Gene Therapy Situation Gets More Awkward With Another Death In Astellas XLMTM Study

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.

There has been a spate of serious adverse events, deaths and clinical holds in AAV vector-based gene therapy trials, which prompted the US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee to hold a meeting on 2-3 September on how to address the growing safety concerns. (Also see "BioMarin Trial Halt Raises Another Safety Flag On AAV Gene Therapy" - Scrip, 6 September, 2021