Second death in Audentes gene therapy trial may highlight need for more careful dosing

The report of a second patient death in a trial of an AAV8-based gene therapy from Audentes and Astellas renewed decades-old concerns over the modality’s safety, but the adverse events may reflect a need for more careful dosing rather than a systemic safety issue.

FDA placed a clinical hold on the Phase I/II ASPIRO trial of AT132 in X-linked myotubular myopathy (XLMTM) after the second of three patients who received a high dose of the liver-targeted gene therapy passed away following progressive liver dysfunction...