A participant in Neurogene’s clinical trial for Rett syndrome gene therapy has died after severe complications from a high dose. The FDA has allowed the study to continue with the low dose, NGN-401. This therapy delivers a functioning MECP2 gene to brain cells using an adeno-associated virus, which can trigger immune responses. The patient complication was consistent with known risks of high doses. Neurogene’s participation in the FDA’s START program allowed for swift safety data review and continuation with the low dose. The company plans to update the clinical trial protocol and provide additional data in 2025.
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Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose
