Drugmakers have long studied hemophilia gene therapies, seeking to replace the infusions that people with the disease take for life to control their bleeding.
In recent years, their research has delivered clinical successes in both the “A” and “B” forms of the disease and, in 2022, the first regulatory approvals. Health authorities in Europe in August authorized BioMarin Pharmaceutical’s gene therapy for hemophilia A, while last month the Food and Drug Administration approved CSL and UniQure’s gene therapy for hemophilia B.
