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FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

biopharmadive.com
 2021-09-07

Cover picture for the articleThe Food and Drug Administration has suspended testing of an experimental BioMarin Pharmaceutical gene therapy for the rare inherited disease phenylketonuria due to safety concerns reported in preclinical testing. In a study meant to test the durability of BioMarin's gene therapy, researchers found evidence the virus delivering the corrected...

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