---
schema_version: "secwatch.filing_event.v1"
accession: "0001404644-24-000049"
form_type: "8-K"
ticker: "NGNE"
cik: "0001404644"
company_name: "Neurogene Inc."
filed_at: "2024-05-07T23:59:59+00:00"
generated_at: "2026-06-02T17:21:29.633519+00:00"
event_type: "other_material"
sentiment: "positive"
materiality_score: 0.65
calibrated_materiality_score: 0.65
confidence: "high"
source: SEC EDGAR
---

# Neurogene's NGN-401 gene therapy well tolerated in first three Rett syndrome patients; no overexpression toxicity

## Summary
- All three pediatric patients (ages 4-7) treated with single ICV dose show mild, transient AEs; no serious adverse events.
- No signs of MeCP2 overexpression toxicity in any patient, including one with a mild variant predicted to retain residual MeCP2 expression.
- Follow-up ranges from ~3 to ~9 months; data cut-off April 19, 2024.
- Interim efficacy data from Cohort 1 expected in Q4 2024; additional data including Cohort 2 in H2 2025.
- NGN-401 uses EXACT transgene regulation technology to limit overexpression risk.

## SEC filing metadata
- accession: 0001404644-24-000049
- form_type: 8-K
- ticker: NGNE
- cik: 0001404644
- company_name: Neurogene Inc.
- filed_at: 2024-05-07T23:59:59+00:00
- event_type: other_material
- sentiment: positive
- materiality_score: 0.65
- calibrated_materiality_score: 0.65
- confidence: high
- sec_items: 7.01, 9.01
- EDGAR index: https://www.sec.gov/Archives/edgar/data/1404644/000140464424000049/0001404644-24-000049-index.htm
- EDGAR primary document: https://www.sec.gov/Archives/edgar/data/1404644/000140464424000049/ngne-20240507.htm

## Machine-readable alternates
- HTML: https://secwatch.observer/filing/0001404644-24-000049
- JSON: https://secwatch.observer/filing/0001404644-24-000049.json
- Plain text: https://secwatch.observer/filing/0001404644-24-000049.txt

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