Astellas Pauses XLMTM Gene Therapy Trial After Another Safety Scare

 
• By Alaric DeArment
  
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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.

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Less than a year after the US Food and Drug Administration allowed its Phase I/II study of AT132 for X-linked myotubular myopathy (XLMTM) to resume following a months-long clinical hold over adverse events, Astellas Pharma, Inc. has hit the pause button on the gene therapy trial again due to a safety issue. The hold emphasizes the growing concern about the safety of adeno-associated viral (AAV) vector-based gene therapies in general on the eve of an FDA advisory committee on the topic.

The Tokyo-based drug maker said 1 September that it had paused screening and enrollment into the ASPIRO trial after a...

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