First brain-delivered AAV therapy approved by FDA

Drug Discovery News·Feb 2

✦ NabkaNews BriefAuto-summarized from multiple outlets · verify with the source

The FDA has approved the first brain-delivered AAV therapy. However, the outlook for this area of gene therapy research is uncertain, as a child's death has been reported in a related trial, and some outlets indicate that more data is needed for certain therapies. Additionally, various companies are reportedly moving forward with their own gene therapy trials, including those targeting Alzheimer's disease and Hunter syndrome.

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First brain-delivered AAV therapy approved by FDA

Full coverage

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