---
schema_version: "secwatch.filing_event.v1"
accession: "0001404644-25-000070"
form_type: "8-K"
ticker: "NGNE"
cik: "0001404644"
company_name: "Neurogene Inc."
filed_at: "2025-06-30T23:59:59+00:00"
generated_at: "2026-05-18T12:28:46.989362+00:00"
event_type: "other_material"
sentiment: "positive"
materiality_score: 0.8
calibrated_materiality_score: 0.8
confidence: "high"
source: SEC EDGAR
---

# Neurogene announces FDA agreement on registrational trial design for NGN-401 gene therapy in Rett syndrome

## Summary
- FDA agreed on single-arm, open-label, baseline-controlled design; N=18 proposed; primary endpoint: CGI-I improvement plus gain of developmental milestone.
- Phase 1/2 fully enrolled; 5 participants dosed in 1H2025; no HLH/hyperinflammatory syndrome observed at 1E15 vg dose.
- First four participants achieved 23 developmental milestones across hand function, communication, and ambulation by 3–15 months.
- Cash, cash equivalents & investments of $292.6M as of March 31, 2025; runway extended into early 2028.
- Updated efficacy and safety data expected in second half of 2025.

## SEC filing metadata
- accession: 0001404644-25-000070
- form_type: 8-K
- ticker: NGNE
- cik: 0001404644
- company_name: Neurogene Inc.
- filed_at: 2025-06-30T23:59:59+00:00
- event_type: other_material
- sentiment: positive
- materiality_score: 0.8
- calibrated_materiality_score: 0.8
- confidence: high
- sec_items: 7.01, 9.01
- EDGAR index: https://www.sec.gov/Archives/edgar/data/1404644/000140464425000070/0001404644-25-000070-index.htm
- EDGAR primary document: https://www.sec.gov/Archives/edgar/data/1404644/000140464425000070/ngne-20250630.htm

## Machine-readable alternates
- HTML: https://secwatch.observer/filing/0001404644-25-000070
- JSON: https://secwatch.observer/filing/0001404644-25-000070.json
- Plain text: https://secwatch.observer/filing/0001404644-25-000070.txt

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