---
schema_version: "secwatch.filing_event.v1"
accession: "0001404644-24-000091"
form_type: "8-K"
ticker: "NGNE"
cik: "0001404644"
company_name: "Neurogene Inc."
filed_at: "2024-11-12T23:59:59+00:00"
generated_at: "2026-05-30T01:42:00.301495+00:00"
event_type: "other_material"
sentiment: "positive"
materiality_score: 0.9
calibrated_materiality_score: 0.9
confidence: "high"
source: SEC EDGAR
---

# Neurogene reports positive interim efficacy data from low-dose cohort of NGN-401 gene therapy for Rett syndrome

## Summary
- All 4 low-dose participants achieved a 2-point improvement on clinician-rated CGI-I scale from baseline.
- Caregiver-completed RSBQ scores improved 28–52% from baseline; participants acquired developmental milestones not seen in natural history.
- Low-dose NGN-401 well-tolerated with no treatment-related SAEs; emerging SAE noted in a third high-dose participant.
- Initiated adolescent/adult cohort (age ≥16) and gained FDA alignment on potency assay and manufacturing scale-up for registrational trial.
- Company discontinued CLN5 Batten disease program after RMAT application denial; evaluating options.

## SEC filing metadata
- accession: 0001404644-24-000091
- form_type: 8-K
- ticker: NGNE
- cik: 0001404644
- company_name: Neurogene Inc.
- filed_at: 2024-11-12T23:59:59+00:00
- event_type: other_material
- sentiment: positive
- materiality_score: 0.9
- calibrated_materiality_score: 0.9
- confidence: high
- sec_items: 7.01, 9.01
- EDGAR index: https://www.sec.gov/Archives/edgar/data/1404644/000140464424000091/0001404644-24-000091-index.htm
- EDGAR primary document: https://www.sec.gov/Archives/edgar/data/1404644/000140464424000091/ngne-20241111.htm

## Machine-readable alternates
- HTML: https://secwatch.observer/filing/0001404644-24-000091
- JSON: https://secwatch.observer/filing/0001404644-24-000091.json
- Plain text: https://secwatch.observer/filing/0001404644-24-000091.txt

This AI-assisted summary is a reading aid. Review the linked SEC EDGAR filing before relying on any specific claim.