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BioMarin reports cancer case in hemophilia gene therapy trial

Posted on: 14 September 2022, source: Biopharmadive
Drug regulators have not ordered a trial hold after an analysis suggested leukemia was naturally occurring, company says. BioMarin Pharmaceutical, a California-based biotechnology company, said a patient in a clinical trial of its hemophilia gene therapy Roctavian has developed leukemia, although testing suggests that the case may be naturally occurring..