Changing the world

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for the treatment of severe haemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

Bloomsbury Genetic Therapies is a new UCLB biotech spinout with ambitions to go far and fast. Formally launched in October, the company is leveraging a powerful partnership of four UCL academics to develop new treatments for rare genetic diseases.