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Neurogene Inc.

Clinical trials sponsored by Neurogene Inc., explained in plain language.

Neurogene Inc. NGNE NASDAQ

A clinical-stage gene therapy company focused on rare neurological diseases.

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Not investment advice. Company information is shown for context only. Clinical trials frequently fail.

Not yet finished but already full! 1

Gene therapy trial aims to help girls with rett syndrome gain new skills

Disease control Ongoing

This study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whe…

Phase: PHASE3 • Sponsor: Neurogene Inc. • Aim: Disease control

Last updated Jun 27, 2026 13:06 UTC