Neurogene Announces Expansion of Gene Therapy Clinical Trial

Neurogene Announces Expansion of Gene Therapy Clinical Trial

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Today, Neurogene announced plans to expand and more rapidly enroll patients their Phase 1/2 clinical trial of investigational gene therapy NGN-401. For the full press release, click here.

Key Facts:

  • The study will now expand to include a total of 16 pediatric participants (age 4-10). Cohort 1 (low dose group) will increase from 5 to 8 participants and Cohort 2 (high dose) will enroll an additional eight participants.
  • A 3rd patient in Cohort 1 was dosed early in Q1. NGN-401 has been generally well-tolerated and there have been no treatment-emergent or procedure-related serious adverse events, or signs of overexpression-related toxicity observed in any patient.
  • Neurogene will dose the remaining patients in Cohort 1 in parallel, without a stagger or delay between patients, enabling the anticipated completion of dosing in the second half of 2024.


Thank you to the patients, families, scientists, and researchers participating in this trial. You are giving hope to so many in our community.

Click here to learn more about the study and its recruiting sites, all IRSF-designated Centers of Excellence for Rett syndrome research and care: Texas Children’s Hospital, Boston Children’s Hospital, and Colorado Children’s Hospital.

IRSF is with you every step of the way. For the most up-to-date information on this and all other research and clinical trials in the Rett pipeline, click here. Questions? Email us at [email protected].