NGNE Stock — Neurogene Inc.: Cash, EV, Pipeline

What they do

We are a clinical-stage biotechnology company committed to overcoming these limitations and turning today’s complex devastating neurological diseases into treatable conditions. We are building a robust and differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet need not otherwise addressable by conventional gene therapy.

Source: 10-K FY2025 via SEC EDGAR

Share price

$25.31

Market cap

$506.5M

Cash

$225.4M

Enterprise value

$281.1M

Balance sheet

Cash	$225.4M
Debt	$0.0M
Net cash	$225.4M
Enterprise value	$281.1M
Basic shares	20.0M shares
Fully diluted shares	31.5M shares
Fully diluted market cap	$797.8M
Cash per share	$11.26
Quarterly burn	$23.8M
Cash runway	9.5 quarters

Cash figures as of 2026-06-30. Database snapshot: 2026-11-07.

Share structure

Common

20.0M

Pre-funded warrants

7.5M

Options

3.0M

RSUs

0.5M

Warrants

0.0M

Converts

0.0M

Conv preferred

0.0M

ESPP reserve

0.5M

FDSO

31.5M

Cap table as of 2026-06-30.

Price history

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Next catalyst

NGN-401 · Est. Sep 30, 2027 · half midpoint · guided in 8 mo

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
GUIDEDEst. Sep 30, 2027 in 8 mo half midpoint	NGN-401 Embolden registrational trial in Rett syndrome	Not disclosed	Rett syndrome	2H 2027
Jan 12 - Feb 23 in 39 mo	NGN-401	Phase 3	Rett Syndrome	—

Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.

Full readout calendar →

Pipeline assets

NGN-401

Phase 3 · Active Modality: Gene therapy with EXACT transgene regulation technology

Target MECP2

Partnerships University of Edinburgh: exclusive worldwide license to EXACT technology and optimized MECP2 cassettes (Mar 2022); up to $5.3M regulatory milestones ($0.3M paid for Phase 1/2), up to $25M sales milestones, low single-digit royalties

Indications

Rett syndrome (females) (Stage not disclosed)

Status note First clinical-stage program using EXACT technology. Completed dosing in Phase 1/2 trial for Rett syndrome and completed dosing in the registrational Embolden study (single-arm, baseline-controlled, open-label in females with Rett syndrome), designed to support a potential BLA submission.

Per-asset detail extracted from the 10-Q filed 2026-08-11. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

Pipeline

Lead drug	NGN-401
Lead indication	Rett Syndrome
Lead phase	Phase 3
Active trials	2

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT05898620	Phase 3	Active, not recruiting	Sponsor	NGNE	—	Rett Syndrome	2H 2027 Est. Sep 30, 2027 · half midpoint
NCT05228145	Phase 2	Active, not recruiting	Sponsor	NGNE	—	Neuronal Ceroid Lipofuscinosis CLN5	—

Sponsor-matched active trials from ClinicalTrials.gov (2 as sponsor, 0 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.

View all 2 active trials for Neurogene Inc. on ClinicalTrials.gov

Specialist fund holders

Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.

Fund	Shares	Q/Q change	% of portfolio	Filing
Casdin Capital	1,545,361	+125,000	2.43%	2026-08-14
RTW Investments	1,362,456	no change	0.37%	2026-08-14
EcoR1 Capital	1,271,342	no change	1.98%	2026-08-14
Baker Bros. Advisors	991,691	no change	0.16%	2026-08-14
Great Point Partners	352,180	-147,820	2.61%	2026-08-14
DAFNA Capital	89,032	+32,397	0.53%	2026-08-14
ADAR1 Capital Management	7,800	+7,800	0.01%	2026-08-14

Specialist-fund positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.

Latest news

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NGNE — Neurogene Inc. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Upcoming catalysts

Pipeline assets

Pipeline

Active clinical trials

Specialist fund holders

Latest news

NGNE — Neurogene Inc.