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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An expert pointed out that XLMTM patients are already at high risk, but if linked to therapy toxicity, the death could doom the ASPIRO trial.
In this week's podcast edition of Scrip's Five Must-Know Things: US industry warns over pricing; a setback for another gene therapy; a Phase II disappointment for Novartis; Actemra shortages cause CAR-T worries; and Sweden's Sobi set to go private.
The day before an FDA advisory committee meeting about the safety of AAV vector-based gene therapies, Astellas reported liver damage in a patient in its trial of AT132.