data on stem cell gene therapy, which is bone marrow transplant using gene-modified stem cells, suggest the potential for meaningful benefit with this newer form of transplant. As it exists today, bone marrow transplant is a large market opportunity, but is limited by the challenge of obtaining sufficient cells to perform the procedure, as well as the inherent mortality of current methods, and complications following transplant. Our portfolio of product candidates includes biologics, small molecules and a cell therapy designed to address deficiencies in existing approaches. Our programs will improve stem cell dose (expansion), stem cell collection (mobilization), patient preparation for transplant (conditioning). Our most advanced product candidate, MGTA-456, is a novel proprietary allogeneic stem cell therapy developed based on the mechanisms that control stem cell growth. MGTA-456 aims to extend the use of cord blood transplant to more patients by increasing the number of stem cells in a single cord blood unit to yield a higher stem cell dose. MGTA-456 achieved human proof-of-concept in Phase I/II trials in a total of 36 patients with blood cancers and is now being studied in patients with fatal inherited metabolic diseases.