Author Archives: Marta Figueiredo PhD

BioMarin Resubmits Roctavian Gene Therapy for EU Approval

BioMarin Pharmaceutical has resubmitted a regulatory application seeking approval in Europe for its experimental gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. That resubmission, to the European Medicines Agency (EMA), included one-year follow-up data from BioMarin on the therapy’s safety and effectiveness in treating the rare genetic…

Hemophilia B Gene Therapy AMT-061 Safe, Effective, Trial Data Show

AMT-061 (etranacogene dezaparvovec), uniQure‘s investigational gene therapy for hemophilia B, is safe and effective — even in patients with pre-existing antibodies against the treatment’s viral carrier, according to new trial data. Data from the Phase 3 HOPE-B trial suggest that nearly all hemophilia B patients, regardless of pre-existing viral…

1st Patient Dosed in Phase 3 Trial of Injectable MarzAA

The first participant has been dosed in a Phase 3 clinical trial testing marzeptacog alfa activated (MarzAA), an experimental under-the-skin therapy for hemophilia A and B patients with inhibitors. The study, called Crimson-1 (NCT04489537) and sponsored by the therapy’s developer, Catalyst Biosciences, is currently recruiting patients at…