Updated Follow-Up Of The Alta Study, A Phase 1/2, Open Label, Adaptive, Dose-Ranging Study To Assess The Safety And Tolerability Of Sb-525 Gene Therapy In Adult Patients With Severe Hemophilia A

Introduction: Hemophilia A is a rare blood disorder caused by an F8 variant resulting in insufficient Factor VIII (FVIII) activity. Updated results and follow-up of an ongoing gene therapy study in patients with severe hemophilia A are presented.