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- NGN-401 / Neurogene
Trial completion date, Trial primary completion date, Gene therapy: A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children with Rett Syndrome (clinicaltrials.gov) - Nov 30, 2023
P1/2, N=5, Recruiting, Sponsor: Neurogene Inc.
- NGN-401 / Neurogene
Enrollment open, Gene therapy: A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children with Rett Syndrome (clinicaltrials.gov) - Jun 15, 2023
P1/2, N=5, Recruiting, Sponsor: Neurogene Inc.
Not yet recruiting --> Recruiting
- NGN-401 / Neurogene
New P1/2 trial, Gene therapy: A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children with Rett Syndrome (clinicaltrials.gov) - Jun 12, 2023
P1/2, N=5, Not yet recruiting, Sponsor: Neurogene Inc.
- NGN-401 / Neurogene
Preclinical Safety Assessment of NGN-401, a Clinical-Stage Gene Therapy Product for Rett Syndrome (Petree Hall D) - May 3, 2023 - Abstract #ASGCT2023ASGCT_2266;
Not yet recruiting --> Recruiting Overall, the totality of data generated in multiple preclinical models demonstrated that NGN-401 allowed for MeCP2 expression that provided therapeutic benefit while mitigating the risk of overexpression toxicity, supporting FDA clearance to initiate a first-in-human pediatric clinical study.