Neurogene’s NGN-401 Shows Promise for Rett Syndrome

Neurogene (NGNE) has released an update.

Neurogene Inc. has achieved significant regulatory milestones with its NGN-401 gene therapy for Rett syndrome, including an RMAT designation and selection for the FDA’s START Program, indicating potential to meet unmet medical needs. The company has also reported progress in its clinical trials, with favorable safety profiles observed and interim efficacy data expected in the fourth quarter.

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