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Neurogene Lowers Dosage in Rett Syndrome Gene Therapy Trial

4/3/2025

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Summary

Story summary

Neurogene has adjusted its Phase 1/2 trial for NGN-401 gene therapy aimed at girls with Rett syndrome, opting for a lower treatment dose for all participants following a severe immune response in a high-dose patient. The trial now welcomes girls aged 11 and older, with promising interim results anticipated by the end of the year.

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Story summary

Neurogene has adjusted its Phase 1/2 trial for NGN-401 gene therapy aimed at girls with Rett syndrome, opting for a lower treatment dose for all participants following a severe immune response in a high-dose patient. The trial now welcomes girls aged 11 and older, with promising interim results anticipated by the end of the year.