• FDA clears IND application for gene therapy to treat children with Rett syndrome

    2 days ago - By Healio

    Neurogene Inc. announced the FDA has granted clearance to its investigational new drug application for NGN-401, an adeno-associated virus gene therapy candidate for treating Rett syndrome in female pediatric patients.
    According to a company press release, the FDA clearance allows the initiation of a phase 1/2 study in 2023 to assess safety, efficacy and tolerability of the novel MECP2 gene product. The open-label, single-arm, multicenter clinical trial will evaluate a single dose of NGN-401 delivered via a one-time intracerebroventricular procedure, which achieved safety and efficacy outcomes
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