The US Food and Drug Administration has placed on clinical hold a trial of BioMarin’s AAV gene therapy BMN 307 for the rare disease phenylketonuria (PKU) after cases of liver cancer in mice receiving high doses of the therapy were reported.
BioMarin Trial Halt Raises Another Safety Flag On AAV Gene Therapy
US FDA Safety Probe Is Ongoing
Animal studies have shown that AAV viral vectors can integrate into animal cell DNA – but whether that causes cancer, or could happen in humans, remains uncertain.
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