Neurogene
Builds a single gene therapy for Rett syndrome using a proprietary expression-control platform.
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Profile
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
Key statistics
Year to date +81.2% · Average volume (30d) 196.3K
Analysts
Ratings
Target range
Latest analyst notes
| Date | Firm | Action | Prior rating | Rating | Prior target | Target |
|---|---|---|---|---|---|---|
| Aug 12, 2026 | H.C. Wainwright | Reiterate | Buy | $70 | $65 | |
| Aug 12, 2026 | Guggenheim | Reiterate | Buy | $69 | $81 | |
| Jun 9, 2026 | H.C. Wainwright | Target | $45 | $70 | ||
| Jun 3, 2026 | UBS | Target | $70 | |||
| Apr 28, 2026 | Guggenheim | Target | $69 |
Earnings
EPS
Revenue
History
| Report date | EPS est | EPS | Surprise | Revenue est | Revenue | Surprise | Next session |
|---|---|---|---|---|---|---|---|
| Aug 11, 2026Q2 FY26 | −$1.42 | −$1.53 | −7.7% | — | — | — | −2.05% |
| May 12, 2026Q1 FY26 | −$1.30 | −$1.39 | −7.2% | — | — | — | +3.42% |
| Mar 24, 2026Q4 FY25 | −$1.24 | −$1.12 | +9.6% | — | — | — | +5.41% |
| Nov 13, 2025Q3 FY25 | −$1.26 | −$0.99 | +21.4% | — | — | — | −14.04% |
Financial highlights
| 12 quarters | Q1 FY25 | Q2 FY25 | Q3 FY25 | Q4 FY25 | Q1 FY26 | Q2 FY26 | |
|---|---|---|---|---|---|---|---|
| Revenue | $0 | $0 | $0 | $0 | $0 | $0 | |
| Gross profit | −$17.8M | −$19.4M | −$17.2M | −$787.0K | −$854.0K | $0 | |
| Operating income | −$25.9M | −$26.1M | −$23.8M | −$27.5M | −$33.3M | −$36.7M | |
| Net income | −$22.6M | −$22.0M | −$21.0M | −$24.7M | −$30.9M | −$34.5M | |
| Diluted EPS | −$1.08 | −$1.05 | −$0.99 | −$1.60 | −$1.39 | −$1.53 | |
| Gross margin | — | — | — | — | — | — | |
| Operating margin | — | — | — | — | — | — |
Fiscal years (4-quarter sums)FY21 $0 · FY22 $0 · FY23 $0 · FY24 $925.0K · FY25 $0
Revenue by product · FY24
Balance sheet, Jun 30, 2026Cash & short-term investments $225.4M · Total debt $8.3M · Total assets $244.4M · Shareholders' equity $217.1M
Dividends
No dividends on record.
Ownership
A 13F quarter can total more than the shares outstanding — sub-advisers file the same block twice, and a filer's own share count can be a quarter stale. No split is drawn from it.
Shares outstanding 15.80M · Float 12.54M · 79.3% free float
Top shareholders13F · 13D/G · Form 4
| Holder | Shares | % outstanding | Form | Filed |
|---|---|---|---|---|
| Samsara Biocapital, LLC | 1.72M | 10.87% | 13F | May 15, 2026 |
| Redmile Group, LLC | 1.46M | 9.24% | 13F | May 15, 2026 |
| Casdin Capital, LLC | 1.42M | 8.99% | 13F | May 15, 2026 |
| RTW Investments, LP | 1.36M | 8.62% | 13F | May 15, 2026 |
| Rachel McMinn · director, officer: Chief Executive Officer | 1.34M | 8.51% | Form 4 | Mar 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 |
Peers
- 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 close
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Neurogene | $36.58 | +0.16% | $578.0M | — | — | — | — |
| Taysha Gene Therapies | $5.87 | +1.73% | $1.85B | — | — | 336.5 | −100.0% |
| Acadia Pharmaceuticals | $29.17 | +0.18% | $4.98B | 13.1 | 32.7 | 4.4 | +16.4% |
| REGENXBIO | $9.65 | −0.21% | $499.4M | — | — | 2.9 | +405.7% |
| Ultragenyx Pharmaceutical | $25.68 | +0.27% | $2.52B | — | — | 3.5 | +28.5% |
| Anavex Life Sciences | $2.99 | +0.14% | $277.1M | — | — | — | — |
| Median | 13.1 | 32.7 | 3.9 | +22.5% |