Liver tumors in animals trigger clinical hold for BioMarin gene therapy — highlighting a recurring concern
Hours after the FDA wrapped its marathon listening session on the safety risks of gene therapy, BioMarin offered a reminder of why the advisory committee meeting was convened in the first place.
BioMarin is halting worldwide enrollment to a Phase I/II study of its experimental gene therapy for phenylketonuria following a clinical hold based on interim safety findings from a preclinical pharmacology study — namely, tumors found in the liver of certain animals given the drug.
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