Pleightlet / Platelet Targeted Therapeutics - LARVOL DELTA

Follow-up on Platelet-Targeted Gene Therapy for Hemophilia A (ASGCT 2025) - P1 | "At approximately 3.5 months after HSC transplant the patient discontinued his weekly prophylaxis regimen with Emicizumab (FVIII-mimetic) and has reported an absence of bleeding events and denied administration of rFVIII prophylaxis at least 24 months after transplant. Two patients have safely undergone treatment of severe HA with a history of inhibitors using Pleightlet LV HSC FVIII gene therapy causing platelet-targeted expression of BDDFVIII now sustained for greater than 24 and 6 months (respectively) after transplant with zero bleeding events requiring infusion of FVIII products. Regular testing for gene vector copy number, insertion site analysis, platelet FVIII levels, and coagulation function indicates the potential feasibility for this approach to treat hemophilia A in a safe and efficient manner. This study will require periodic long-term follow-up to further demonstrate the efficacy of this approach."

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis • CD34 • ITGA2B