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Neurogene Presents Favorable Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome during ASGCT Annual Meeting
NGN-401 has been generally well-tolerated by first three patients dosed, with three to nine months of follow-up No signs or symptoms of overexpression
About this update from Neurogene Inc.
[{"type":"text","content":"\nNGN-401 has been generally well-tolerated by first three patients dosed, with three to nine months of follow-up\n\n\nNo signs or symptoms of overexpression toxicity, including in one patient with a mild variant predicted to result in residual MeCP2 expression\n\n\nNeurogene remains on track to provide interim efficacy data from the trial in 4Q:24\n\n\n NEW YORK--(BUSINESS WIRE)--\nNeurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome, which showed that NGN-401 was generally well-tolerated by all three patients dosed, with follow-up of approximately nine, six and three months post-dosing. These data were presented during the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.\n\n\n“We designed NGN-401 to overcome the limitations of conventional gene therapy for Rett syndrome by incorporating our EXACTTM transgene regulation technology, which we believe provides tolerable and therapeutic levels of protein expression to the key areas of the brain and nervous system that drive disease,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “The NGN-401 data presented at ASGCT demonstrate a favorable tolerability profile in the first three pediatric patients, including one with a mild variant predicted to result in residual MeCP2 expression, with no signs or symptoms of overexpression-related toxicity reported in any patient. We remain on track to share interim efficacy data from the first cohort of the trial in the fourth quarter of 2024.”\n\n\nThe Phase 1/2 open-label trial is evaluating the safety, tolerability, and preliminary efficacy of two dose levels of NGN-401 delivered via one-time intracerebroventricular (ICV) infusion. Enrollment of female patients ages 4-10 years old with classic Rett Syndrome and a Clinical Global Impression-Severity (CGI-S) score of 4-6 is ongoing in low-dose Cohort 1 and high-dose Cohort 2.\n\n\nThe baseline demographics of the first three patients who received NGN-401 in Cohort 1 (1E15 vector genomes) include:\n\n\n\n\n\n\n\n\n\n\n\nPatient 1\n\n\n\n\n\n\nPatient 2\n\n\n\n\n\n\nPatient 3\n\n\n\n\n\n\n\n\nAge at Dosing\n...