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Gene Therapies: US Panel To Weigh Strategies For Preventing, Mitigating AAV Vector Toxicities

 
• By Sue Sutter

FDA seeks advice on screening patients potentially at higher risk for liver and other injuries, strategies to implement before or after gene therapy administration to head off or mitigate potential adverse events, and whether upper limits should be set for the total vector genome dose and total capsid dose.

Gene therapy concept
US FDA is seeking expert panel advice on how to make AAV vector gene therapy studies safer. • Source: Alamy

A US Food and Drug Administration advisory committee will discuss potential strategies for mitigating, monitoring and preventing various types of toxicities with adeno-associated virus (AAV) vector-based gene therapies at 2-3 September meeting.

The Cellular, Tissue and Gene Therapies Advisory Committee will consider recommendations on screening patients potentially at higher risk for liver and other injuries, strategies to implement before or after gene therapy administration to head off or mitigate potential adverse

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