What the latest patient death means for gene therapy development

The fourth death of a patient in Astellas’ gene therapy trial for X-linked myotubular myopathy is not likely to have the same broad derailing effect across gene therapies as earlier setbacks. But it is another huge blow for the XLMTM patient community, and for the gene therapy, that will deepen modality-wide concerns about toxicity — in this case, hepatotoxicity — of AAV vectors.

On Monday, Astellas Pharma Inc. (Tokyo:4503) said that a patient treated with the lower dose of AT132 (1.3x10^14 vg/kg) in the Phase I/II ASPIRO study had died. The company first reported the patient had abnormal liver function tests on Sept. 1. Details on the cause of death were not disclosed. ...