Neurogene : IRSF 2025 Oral Presentation

Published: 2025-06-11 11:43:08 ET NGNE

Published on 06/11/2025 at 11:43

Complementary Recognition Sites

NGN-401's intracerebroventricular (ICV) route of delivery has been shown in preclinical models to have the broadest targeting of brain and nervous system regions underlying Rett syndrome pathophysiology.

An estimated 30,000 ICV procedures are performed by neurosurgeons annually in the U.S. and require minimal downtime/recovery.

NGN-401 includes the full-length human MECP2 gene, which creates a fully functioning MeCP2 protein.

NGN-401: Positioned to be Best-in-Class Gene Therapy for Rett Syndrome

NGN-401 Phase 1/2 Clinical Trial Design in Females with Rett Syndrome

Trial Design

Key Eligibility Criteria

N=3

2 Participants Dosed

Females with Classic Rett syndrome in post regression stage of illness

Clinical diagnosis and genetic confirmation of pathogenic MECP2 mutation

Pediatric: 4-10 years old; Adolescent/Adult: 11+ years old

Key Efficacy Assessments

Clinical Global Impression-Severity (CGI-S) score of 4-6

Dosing Complete

Clinician Global Impression-Improvement (CGI-I)

N=8

Clinician Global Impression-Severity with Rett syndrome-specific anchors (CGI-S)

Rett Syndrome Behavior Questionnaire (RSBQ)

Autonomic function

Improvements in Clinician and Caregiver Assessments with 23 Skills Acquired Across 4 Participants

CGI-I

CGI-S Total Score

RSBQ

Improved?

How many points?*

Improved?

How many points?

Improved?

How many points?

(% Change)

Pt:1

15 mos. post-NGN-401

2 pts.

10 pts.

(-28%)

Pt:2

12 mos. post-NGN-401

2 pts.

1 pt.

32 pts.

(-52%)

Pt:3

9 mos. post-NGN-401

2 pts.

5 pts.

(-29%)

Pt:4

3 mos. post-NGN-401

2 pts.

8 pts.

(-28%)

Gain of Skills, Developmental Milestones and Symptom Improvement in RTT Clinical Domains

Hand Function

Gross Motor

Communication

Autonomic

Attentive-ness

Consistent Improvement Across Key Rett Syndrome Scales, Bolstered by Functional Improvements in Core Clinical Domains

As of data cut-off date of 17 October 2024

Disclaimer

Neurogene Inc. published this content on June 11, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on June 11, 2025 at 15:42 UTC.