Neurogene reports updated positive Phase 1/2 data for NGN-401 gene therapy in Rett syndrome
Neurogene released updated Phase 1/2 results for its NGN-401 gene therapy in females with Rett syndrome, reporting 47 developmental milestones gained across 10 participants with follow-up of 12 to 30 months. The company reported no plateau or loss of milestones and no new treatment-related serious adverse events since its October 2025 data cutoff. The filing contains clinical data only, with no financial results or guidance changes, and topline results from the Embolden registrational trial are expected in the second half of 2027.
Key points
- 47 total developmental milestones gained across 10 participants, an average of 4.7 per participant, as of the June 16, 2026 data cutoff
- Follow-up ranged from 12 to 30 months, with no plateau observed and no milestones lost
- No new treatment-related serious adverse events reported since the October 2025 data cutoff; no treatment-related SAEs or DLTs in Embolden as of June 16, 2026
- Embolden registrational trial dosing is complete with 25 participants; topline results expected in 2H 2027
The filing
Written by AI on Nov 9, 2026, 10:08 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on June 29, 2026.
Briefs are written by AI from the filing text and can contain mistakes — open the filing before acting on anything. Impact (1-5) and tone are the AI's reading of the filing for shareholders, not a price forecast. Not investment advice.