On April 22, 2024 Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, reported that initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome will be presented at the American Society for Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting (Press release, Neurogene, APR 22, 2024, View Source [SID1234642198]). The data show that the NGN-401 gene therapy candidate has been generally well-tolerated, and there have been no treatment-emergent or procedure-related serious adverse events or signs of MeCP2 overexpression-related toxicity observed in any patient, including a patient with a mild variant predicted to result in residual MeCP2 function.

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"We look forward to sharing the initial, favorable safety data of NGN-401, which is designed to use our differentiated EXACT transgene regulation technology and deliver a full-length MECP2 gene to key areas of the brain underlying the pathophysiology of Rett syndrome," said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "Rett syndrome is a complex neurological disorder with a narrow therapeutic window, and conventional gene therapy approaches have been unable to provide therapeutic protein levels without detrimental overexpression. Therefore, we believe it is important to share this safety update at the ASGCT (Free ASGCT Whitepaper) Meeting, in advance of our expected fourth quarter 2024 interim efficacy read-out, as we have multiple months of data from three patients showing NGN-401 has been generally well-tolerated."

Neurogene’s ASGCT (Free ASGCT Whitepaper) Meeting Poster Presentation Details

•Title: Preliminary Safety Results from the Ph1/2 study of NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome
•Date: May 9, 2024
•Time: 12:00 p.m. ET
•Location: Exhibit Hall
The abstract can be accessed at this link View Source