BioMarin gene therapy could be on hold 'for several quarters' after FDA mandates new preclinical tests
The FDA is staying cautious after data came out suggesting that one of BioMarin’s experimental gene therapies might have the potential to drive cancers.
Although details remain sparse, BioMarin said on Friday that its gene therapy for the rare disease phenylketonuria, or PKU, will remain on hold after the FDA asked for additional non-clinical — i.e., lab-based or animal studies — to assess “the theoretical oncogenic risk to human study participants.”
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