The exact cause of a fourth death in the Phase I/II ASPIRO study of Astellas Pharma, Inc.’s gene therapy AT132 in X-linked myotubular myopathy (XLMTM) remains under investigation. But while it occurred in a disease whose patients are already known to be highly vulnerable, it comes at an awkward time as the safety of adeno-associated viral (AAV) vector-based gene therapies are facing increased scrutiny.
Gene Therapy Situation Gets More Awkward With Another Death In Astellas XLMTM Study
Patient Received Lower Dose Of AT132 After Initial Hold Lifted
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.
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