uniQure N.V.
QURE evidence brief
What we can confirm
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. (QURE) is a gene therapy company focused on developing treatments for central nervous system and liver diseases, with its lead candidate AMT-130 for Huntington's disease pursuing an accelerated FDA approval pathway.
Key risk: Regulatory approval uncertainty for AMT-130
BioSniper Score
Based on 5 of 6 signals
Financial data as of Mar 31, 2026.
Tracking 4 pipeline candidates · 4 clinical trials for uniQure N.V..
uniQure N.V. (QURE) is a gene therapy company focused on developing treatments for central nervous system and liver diseases, with its lead candidate AMT-130 for Huntington's disease pursuing an accelerated FDA approval pathway.
- Lead candidate AMT-130 for Huntington's disease has received FDA Breakthrough Therapy designation and Rare Pediatric Disease (RMAT) designation, with pivotal Phase I/II 3-year data meeting the primary endpoint [sec · filed 2026-04-27].
- Cash, cash equivalents, restricted cash, and investment securities totaled $624.1 million as of December 31, 2025, projected to fund operations into the second half of 2029 [sec_mda · as of 2026-03-02].
- As of March 31, 2026, the company reported cash and equivalents of $141.5 million, total revenue of $3.6 million, net loss of $53.5 million, shareholders' equity of $33.7 million, and total debt of $49.9 million [latest reported financials].
- The company is advancing multiple clinical candidates including AMT-162, AMT-191, and AMT-260 alongside AMT-130 [sec_mda · as of 2026-03-02].
- uniQure has held multiple Type B meetings with the FDA regarding the AMT-130 accelerated approval pathway and completed enrollment of Cohorts 3 and 4 of the Phase I/II studies [sec · filed 2026-04-27].
What works
- AMT-130 has received FDA Breakthrough Therapy designation and RMAT designation, supporting a potential accelerated approval pathway for Huntington's disease [sec · filed 2026-04-27].
- The company has a cash runway projected into the second half of 2029, providing financial flexibility to advance its pipeline [sec_mda · as of 2026-03-02].
- Pivotal Phase I/II 3-year data for AMT-130 met the primary endpoint, and the FDA has provided favorable feedback supportive of an accelerated approval pathway [sec · filed 2026-04-27].
What to weigh
- The company reported a net loss of $53.5 million for the quarter ended March 31, 2026, with accumulated deficits and negative shareholders' equity of $33.7 million [latest reported financials].
- There is risk that the FDA may ultimately conclude that the Phase I/II trial data are not sufficient to support a BLA or accelerated approval [sec · filed 2026-06-17].
- The company faces foreign currency exposure, recognizing a net foreign currency loss of $4.0 million in the six months ended June 30, 2026 [sec_mda · as of 2026-07-29].
- uniQure has total debt of $49.9 million as of March 31, 2026, with associated interest expense and repayment obligations [latest reported financials].
From the filings
Quoted directly from source documents.
AMT-130 pivotal Phase I/II 3-year data met the primary endpoint and received FDA Breakthrough designation.
“Presented pivotal Phase I/II 3-year data for AMT-130 meeting primary endpoint●Granted Breakthrough designation by the FDA”
SEC filings · April 27, 2026
Cash and investment securities of $624.1 million as of December 31, 2025, with runway into second half of 2029.
“As of December 31, 2025, we had cash and cash equivalents, restricted cash and investment securities of $624.1 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 · March 2, 2026
Risk that FDA may not accept Phase I/II data as sufficient for BLA or accelerated approval.
“the risk that the FDA ultimately concludes that the Phase I/II trial data are not sufficient to support a BLA or accelerated approval”
SEC filings · June 17, 2026
Risks & what to watch (4)›
Key risks
- Regulatory approval uncertainty for AMT-130 — FDA may conclude Phase I/II data are insufficient for BLA or accelerated approval [sec · filed 2026-06-17].
- Negative shareholders' equity
- Foreign currency exposure — Net foreign currency loss of $4.0 million in six months ended June 30, 2026, due to borrowings in euros [sec_mda · as of 2026-07-29].
- Debt repayment obligations — Total debt of $49.9 million as of March 31, 2026, with interest expense and repayment risks [latest reported financials].
What to watch
- FDA decision on whether Phase I/II data for AMT-130 are sufficient to support a BLA submission for accelerated approval [sec · filed 2026-06-17].
- Progress of ongoing clinical trials for AMT-162, AMT-191, and AMT-260 [sec_mda · as of 2026-03-02].
- Cash burn rate and any changes to the projected runway into the second half of 2029 [sec_mda · as of 2026-03-02].
- Updates on foreign currency movements and their impact on financial results [sec_mda · as of 2026-07-29].
Sources: SEC filings · SEC MD&A · Generated August 28, 2026 · How we verify sources →
Run uniQure N.V. through your own thesis
Use the pipeline, catalyst, financial, FDA, and market facts on this page as inputs to a protocol you control.
Your protocol can:
- Turn available uniQure N.V. facts into screen rules or deeper checks
- Show cited findings, blockers, and unresolved evidence together
- Save the logic and rerun it as the company evidence changes
Evidence & data
Score breakdownBased on 5 of 6 signals · derived from public data
22.3 months
D/E 4.21 · 0% dilution
Lead: Phase 1/Phase 2
4 candidates
No upcoming catalysts tracked
0 buys · 42 sells (180d)
Runway
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
217
Total value held
$1.02B
| Holder | Shares | Value |
|---|---|---|
| susquehanna international group, llp | 3,644,200 | $59.6M |
| nantahala capital management, llc | 2,837,078 | $46.4M |
| citigroup inc | 2,811,485 | $46.0M |
| two sigma investments, lp | 2,675,871 | $43.8M |
| rtw investments, lp | 2,586,356 | $42.3M |
| caption management, llc | 2,525,231 | $41.3M |
| state street corp | 2,260,512 | $37.0M |
| orbimed advisors llc | 2,136,149 | $34.9M |
| citadel advisors llc | 1,831,065 | $29.9M |
| 683 capital management, llc | 1,753,600 | $28.7M |
Showing top 10 of 217 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. (QURE) is a gene therapy company focused on developing treatments for central nervous system and liver diseases, with its lead candidate AMT-130 for Huntington's disease pursuing an accelerated FDA approval pathway.
What is uniQure N.V.'s lead drug candidate?
uniQure N.V.'s most advanced tracked candidate is amt-130 (Phase 2) for Huntington's disease.
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.