Expression Therapeutics LLC

Hemophilia A and B Pipeline Advances with Novel Therapies in Clinical Trials

8 months ago

• Several companies are actively developing novel therapies for Hemophilia A and B, with many in various phases of clinical trials, showing promise for improving treatment options. • TiumBio Co., Ltd. has submitted a Clinical Trial Application to initiate a Phase 1b investigation of TU7710, a new recombinant activated factor VII for hemophilia patients with inhibitors. • The FDA approved ALTUVIIIO (efanesoctocog alfa) in February 2023, a factor VIII replacement therapy for adults and children with Hemophilia A, for routine prophylaxis and on-demand treatment. • BioMarin received FDA approval for Roctavian in June 2023, a single-dose gene therapy for severe Hemophilia A, marking a significant advancement in treatment.