Neurogene Inc. has reported positive initial safety and tolerability data from its ongoing Phase 1/2 gene therapy trial for Rett syndrome. The treatment, NGN-401, was well-tolerated in the first three patients dosed, with follow-up periods ranging from three to nine months.

NGN-401 incorporates Neurogene’s EXACTTM technology, aiming to provide therapeutic levels of protein expression without overexpression toxicity. Despite one patient having a mild variant predicted to result in residual MeCP2 expression, no signs or symptoms of overexpression-related toxicity were observed in any patient.

The Phase 1/2 trial is evaluating the safety and preliminary efficacy of two dose levels of NGN-401 delivered via a single intracerebroventricular infusion in female patients aged 4-10 years with classic Rett syndrome. All adverse events related to NGN-401 have been mild and transient, with no serious or treatment-emergent adverse events reported.

Rachel McMinn, PhD, Founder and CEO of Neurogene, indicated that these data demonstrate a favorable tolerability profile in the first three pediatric patients, including one with a mild variant predicted to result in residual MeCP2 expression, and that they anticipate sharing interim efficacy data for the first cohort in the fourth quarter of 2024.

The promising safety and tolerability data support the potential of NGN-401 as a treatment for Rett syndrome, addressing the urgent need for therapies that target the underlying cause of the disease and improve the quality of life for patients and their families.

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Ferry Darma
Ferry Darma is Director of Media Relations at The Clinical Trial Vanguard. Ferry, a computer data scientist, focuses on the latest clinical trial industry news and trends.