Neurogene Inc.
NGNE evidence brief
What we can confirm
- BioSniper Score:
- 5.0 / 10
- Market Cap:
- $710.0M
What it means
What to watch
Prepared research question
What matters most for NGNE—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · NGNE
Neurogene Inc. is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases, with its lead program NGN-401 in a registrational trial for Rett syndrome.
Key risk: No approved products or revenue
BioSniper Score
Based on 2 of 6 signals
Limited data coverage
Market Cap
$710.0M
Cash Runway
—
Lead Asset
—
Next Catalyst
—
Financial data as of Jun 30, 2026.
Tracking 2 clinical trials for Neurogene Inc..
Neurogene Inc. is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases, with its lead program NGN-401 in a registrational trial for Rett syndrome.
- Lead program NGN-401 is in a registrational trial (Embolden) for Rett syndrome, with dosing completed as of June 2026 [news · 2026-06-08].
- NGN-401 received Breakthrough Therapy designation from the FDA in February 2026 [sec_mda · as of 2026-08-11].
- Cash, cash equivalents, and short-term investments were approximately $360.2 million as of July 2026, expected to fund operations into Q1 2029 [sec_mda · as of 2026-08-11].
- The company has not generated any product revenue and expects to continue incurring significant operating losses [sec_mda · as of 2026-05-12].
- NGN-401 also holds Orphan and Advanced Therapy Medicinal Product designations from the EMA [sec_mda · as of 2026-08-11].
What works
- Proprietary EXACT transgene regulation platform designed to deliver therapeutic transgene levels while mitigating toxicity in neurological diseases [sec_mda · as of 2026-08-11].
- NGN-401 has FDA Breakthrough Therapy designation and FDA alignment on key registrational trial design elements, supporting an efficient path to market [sec_mda · as of 2026-08-11].
- Cash runway into Q1 2029 provides financial flexibility to advance NGN-401 through clinical development and pre-commercial activities [sec_mda · as of 2026-08-11].
What to weigh
- No approved products or commercial revenue; the company has a history of significant operating losses and negative cash flows [sec_mda · as of 2026-05-12].
- Dependence on raising additional capital; if unsuccessful, the company may need to reduce operations or sell assets [sec_mda · as of 2026-08-11].
- The CLN5 Batten disease program (NGN-101) was discontinued after Phase 1/2 enrollment, highlighting pipeline risk [sec_mda · as of 2026-08-11].
From the filings
Quoted directly from source documents.
NGN-401 received Breakthrough Therapy designation.
“In February 2026, we announced that NGN-401 received Breakthrough Therapy designation”
SEC MD&A · August 11, 2026
Cash runway extends into Q1 2029.
“we believe that our existing cash, cash equivalents and short-term investments should be sufficient to fund our operations into the first quarter of 2029”
SEC MD&A · August 11, 2026
Company has no product revenue and expects continued losses.
“we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from our operations”
SEC MD&A · May 12, 2026
Risks & what to watch (4)›
Key risks
- No approved products or revenue — No commercial revenue; relies on future product sales that may take many years [sec_mda · as of 2026-08-11].
- Need for additional capital — May require further financing; failure could force asset sales or reduced operations [sec_mda · as of 2026-08-11].
- Clinical and regulatory uncertainty — NGN-401 registrational trial results and FDA/EMA decisions are uncertain [sec_mda · as of 2026-08-11].
- Pipeline concentration — Heavy reliance on NGN-401; other programs are early-stage or discontinued [sec_mda · as of 2026-08-11].
What to watch
- Topline data from the Embolden registrational trial expected in H2 2027 [news · 2026-07-02].
- Completion of Process Performance Qualification runs by end of 2026 [sec_mda · as of 2026-08-11].
- Any updates on early-stage EXACT platform programs for other neurological indications [sec_mda · as of 2026-08-11].
Sources: News · SEC MD&A · Generated October 8, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 2 of 6 signals · derived from public data
Pipeline inputs are unavailable for this score. It uses only the available signals shown below.
Cash runway data unavailable
D/E 0.13 · 0% dilution
No tracked pipeline phase
No tracked pipeline candidates
No upcoming catalysts tracked
0 buys · 7 sells (180d)
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
96
Total value held
$342.0M
| Holder | Shares | Value |
|---|---|---|
| samsara biocapital, llc | 1,717,127 | $34.6M |
| redmile group, llc | 1,459,599 | $29.4M |
| casdin capital, llc | 1,420,361 | $28.6M |
| rtw investments, lp | 1,362,456 | $27.5M |
| ecor1 capital, llc | 1,271,342 | $25.6M |
| blackrock, inc. | 1,005,070 | $20.3M |
| baker bros. advisors lp | 991,691 | $20.0M |
| trails edge capital partners, lp | 782,787 | $15.8M |
| balyasny asset management l.p. | 718,389 | $14.5M |
| toronto dominion bank | 681,337 | $13.7M |
Showing top 10 of 96 institutional holders of Neurogene Inc.. Open the full 13F history after sign-in.
Short Interest
as of May 21, 2026- % of float
- 29.8%
- Days to cover
- 22.6
- Shares short
- 3,720,441
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
NGNE short interestGovernance
Frequently asked questions
What is the AI investment thesis for Neurogene Inc.?
Neurogene Inc. is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases, with its lead program NGN-401 in a registrational trial for Rett syndrome.
Who is the CEO of Neurogene Inc.?
Dr. Rachel L. McMinn Ph.D. is the chief executive officer of Neurogene Inc. (NGNE).
Where is Neurogene Inc. headquartered?
Neurogene Inc. (NGNE) is headquartered in New York, NY, United States.
What therapeutic areas does Neurogene Inc. focus on?
Neurogene Inc. develops therapies across Rare Disease, Neurology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.