Close this search box.

Neurogene reports initial data from Rett syndrome gene therapy trial

Neurogene has shared initial safety and tolerability results from its ongoing Phase I/II clinical trial of NGN-401, a gene therapy candidate for Rett syndrome.

The open-label study is focused on assessing the safety, tolerability, and initial efficacy of NGN-401, administered via a one-time intracerebroventricular (ICV) infusion across two dosage cohorts.

The trial is currently enrolling female patients aged four to ten years with classic Rett Syndrome.

It also assesses the Clinical Global Impression-Severity (CGI-S) score, ranging from four to six, in both the low-dose Cohort 1 and the high-dose Cohort 2.

According to the initial data, NGN-401 was found to be well-tolerated by all three patients dosed, with follow-up periods of nearly nine, six, and three months post-dosing.

The reported adverse events (AEs) linked to NGN-401 were mild, or Grade 1, and either transient or resolving in nature.

Access the most comprehensive Company Profiles
on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free

Your download email will arrive shortly

We are confident about the
quality of our Company Profiles. However, we want you to make the most
decision for your business, so we offer a free sample that you can download by
submitting the below form

By GlobalData



Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The majority of AEs were found to be in line with known potential risks associated with adeno-associated virus (AAV) vectors, including asymptomatic laboratory value changes.

Importantly, no reports of signs or symptoms indicative of MeCP2 overexpression toxicity were reported.

Furthermore, there have been no treatment-emergent or ICV procedure-related serious adverse events (SAEs) observed in the trial.

Neurogene plans to publish interim clinical data, including efficacy outcomes, from Cohort 1 in the fourth quarter of this year.

Additional interim data, including results from Cohort 2, is anticipated in the second half of 2025.

The company presented the data during the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

Neurogene founder and CEO Rachel McMinn said: “We designed NGN-401 to overcome the limitations of conventional gene therapy for Rett syndrome by incorporating our EXACT 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.

“The NGN-401 data presented at ASGCT demonstrate a favourable 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.”

In December 2023, Neurogene dosed the first two female paediatric subjects in Phase I/II trial of NGN-401 to treat Rett Syndrome.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

<!– –>