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
Sanofi and Sobi’s haemophilia partnership has been under competitive pressure from new therapies like Roche’s antibody Hemlibra, and are hoping that a newly-approved long-
BioMarin's has signed the first contract with an insurer in Germany for Roctavian, its recently approved gene therapy for haemophilia A, as it tests out its outcomes-based agreement (OBA) r
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
BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe
In an industry growing as quickly as cell therapy, keeping up with the demands of manufacturing and treatment is a challenge for the whole industry to tackle together, including stakeholder
Drug discovery is usually a goal-driven and iterative process, as researchers look for innovative new routes to target particular diseases and study those diseases in afflicted patients to learn ho