uniQure N.V.
QURE evidence brief
What we can confirm
- BioSniper Score:
- 2.7 / 10
- Market Cap:
- $3.19B
- Cash Runway:
- 11.0 mo
What it means
What to watch
Prepared research question
What matters most for QURE—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · QURE
uniQure N.V. is a gene therapy company advancing a pipeline of clinical-stage candidates for severe neurological and rare diseases, with a lead program in Huntington's disease and a cash position projected to fund operations into the second half of 2029 [sec_mda · as of 2026-05-05].
Key risk: Clinical trial setbacks
BioSniper Score
Based on 3 of 6 signals
Limited data coverage
Financial data as of Mar 31, 2026.
Tracking 4 clinical trials for uniQure N.V..
uniQure N.V. is a gene therapy company advancing a pipeline of clinical-stage candidates for severe neurological and rare diseases, with a lead program in Huntington's disease and a cash position projected to fund operations into the second half of 2029 [sec_mda · as of 2026-05-05].
- Cash and cash equivalents, restricted cash and investment securities totaled $588.1 million as of 2026-05-05, projected to fund operations into the second half of 2029 [sec_mda · as of 2026-05-05].
- Lead candidate AMT-130 (ifezuntirgene inilparvovec) for Huntington's disease reported 36-month data from all high-dose patients and 48-month data from the first 12 high-dose patients as of September 2026 [news · published 2026-09-29].
- AMT-260 for refractory mesial temporal lobe epilepsy showed initial six-month follow-up data with no serious adverse events observed in the first low-dose cohort [news · published 2026-06-19].
- Development of AMT-162 for SOD1-ALS was discontinued after a dose-limiting toxicity-related serious adverse event was observed in one patient [sec · filed 2026-04-27].
- Net cash generated from financing activities in the six months ended June 30, 2026 was $249.7 million, compared to $80.7 million in the same period in 2025 [sec_mda · as of 2026-07-29].
What works
- Strong cash position: $588.1 million in cash, restricted cash, and investment securities as of 2026-05-05, with projected runway into the second half of 2029 [sec_mda · as of 2026-05-05].
- Multiple clinical-stage candidates in Phase 1/2 trials (AMT-130, AMT-191, AMT-260) targeting large unmet neurological needs [catalyst].
- AMT-130 has generated extended follow-up data (up to 48 months) in Huntington's disease, supporting continued late-stage development planning [news · published 2026-09-29].
- AMT-260 early data showed no serious adverse events and early biological signals of potential therapeutic activity in the first cohort [news · published 2026-06-19].
What to weigh
- AMT-162 for SOD1-ALS was discontinued after a dose-limiting toxicity-related serious adverse event, highlighting clinical risk in the pipeline [sec · filed 2026-04-27].
- Net income was -$53.5M as of 2026-03-31, reflecting ongoing operating losses typical of a development-stage biotech [latest reported financials].
- The company's future capital requirements depend on the timing and design of potential Phase III trials for AMT-130, AMT-191, and AMT-260, creating uncertainty in expenditure forecasts [sec_mda · as of 2026-05-05].
- Total revenue was only $3.6M as of 2026-03-31, indicating no approved product revenue and reliance on financing activities for funding [latest reported financials].
From the filings
Quoted directly from source documents.
Cash runway into second half of 2029
“cash and cash equivalents, restricted cash and investment securities of $588.1 million. We believe our cash and cash equivalents, restricted cash, and investment securities will be sufficient to fund our projected operating expenses into the second half of 2029.”
SEC MD&A · May 5, 2026
Discontinuation of AMT-162 due to SAE
“we have decided to discontinue development of AMT-162. We will continue to collect safety data from the five patients dosed in EPISOD1, consistent with applicable safety and regulatory requirements.”
SEC filings · April 27, 2026
AMT-260 early data with no serious adverse events
“AMT-260 was generally well-tolerated with no serious adverse events observed to date, with early biological signals of potential therapeutic activity”
News · June 19, 2026
AMT-130 48-month data reported
“uniQure (NASDAQ:QURE) reported updated Phase I/II data for its investigational Huntington’s disease gene therapy, ifezuntirgene inilparvovec, or AMT-130, including 36-month results from all high-dose patients and 48-month results from the first 12 high-dose patients to reach that milestone.”
News · September 29, 2026
Catalyst timeline
- 2019-09-06 · Phase 1/2 start for AMT-130 (Huntington's disease) — Randomized, double-blind, sham-controlled study of striatally-administered rAAV5-miHTT in early manifest Huntington's disease.
- 2024-06-05 · Phase 1/2 start for AMT-191 (Fabry disease) — Single-dose, dose-ranging study of intravenous AAV5-GLA in adult males with classic Fabry disease.
- 2024-06-12 · Phase 1/2a start for AMT-260 (refractory mTLE) — First-in-human study of AMT-260 administered via MRI-guided convection in adults with unilateral refractory mesial temporal lobe epilepsy.
Risks & what to watch (3)›
Key risks
- Clinical trial setbacks — AMT-162 discontinued after dose-limiting toxicity SAE; similar risks exist for other pipeline candidates [sec · filed 2026-04-27].
- No approved product revenue — Total revenue of $3.6M as of 2026-03-31, with net loss of $53.5M, indicating reliance on financing [latest reported financials].
- Phase III cost uncertainty — Future expenditure depends on timing and design of Phase III trials for AMT-130, AMT-191, and AMT-260 [sec_mda · as of 2026-05-05].
What to watch
- Initiation and design of any Phase III trials for AMT-130, AMT-191, and AMT-260, which will materially affect cash burn rate [sec_mda · as of 2026-05-05].
- Further safety and efficacy data from the ongoing Phase I/IIa trial of AMT-260 in refractory mesial temporal lobe epilepsy [news · published 2026-06-19].
- Regulatory interactions and potential BLA filing for AMT-130 following the September 2026 update [news · published 2026-09-29].
- Cash runway updates as the company progresses toward the second half of 2029 projected funding horizon [sec_mda · as of 2026-05-05].
Sources: Catalysts · News · Pipeline · SEC filings · SEC MD&A · Generated October 13, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 3 of 6 signals · derived from public data
Pipeline inputs are unavailable for this score. It uses only the available signals shown below.
11.0 months
D/E 4.21 · 0% dilution
No tracked pipeline phase
No tracked pipeline candidates
No upcoming catalysts tracked
0 buys · 38 sells (180d)
Runway
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
211
Total value held
$961.1M
| Holder | Shares | Value |
|---|---|---|
| avoro capital advisors llc | 5,959,595 | $97.4M |
| aberdeen group plc | 5,306,386 | $86.8M |
| two sigma investments, lp | 2,675,871 | $43.8M |
| rtw investments, lp | 2,586,356 | $42.3M |
| jpmorgan chase & co | 2,837,466 | $41.8M |
| state street corp | 2,260,512 | $37.0M |
| orbimed advisors llc | 2,136,149 | $34.9M |
| fmr llc | 1,743,808 | $28.5M |
| blackrock, inc. | 1,733,458 | $28.3M |
| mpm bioimpact llc | 1,369,231 | $22.4M |
Showing top 10 of 211 institutional holders of uniQure N.V.. Open the full 13F history after sign-in.
Short Interest
as of Apr 30, 2026- % of float
- 18.0%
- Days to cover
- 6.2
- Shares short
- 11,097,615
- 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 →
QURE short interestGovernance
Frequently asked questions
What is the AI investment thesis for uniQure N.V.?
uniQure N.V. is a gene therapy company advancing a pipeline of clinical-stage candidates for severe neurological and rare diseases, with a lead program in Huntington's disease and a cash position projected to fund operations into the second half of 2029 [sec_mda · as of 2026-05-05].
Who is the CEO of uniQure N.V.?
Mr. Matthew Craig Kapusta CPA is the chief executive officer of uniQure N.V. (QURE).
Where is uniQure N.V. headquartered?
uniQure N.V. (QURE) is headquartered in Amsterdam, Netherlands.
What therapeutic areas does uniQure N.V. focus on?
uniQure N.V. develops therapies across Oncology, Rare Disease, Gene Therapy, 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.