QURE Stock — uniQure N.V.: Cash, EV, Pipeline

What they do

We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, refractory mesial temporal lobe epilepsy (“MTLE”), Fabry disease, and amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”).

Source: 10-K FY2025 via SEC EDGAR

Share price

$21.70

Market cap

$1,522.0M

Cash

$98.4M

Enterprise value

$1,473.9M

Balance sheet

Cash	$98.4M
Debt	$50.3M
Net cash	$48.1M
Enterprise value	$1,473.9M
Basic shares	58.9M shares
Fully diluted shares	66.0M shares
Fully diluted market cap	$1,433.3M
Cash per share	$1.67
Quarterly burn	$19.1M
Cash runway	42.4 quarters

Cash figures as of 2026-09-30. Database snapshot: 2026-11-07.

Share structure

Common

58.9M

Pre-funded warrants

0.0M

Options

5.0M

RSUs

2.1M

Warrants

0.0M

Converts

0.0M

Conv preferred

0.0M

ESPP reserve

0.1M

FDSO

66.0M

Cap table as of 2026-07-24.

Price history

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Next catalyst

AMT-191 · Jan 12 - Feb 23 in 14 mo · Phase 2 · Fabry Disease

Recently reported

AMT-130 Mixed · 2026-09-29

Source release →

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
Jan 12 - Feb 23 in 14 mo	AMT-191	Phase 2	Fabry Disease	—

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

AMT-130

Phase 1 · Active Modality: AAV gene therapy (miQURE miRNA gene-silencing)

Target huntingtin (HTT)

Indications

Huntington's disease (Phase 1)

Catalysts

— Q3 2026

Status note Phase I/II trials in the US and Europe; FDA aligned June 2026 that a BLA under the accelerated approval pathway is reasonable; confirmatory study design pending alignment.

AMT-260

Phase 1 · Active Modality: AAV gene therapy

Target GRIK2

Indications

refractory mesial temporal lobe epilepsy (MTLE) (Phase 1)

Status note GenTLE Phase I/IIa trial ongoing; first cohort enrollment completed 2025.

AMT-191

Phase 1 · Active Modality: AAV gene therapy

Target GLA (from 10-K fallback)

Indications

Fabry disease (Phase 1)

Status note Phase I/II trial ongoing; GLA target from 10-K fallback (target/modality only).

AMT-162

Phase 1 · Active Modality: AAVrh10 gene therapy (miRNA)

Aliases APB-102

Target SOD1

Partnerships Apic Bio (global license)

Indications

SOD1-ALS (Phase 1)

Status note EPISOD1 Phase I/II enrollment voluntarily paused September 2025 upon IDMC recommendation after a dose-limiting toxicity; data collection from the 5 dosed patients continues; no discontinuation disclosed.

HEMGENIX

Approved · Active Modality: AAV gene therapy

Target factor IX

Partnerships CSL Behring (global commercialization rights; uniQure receives royalties and manufacturing income)

Indications

hemophilia B (Approved)

Status note Economic asset via the global CSL Behring license; royalty and manufacturing income.

Per-asset detail extracted from the 10-Q filed 2026-07-29. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

No lead program on file for this company.

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT06100276	Phase 2	Active, not recruiting	Sponsor	QURE	—	Amyotrophic Lateral Sclerosis	—
NCT06063850	Phase 2	Recruiting	Sponsor	QURE	—	Mesial Temporal Lobe Epilepsy	—
NCT06270316	Phase 2	Recruiting	Sponsor	QURE	—	Fabry Disease	—
NCT05243017	Phase 2	Active, not recruiting	Sponsor	QURE	—	Huntington Disease	September 2026
NCT04120493	Phase 2	Active, not recruiting	Sponsor	QURE	—	Huntington's Disease	September 2026

Sponsor-matched active trials from ClinicalTrials.gov (5 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 5 active trials for UniQure Biopharma B.V. 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
Avoro Capital Advisors	6,727,777	+768,182	2.78%	2026-08-14
RTW Investments	3,941,572	+1,355,216	1.55%	2026-08-14
OrbiMed Advisors	1,807,486	-328,663	1.58%	2026-08-14
Boxer Capital	1,503,300	+841,300	5.21%	2026-08-14
ADAR1 Capital Management	324,672	+248,466	0.70%	2026-08-14
Braidwell LP	301,652	+301,652	0.51%	2026-08-14
Perceptive Advisors	168,000	+168,000	0.12%	2026-08-14
Casdin Capital	150,000	+150,000	0.35%	2026-08-14
Logos Global Management	30,000	+30,000	0.06%	2026-08-14
Ikarian Capital	0	-91,060	0.00%	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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QURE — uniQure N.V. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Recently reported

Upcoming catalysts

Pipeline assets

Active clinical trials

Specialist fund holders

Latest news

QURE — uniQure N.V.