Asc Therapeutics

ASC Therapeutics is a biopharmaceutical company pioneering the development of gene replacement therapies, in vivo gene editing and allogeneic cell therapies for hematological, metabolic, and other rare diseases. Led by a management team of industry veterans with significant global experience in gene and cell therapy, ASC Therapeutics is developing... multiple therapeutic programs based on four technology platforms: 1) In-vivo gene therapy of inherited blood clotting disorders, initially focusing on ASC618, second generation gene therapy for hemophilia A, for which U.S. FDA IND clearance, Orphan Drug and Fast Track Designations were received; 2) In-vivo gene therapy in metabolic disorders, initially focusing on Maple Syrup Urine Disease, in collaboration with the Universities of Massachusetts and Pennsylvania; 3) In-vivo gene editing, initially focusing on ASC518 for hemophilia A; and 4) Allogeneic cell therapy, the first indication with a Decidua Stromal Cell-based therapy for steroid-refractory acute Graft-versus-Host Disease, for which U.S. FDA IND clearance and Orphan Drug Designation were received.Read More