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BioMarin Trial Halt Raises Another Safety Flag On AAV Gene Therapy

US FDA Safety Probe Is Ongoing

06 Sep 2021

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.

BioMarin is the latest company to be hit by AAV gene therapy safety doubts, as the FDA takes a cautious approach to the burgeoning field.

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.

The animal study showed evidence that portions of adeno-associated viral (AAV) vector had integrated into the host genome – a mechanism that has been observed in animal studies for other AAV