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uniQure

Biotech firm developing curative gene therapies

Overview

uniQure develops gene therapies aimed at curing severe genetic diseases. Its work centers on research and development, clinical trials, and partnerships to bring therapies to patients, including potential treatments for hemophilia B and Huntington's disease. The company’s products work by delivering genetic material to patients’ cells to correct or compensate for defective genes, with clinical programs progressing through trials to reach approval. Compared with peers, uniQure emphasizes curative gene therapy approaches and relies on licensing agreements and collaborations to fund development and scale its therapies. The ultimate goal is to provide approved, long‑lasting treatments that address unmet medical needs and improve patient outcomes.

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About uniQure

Simplify's Rating
Why uniQure is rated
B-
Rated B on Competitive Edge
Rated B on Growth Potential
Rated C on Differentiation

Industries

BiotechnologyHealthcare

Company Size

201-500

Company Stage

IPO

Headquarters

Amsterdam, Netherlands

Founded

1998

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Simplify's take

What believers are saying

  • FDA set a May 1, 2027 PDUFA date after accepting AMT-130's BLA.
  • UniQure reported $768.4 million cash on September 30, 2026, funding operations into 2030.
  • Management aligned with FDA on a 200-patient confirmatory study and U.S. screening by end-2026.

What critics are saying

  • The FDA requested a tentative advisory committee, raising approval risk before May 1, 2027.
  • Scocco v. uniQure remains active in SDNY, with a dismissal motion due November 4, 2026.
  • A May 2027 rejection would collapse uniQure's lead program and threaten the company.

What makes uniQure unique

  • AMT-130 targets Huntington's disease with a one-time brain-delivered gene-silencing therapy.
  • UniQure won FDA priority review on November 2, 2026, for AMT-130.
  • Its CSL Behring hemophilia B deal provides commercialization credibility and non-dilutive capital.

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Funding

Total Funding

$1.7B

Above

Industry Average

Funded Over

14 Rounds

Post IPO Equity funding comparison data is currently unavailable. We're working to provide this information soon!
Post IPO Equity Funding Comparison Coming Soon

Benefits

  • Remote Work Options
  • Hybrid Work Options

Stock Price

Company News

Yahoo Finance
Nov 6th, 2026
uniQure narrows Q3 loss to $61.2M as revenue rises to $5.2M

uniQure reported third quarter 2026 revenue of $5.2 million and a net loss of $61.22 million. Basic loss per share from continuing operations was $0.88, down from $1.38 a year earlier. For the first nine months, revenue was $14.6 million and the net loss was $195.81 million. The gene therapy company's main catalyst remains AMT-130, its Huntington's disease candidate. The company is also developing programmes for epilepsy, ALS, and Fabry disease. The biggest risk is that AMT-130 timelines or trial results slip while cash burn stays high. Analysts expect earnings of $20.0 million by 2029, with revenue reaching $445.8 million.

Yahoo Finance
Nov 4th, 2026
UniQure posts $61.2M Q3 loss, wider than expected, on $5.2M revenue

UniQure NV, the Amsterdam-based human gene therapy company, reported a third-quarter loss of $61.2 million on Wednesday. The loss came to 88 cents per share, wider than the 84-cent loss that four analysts surveyed by Zacks Investment Research had expected on average. Revenue for the period was $5.2 million.

WTOP
Nov 4th, 2026
UniQure: Q3 earnings snapshot.

UniQure: Q3 earnings snapshot. November 4, 2026 | 7:26 AM AMSTERDAM (AP) - AMSTERDAM (AP) - UniQure NV (QURE) on Wednesday reported a loss of $61.2 million in its third quarter. The Amsterdam-based company said it had a loss of 88 cents per share. The results fell short of Wall Street expectations. The average estimate of four analysts surveyed by Zacks Investment Research was for a loss of 84 cents per share. The human gene therapy company posted revenue of $5.2 million in the period. This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on QURE at https://www.zacks.com/ap/QURE Jayden West is a force on the field for McKinley Tech Jayden West is a force on the field for McKinley Tech Watch More

Associated Press
Nov 4th, 2026
uniQure wins FDA priority review for Huntington's therapy, with $768M cash funding it into 2030

uniQure's application to treat Huntington's disease with its gene therapy AMT-130 (ifezuntirgene inilparvovec) has been accepted for priority review by the US Food and Drug Administration (FDA). The agency set a target action date of May 1, 2027. The application seeks accelerated approval and rests on 36-month data from 12 high-dose patients, compared with an external control. The company says it has agreed key elements of a confirmatory study with the FDA, and US patient screening should begin by the end of 2026. For the third quarter, uniQure reported revenue of $5.2 million and a net loss of $61.2 million, down from $80.5 million a year earlier. Cash and investments stood at $768.4 million on September 30, 2026, which it expects will fund operations into 2030.

Simply Wall St
Oct 31st, 2026
How clinical scrutiny will impact uniQure (QURE) stock investors.

How clinical scrutiny will impact uniQure (QURE) stock investors. October 31, 2026 * uniQure recently faced fresh clinical scrutiny around its AMT-130 Huntington's program alongside a securities investigation, which has sharpened focus on treatment durability, regulatory risk and whether the balance sheet justifies the current premium price-to-book valuation. * ClearPoint Neuro's Q3 2026 revenue, of which 36% growth was reported, and its support for uniQure's AMT-130 regulatory work highlight how external partners are scaling infrastructure around the therapy even as questions build over long term outcomes and economic returns. * We will now assess how uniQure's investment narrative is affected by AMT-130 durability concerns revealed through new scrutiny of its clinical program. Compare uniQure's risk reward profile with other gene and neurology plays by scanning our hand picked 34 healthcare AI stocks that could be building similar treatment platforms. uniQure investment narrative recap. To own uniQure, you have to believe AMT-130 can move from an early Huntington's study into a real product while the rest of the pipeline steadily matures. The recent scrutiny around treatment durability and the securities investigation mostly circles that same question. For now, the key near term catalyst still looks like regulatory clarity around an AMT-130 filing timeline. The flip side is concentration risk. Revenue remains small at US$18.7 million against a reported loss of US$252.2 million, and AMT-130 carries clinical, safety and FDA decision risk. Any delay, new safety signal or tougher regulatory stance could pressure cash needs and challenge a stock that already trades at a premium P/B multiple. The most relevant operational backdrop here is ClearPoint Neuro's Q3 2026 update, which reported 36% revenue growth and continued investment to support cell and gene therapy trials, including AMT-130. This indicates that infrastructure around uniQure's Huntington's program is still being built out even as questions around long term efficacy and economics increase. For investors, that ClearPoint read through matters because it touches execution risk. A smoother neurosurgical delivery pathway can help reduce parts of the AMT-130 launch playbook risk if the therapy progresses. However, any stumble on procedure logistics or regulatory coordination could compound existing concerns about durability, manufacturing reliance and the current valuation premium. uniQure's narrative incorporates analyst expectations that revenue will grow at 188.0% annually and that earnings will shift from a loss of US$252.2 million today to US$20.0 million by 2029. This represents a swing of roughly US$272 million, with those projections implying about US$445.8 million of revenue and US$20.0 million of earnings in 2029. Uncover why uniQure's fair value indicates a 151% potential upside to its current price that could narrow quickly. Exploring other perspectives. Some readers will focus on a different catalyst for uniQure. The most bearish analysts fixate on the risk that AMT-130 long term data never fully convinces regulators, even though they still modeled revenue reaching about US$121.7 million and earnings of roughly US$19.4 million by 2029. Those views were set before this latest news. Investors may see opinions shift and explore several angles before deciding where they sit. Explore 4 other uniQure fair value estimates, including one that suggests it could be worth just $27.96. Reach your own conclusion. Disagree with existing narratives? Extraordinary investment returns rarely come from following the herd, so go with your instincts. * A great starting point for your uniQure research is our analysis highlighting 2 key rewards and 1 important warning sign that could impact your investment decision. * See our latest analysis for uniQure. The report includes a comprehensive fundamental analysis summarized in a single visual, the Snowflake, making it easy to evaluate uniQure's overall financial health at a glance. Looking for more uniQure sized investment ideas? If uniQure's risk reward trade off has you thinking about position sizing and diversification, it can help to line it up against a broader bench of companies with different balance sheet strength, valuation profiles and income potential. * For steadier sleep at night, scan a 30 resilient stocks with low risk scores that combine resilient financial profiles with lower risk scores. * If income is your focus, sort through a 5 dividend fortresses that aim to pair yields above 5% with robust payout support. * To hunt for earlier stage potential, filter a 9 elite penny stocks with strong financials that already show stronger fundamentals than the typical low priced stock. Valuation is complex, but we're here to simplify it. Discover if uniQure might be undervalued or overvalued with our detailed analysis, featuring fair value estimates, potential risks, dividends, insider trades, and its financial condition. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email [email protected]

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