Neurogene (NGNE)
Builds a single gene therapy for Rett syndrome using a proprietary expression-control platform.
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Develops a single clinical-stage gene therapy, NGN-401, that delivers a regulated copy of the MECP2 gene into the brain to treat Rett syndrome, using proprietary technology meant to avoid the expression toxicity that undermined earlier attempts. A second, earlier-stage program for a related rare disease has been set aside.
Develops NGN-401, a gene therapy that delivers a working copy of the MECP2 gene to treat Rett syndrome. A proprietary regulation platform, EXACT, tunes expression cell by cell, addressing the overexpression toxicity that undermined earlier gene-therapy attempts in the disease.
The program has moved into a registrational trial after earlier dosing showed developmental gains without the safety signal seen with rival approaches, and has collected breakthrough and other expedited designations from regulators in the United States, Europe, and the United Kingdom.
A second, earlier-stage program for a related rare disease has been set aside following a regulatory setback. Manufacturing runs through an in-house facility in Texas rather than outside contractors. Founded in 2018 in New York, the company reached public markets in 2023 through a reverse merger and remains pre-revenue.
Company facts
Target rangeAs of Sep 22, 2026
Latest analyst notes
| Date | Firm | Action | Prior | Current |
|---|---|---|---|---|
| Aug 12, 2026 | H.C. Wainwright | Reiterate | Rating: —Target: $70 | Target: $65 |
| Aug 12, 2026 | Guggenheim | Reiterate | Rating: —Target: $69 | Target: $81 |
| Jun 9, 2026 | H.C. Wainwright | Target | Target: $45 | Target: $70 |
| Jun 3, 2026 | UBS | Target | Target: — | Target: $70 |
| Apr 28, 2026 | Guggenheim | Target | Target: — | Target: $69 |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $225.4M · Total debt $8.3M · Total assets $244.4M · Shareholders' equity $217.1M
- Revenue
- $0
- Operating income
- −$36.7M
- Net income
- −$34.5M
No dividends on record.
| Institutions | ≈ 109.77% | 17.35M shares | |
|---|---|---|---|
| 117 holders · as of Jun 30, 2026 · largest filers plus last quarter's tail | |||
| Insiders | 0.80% | 127.0K shares | |
| 3 holders · as of Jul 7, 2026 | |||
| Adds to | 110.57% | ||
15.80M company shares as of Sep 12, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
Two share counts disagree (20.01M in the company's Sep 22, 2026 report, 15.80M elsewhere); stakes use the report.
This quarter's institutional total failed a plausibility check; shown: the largest filers plus last quarter's remainder.
These groups come from separate filings that can overlap, so they add to 110.57%; nothing is left for Other.
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Date |
|---|---|---|---|
Samsara Biocapital, LLCInstitution | 10.87% | 1.72M | as of Jun 30, 2026 |
Casdin Capital, LLCInstitution | 9.78% | 1.55M | as of Jun 30, 2026 |
Redmile Group, LLCInstitution | 8.94% | 1.41M | as of Jun 30, 2026 |
RTW Investments, LPInstitution | 8.62% | 1.36M | as of Jun 30, 2026 |
Ecor1 Capital, LLCInstitution | 8.05% | 1.27M | as of Jun 30, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Jul 7, 2026 | Christine Mikail Cvijic | director, officer: President and CFO | −4,200 | $36.13 |
| Jul 6, 2026 | Christine Mikail Cvijic | director, officer: President and CFO | −600 | $36.02 |
| Jul 2, 2026 | Christine Mikail Cvijic | director, officer: President and CFO | −4,061 | $34.82 |
| Jul 1, 2026 | Christine Mikail Cvijic | director, officer: President and CFO | −739 | $34.06 |
| May 6, 2026 | Christine Mikail Cvijic | director, officer: President and CFO | −9,600 | $31.25 |
- Taysha Gene Therapies
Intrathecal mini-MECP2 AAV9 gene therapy for Rett, pivotal data sooner
- Acadia Pharmaceuticals
Sole FDA-approved Rett therapy; oral, chronic symptomatic standard of care
- REGENXBIO
Broader AAV9 CNS gene-therapy peer for other rare pediatric neuro diseases
- Ultragenyx Pharmaceutical
Large diversified rare-disease gene-therapy peer with CNS AAV/ASO pipeline
- Anavex Life Sciences
Oral daily pill rivaling one-time AAV gene therapy for Rett syndrome
CompareAfter hours
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Neurogene | $33.60 | +0.06% | $514.7M | — | — | — | — |
| Taysha Gene Therapies | $5.16 | +0.10% | $1.61B | — | — | 293.9 | −100.0% |
| Acadia Pharmaceuticals | $20.61 | −21.95% | $4.52B | 11.9 | 29.5 | 4.0 | +16.4% |
| REGENXBIO | $8.15 | −0.12% | $407.4M | — | — | 2.3 | +405.7% |
| Ultragenyx Pharmaceutical | $15.68 | +0.44% | $1.44B | — | — | 2.0 | +28.5% |
| Anavex Life Sciences | $2.51 | +1.83% | $222.4M | — | — | — | — |
| Median | 11.9 | 29.5 | 3.2 | +22.5% |