GC1126A

GC1126A is a novel, engineered ADAMTS13 mutein (recombinant protease) being developed by GC Biopharma for the treatment of Thrombotic Thrombocytopenic Purpura (TTP). TTP is a rare and life-threatening hematologic disorder characterized by a deficiency in ADAMTS13 activity, which leads to the accumulation of ultra-large von Willebrand factor (vWF) multimers and subsequent systemic microvascular thrombosis. GC1126A is specifically designed to evade anti-ADAMTS13 autoantibodies, which are common in acquired TTP, and features an extended half-life compared to wild-type ADAMTS13. By restoring the proteolytic cleavage of vWF, the drug aims to prevent platelet aggregation and organ damage. It received Orphan Drug Designation from the U.S. FDA in September 2023.