David Melamed, PhD,  —

The first participant was dosed with the investigational gene therapy treatment SB-525 (giroctocogene fitelparvovec) in a Phase 3 clinical trial in men with hemophilia A, according to developers Pfizer and Sangamo Therapeutics. AFFINE (NCT04370054) is a pivotal trial, which means it is intended to…

Long-term prophylaxis, or preventive treatment with Adynovate continues to safely reduce bleeding events over multiple years in children and adults with severe hemophilia A without causing neutralizing antibodies, a Phase 3b trial found. The study, “Long‐term safety and efficacy results from the phase…

A Phase 3 clinical trial investigating gene therapy candidate AMT-061 (etranacogene dezaparvovec) has reached its target of dosing 50 participants with moderate or severe hemophilia B. In fact, according to AMT-061 developer uniQure, 54 patients have been given the one-time dose of AMT-061 in the HOPE-B…

When given at low doses, extended half-life factor IX concentrates Alprolix (eftrenonacog alfa) and Idelvion (albutrepenonacog alfa) can prevent spontaneous bleeds in patients with severe hemophilia B, a study has found. The study, “A single centre retrospective study of low dose prophylaxis with…