The FDA has approved BioMarin Pharma’s Roctavian as the first gene therapy for haemophilia A, giving patients an alternative to regular injections of blood clotting factor
Pfizer has reported that its anti-tissue factor pathway inhibitor marstacimab is effective at reducing bleed rates in patients with haemophilia A and B, working as well as
uniQure and CSL have become the first pharma companies to get regulatory approval for a gene therapy for haemophilia B anywhere in the world, after the FDA cleared their Hemgenix therapy fo
Pfizer and Sangamo have restarted recruitment of patients into their registration trial for haemophilia A gene therapy giroctocogene fitelparvovec, after the FDA lifted a clinical hold on t
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