Tearsheet

Investment Highlights Why It Matters Detailed financial logic regarding cash flow yields vs trend-riding momentum.

0

Cash is significant % of market cap
Net D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -20%

Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.

Meaningful short interest
Short Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 19%

Not profitable at operating income level
Op Inc LTMOperating Income, Last Twelve Months is -188 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -1040%

Expensive valuation multiples
P/SPrice/Sales ratio is 144x

Stock price has recently run up significantly
12M Rtn12 month market price return is 189%

Weak revenue growth
Rev Chg LTMRevenue Change % Last Twelve Months (LTM) is -10%, Rev Chg 3Y AvgRevenue Change % averaged over trailing 3 years is -29%

Significant share based compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 101%

Not cash flow generative
CFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -951%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -954%

Valuation getting more expensive
P/S 6M Chg %Price/Sales change over 6 months. Declining P/S indicates valuation has become less expensive. is 55%

Yield minus risk free rate is negative
ERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -12%

High stock price volatility
Vol 12M is 284%

Key risks
QURE key risks include [1] an uncertain regulatory pathway for its lead Huntington's disease therapy, Show more.

0 Cash is significant % of market cap
Net D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -20%
1 Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.
2 Meaningful short interest
Short Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 19%
3 Not profitable at operating income level
Op Inc LTMOperating Income, Last Twelve Months is -188 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -1040%
4 Expensive valuation multiples
P/SPrice/Sales ratio is 144x
5 Stock price has recently run up significantly
12M Rtn12 month market price return is 189%
6 Weak revenue growth
Rev Chg LTMRevenue Change % Last Twelve Months (LTM) is -10%, Rev Chg 3Y AvgRevenue Change % averaged over trailing 3 years is -29%
7 Significant share based compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 101%
8 Not cash flow generative
CFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -951%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -954%
9 Valuation getting more expensive
P/S 6M Chg %Price/Sales change over 6 months. Declining P/S indicates valuation has become less expensive. is 55%
10 Yield minus risk free rate is negative
ERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -12%
11 High stock price volatility
Vol 12M is 284%
12 Key risks
QURE key risks include [1] an uncertain regulatory pathway for its lead Huntington's disease therapy, Show more.

QURE in ETFs

Weight = QURE's share of each fund

XBI1.0%
SCHA0.06%
VHT0.02%
SCHB0.00%

Valuation & Metrics

Price Chart

Why The Stock Moved

Qualitative Assessment

AI Analysis | Feedback

Updated on 7/2/2026

uniQure (QURE) stock has gained about 155% since 3/31/2026 because of the following key factors:

1. Favorable Shift in U.S. FDA Regulatory Guidance for AMT-130.

uniQure experienced a significant reversal in regulatory sentiment for its Huntington's disease gene therapy candidate, AMT-130, during fiscal Q2 2026. Initially, in March 2026 (the start of the period), the U.S. Food and Drug Administration (FDA) indicated that existing Phase I/II clinical data were insufficient for a marketing application and recommended a prospective, randomized, sham-controlled study. However, by June 17, 2026, uniQure announced that the FDA agreed the three-year Phase I/II data could serve as the primary basis for a Biologics License Application (BLA) seeking accelerated approval. The company plans to submit the BLA in fiscal Q3 2026, and the FDA has indicated it will work expeditiously to align on the confirmatory study design, potentially foregoing a full sham-surgery-controlled Phase III trial, which significantly accelerates the potential commercialization timeline.

2. Advancement Towards UK Regulatory Submission for AMT-130.

Further bolstering the commercial prospects of AMT-130, uniQure held positive pre-submission meetings with the UK Medicines and Healthcare products Regulatory Agency (MHRA) during fiscal Q2 2026. The company now expects to file a Marketing Authorization Application (MAA) for AMT-130 in Huntington's disease in fiscal Q3 2026. This submission will also rely on the three-year Phase I/II data, which demonstrated a 75% slowing of disease progression at the high dose (p=0.003) compared to an external control, opening another key international market.

Show more
Updated on 7/2/2026

uniQure (QURE) stock has gained about 155% since 3/31/2026 because of the following key factors:

1. Favorable Shift in U.S. FDA Regulatory Guidance for AMT-130.

uniQure experienced a significant reversal in regulatory sentiment for its Huntington's disease gene therapy candidate, AMT-130, during fiscal Q2 2026. Initially, in March 2026 (the start of the period), the U.S. Food and Drug Administration (FDA) indicated that existing Phase I/II clinical data were insufficient for a marketing application and recommended a prospective, randomized, sham-controlled study. However, by June 17, 2026, uniQure announced that the FDA agreed the three-year Phase I/II data could serve as the primary basis for a Biologics License Application (BLA) seeking accelerated approval. The company plans to submit the BLA in fiscal Q3 2026, and the FDA has indicated it will work expeditiously to align on the confirmatory study design, potentially foregoing a full sham-surgery-controlled Phase III trial, which significantly accelerates the potential commercialization timeline.

2. Advancement Towards UK Regulatory Submission for AMT-130.

Further bolstering the commercial prospects of AMT-130, uniQure held positive pre-submission meetings with the UK Medicines and Healthcare products Regulatory Agency (MHRA) during fiscal Q2 2026. The company now expects to file a Marketing Authorization Application (MAA) for AMT-130 in Huntington's disease in fiscal Q3 2026. This submission will also rely on the three-year Phase I/II data, which demonstrated a 75% slowing of disease progression at the high dose (p=0.003) compared to an external control, opening another key international market.

3. Numerous Analyst Upgrades and Increased Price Targets.

The positive regulatory developments in fiscal Q2 2026 led to a wave of favorable analyst revisions. In June 2026, several prominent firms either upgraded uniQure's rating or substantially raised their price targets. For example, Barclays upgraded the stock to Overweight and increased its price target from $25 to $65 on June 18, 2026. Mizuho maintained an Outperform rating, raising its price target from $35 to $68 on June 25, 2026. HC Wainwright & Co. also maintained a Buy rating, increasing its price target from $50 to $80 on June 29, 2026. These upgrades and increased targets reflected renewed confidence in the accelerated pathway for AMT-130.

4. Successful Upsized Public Offering Strengthening Financial Position.

To capitalize on the positive momentum and fund its accelerated development and commercialization plans, uniQure executed a successful upsized public offering. The company initially proposed a $150 million offering on June 22, 2026, which was subsequently upsized to $225 million on June 23, 2026. The offering officially closed on June 25, 2026, with the sale of 5,686,813 ordinary shares at a public offering price of $45.50 per share. This capital raise provides uniQure with significant funding to support commercialization readiness activities, potential commercial launches of AMT-130, a confirmatory study, and the development of its other clinical product candidates.

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Stock Movement Drivers

Fundamental Drivers

The 153.3% change in QURE stock from 3/31/2026 to 7/7/2026 was primarily driven by a 126.7% change in the company's P/S Multiple.
(LTM values as of)33120267072026Change
Stock Price ($)16.3541.41153.3%
Change Contribution By: 
Total Revenues ($ Mil)161812.4%
P/S Multiple63.3143.6126.7%
Shares Outstanding (Mil)6263-0.6%
Cumulative Contribution153.3%

LTM = Last Twelve Months as of date shown

Market Drivers

3/31/2026 to 7/7/2026
ReturnCorrelation
QURE153.3% 
Market (SPY)15.0%-13.6%
Sector (XLV)12.2%-16.8%

Fundamental Drivers

The 73.0% change in QURE stock from 12/31/2025 to 7/7/2026 was primarily driven by a 61.5% change in the company's P/S Multiple.
(LTM values as of)123120257072026Change
Stock Price ($)23.9341.4173.0%
Change Contribution By: 
Total Revenues ($ Mil)161814.9%
P/S Multiple88.9143.661.5%
Shares Outstanding (Mil)5963-6.7%
Cumulative Contribution73.0%

LTM = Last Twelve Months as of date shown

Market Drivers

12/31/2025 to 7/7/2026
ReturnCorrelation
QURE73.0% 
Market (SPY)9.9%2.3%
Sector (XLV)6.7%-4.3%

Fundamental Drivers

The 197.1% change in QURE stock from 6/30/2025 to 7/7/2026 was primarily driven by a 291.8% change in the company's P/S Multiple.
(LTM values as of)63020257072026Change
Stock Price ($)13.9441.41197.1%
Change Contribution By: 
Total Revenues ($ Mil)2018-10.4%
P/S Multiple36.6143.6291.8%
Shares Outstanding (Mil)5363-15.4%
Cumulative Contribution197.1%

LTM = Last Twelve Months as of date shown

Market Drivers

6/30/2025 to 7/7/2026
ReturnCorrelation
QURE197.1% 
Market (SPY)22.0%-1.0%
Sector (XLV)23.6%-2.3%

Fundamental Drivers

The 261.3% change in QURE stock from 6/30/2023 to 7/7/2026 was primarily driven by a 2806.2% change in the company's P/S Multiple.
(LTM values as of)63020237072026Change
Stock Price ($)11.4641.41261.3%
Change Contribution By: 
Total Revenues ($ Mil)11018-83.6%
P/S Multiple4.9143.62806.2%
Shares Outstanding (Mil)4763-24.4%
Cumulative Contribution261.3%

LTM = Last Twelve Months as of date shown

Market Drivers

6/30/2023 to 7/7/2026
ReturnCorrelation
QURE261.3% 
Market (SPY)74.6%8.7%
Sector (XLV)29.7%5.0%

Return vs. Risk

Price Returns Compared

 202120222023202420252026Total [1]
Returns
QURE Return-43%9%-70%161%36%73%14%
Peers Return-26%17%4%-19%27%3%-4%
S&P 500 Return27%-19%24%23%16%10%101%

Monthly Win Rates [3]
QURE Win Rate25%33%8%50%33%57% 
Peers Win Rate43%62%48%42%60%51% 
S&P 500 Win Rate75%42%67%75%67%57% 

Max Drawdowns [4]
QURE Max Drawdown-52%-39%-76%-55%-72%-68% 
Peers Max Drawdown-42%-29%-34%-41%-43%-32% 
S&P 500 Max Drawdown-5%-25%-10%-8%-19%-9% 


[1] Cumulative total returns since the beginning of 2021
[2] Peers: BMRN, SRPT, IONS, VRTX, RGNX. See QURE Returns vs. Peers.
[3] Win Rate = % of calendar months in which monthly returns were positive
[4] Max drawdown represents maximum peak-to-trough decline within a year
[5] 2026 data is for the year up to 7/7/2026 (YTD)

How Low Can It Go

EventQURES&P 500
2025 US Tariff Shock
  % Loss-37.8%-18.8%
  % Gain to Breakeven60.8%23.1%
  Time to Breakeven14 days79 days
2024 Yen Carry Trade Unwind
  % Loss-35.1%-7.8%
  % Gain to Breakeven54.0%8.5%
  Time to Breakeven113 days18 days
Summer-Fall 2023 Five Percent Yield Shock
  % Loss-40.8%-9.5%
  % Gain to Breakeven68.9%10.5%
  Time to Breakeven257 days24 days
2023 SVB Regional Banking Crisis
  % Loss-47.9%-6.7%
  % Gain to Breakeven92.1%7.1%
  Time to Breakeven818 days31 days
2022 Inflation Shock & Fed Tightening
  % Loss-38.8%-24.5%
  % Gain to Breakeven63.5%32.4%
  Time to Breakeven57 days427 days
2020 COVID-19 Crash
  % Loss-38.3%-33.7%
  % Gain to Breakeven62.2%50.9%
  Time to Breakeven76 days140 days

Compare to BMRN, SRPT, IONS, VRTX, RGNX

In The Past

uniQure's stock fell -37.8% during the 2025 US Tariff Shock. Such a loss loss requires a 60.8% gain to breakeven.

Preserve Wealth

Limiting losses and compounding gains is essential to preserving wealth.

Asset Allocation

Actively managed asset allocation strategies protect wealth. Learn more.

EventQURES&P 500
2025 US Tariff Shock
  % Loss-37.8%-18.8%
  % Gain to Breakeven60.8%23.1%
  Time to Breakeven14 days79 days
2024 Yen Carry Trade Unwind
  % Loss-35.1%-7.8%
  % Gain to Breakeven54.0%8.5%
  Time to Breakeven113 days18 days
Summer-Fall 2023 Five Percent Yield Shock
  % Loss-40.8%-9.5%
  % Gain to Breakeven68.9%10.5%
  Time to Breakeven257 days24 days
2023 SVB Regional Banking Crisis
  % Loss-47.9%-6.7%
  % Gain to Breakeven92.1%7.1%
  Time to Breakeven818 days31 days
2022 Inflation Shock & Fed Tightening
  % Loss-38.8%-24.5%
  % Gain to Breakeven63.5%32.4%
  Time to Breakeven57 days427 days
2020 COVID-19 Crash
  % Loss-38.3%-33.7%
  % Gain to Breakeven62.2%50.9%
  Time to Breakeven76 days140 days
Q4 2018 Fed Policy Error / Growth Scare
  % Loss-31.1%-19.2%
  % Gain to Breakeven45.1%23.8%
  Time to Breakeven78 days105 days
2016-2017 Trump Reflation Bond Selloff
  % Loss-42.0%-3.7%
  % Gain to Breakeven72.4%3.9%
  Time to Breakeven76 days6 days
2015-2016 China Devaluation / Global Growth Scare
  % Loss-60.7%-12.2%
  % Gain to Breakeven154.2%13.9%
  Time to Breakeven755 days62 days
2014-2016 Oil Price Collapse
  % Loss-20.8%-6.8%
  % Gain to Breakeven26.2%7.3%
  Time to Breakeven27 days15 days
2013 Taper Tantrum
  % Loss-39.1%-0.2%
  % Gain to Breakeven64.2%0.2%
  Time to Breakeven219 days1 days

Compare to BMRN, SRPT, IONS, VRTX, RGNX

In The Past

uniQure's stock fell -37.8% during the 2025 US Tariff Shock. Such a loss loss requires a 60.8% gain to breakeven.

Preserve Wealth

Limiting losses and compounding gains is essential to preserving wealth.

Asset Allocation

Actively managed asset allocation strategies protect wealth. Learn more.

About uniQure (QURE)

uniQure N.V. (QURE) is a biotechnology company specializing in the development of gene therapies. Based in the Netherlands, the company's core mission is to create innovative treatments for individuals affected by severe genetic and other debilitating diseases. Its foundational approach involves leveraging gene therapy technology to address the underlying causes of these conditions, aiming to provide long-term therapeutic solutions.

The company's most advanced product candidate is Etranacogene dezaparvovec (AMT-061), currently in a pivotal Phase III clinical trial for the treatment of hemophilia B, a genetic bleeding disorder. Beyond this lead program, uniQure boasts a robust pipeline of gene therapies targeting other serious neurological conditions. Notable programs include AMT-130, in Phase I/II development for Huntington's disease, and early-stage candidates like AMT-210 for Parkinson's disease, AMT-260 for temporal lobe epilepsy, and AMT-240 for autosomal dominant Alzheimer's disease, among others.

uniQure's primary customers are patients living with rare and devastating genetic or neurological disorders for which current treatment options may be limited or insufficient. By focusing on conditions such as hemophilia B, Huntington's, Parkinson's, and certain forms of epilepsy and Alzheimer's, uniQure aims to serve critical unmet medical needs within the global pharmaceutical and biotechnology market, specifically within the specialty field of advanced gene therapies.

AI Analysis | Feedback

Analogy 1: Imagine **Moderna**, but instead of mRNA technology, uniQure focuses on developing groundbreaking gene therapies for genetic diseases.

Analogy 2: A **Vertex Pharmaceuticals** dedicated to creating gene therapies for severe genetic conditions like hemophilia and Huntington's disease.

AI Analysis | Feedback

  • Etranacogene dezaparvovec (AMT-061): A gene therapy in Phase III clinical trial for the treatment of hemophilia B.
  • AMT-130: A gene therapy in Phase I/II clinical study for the treatment of Huntington's disease.
  • AMT-060: A gene therapy in Phase I/II clinical trial for the treatment of hemophilia B.
  • AMT-210: A product candidate for the treatment of Parkinson's disease.
  • AMT-260: A product candidate for the treatment of temporal lobe epilepsy.
  • AMT-240: A preclinical product candidate for the treatment of autosomal dominant Alzheimer's disease.
  • AMT-161: A product candidate for the treatment of amyotrophic lateral sclerosis.

AI Analysis | Feedback

uniQure N.V. (QURE) is a gene therapy company primarily engaged in the research and development of treatments for genetic and other devastating diseases. As a biopharmaceutical company with most of its pipeline in clinical development stages, uniQure does not typically sell directly to individual patients. Instead, its "customers" or key partners for commercialized products are other pharmaceutical companies or healthcare systems. For its lead program, Etranacogene dezaparvovec (AMT-061), which is now marketed under the brand name HEMGENIX® for hemophilia B, uniQure has licensed the global commercialization rights. Therefore, its major customer/partner for this commercialized product is: * **CSL Behring**, a subsidiary of **CSL Limited** (ASX: CSL). CSL Behring holds the exclusive global license for the commercialization of HEMGENIX®. For its other pipeline product candidates, such as AMT-130 for Huntington's disease, AMT-210 for Parkinson's disease, and others, these are still in various stages of clinical trials or preclinical development. Consequently, uniQure currently does not have commercial customers for these investigational therapies. If these programs are successfully developed and approved, uniQure may commercialize them independently, partner with other pharmaceutical companies, or license them out, similar to the arrangement for HEMGENIX®.

AI Analysis | Feedback

  • Bristol Myers Squibb Company (BMY)
  • Catalent, Inc. (CTLT)
  • Lonza Group Ltd (LONN)

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Matt Kapusta, Chief Executive Officer
Matt Kapusta has served as Chief Executive Officer of uniQure since December 2016 and is also an Executive Director on the Board. He previously held the role of Chief Financial Officer for uniQure from January 2015 until June 2021. Prior to joining uniQure, Mr. Kapusta held executive positions at AngioDynamics from 2011 to 2015 and Smith & Nephew from 2009 to 2011. His career also includes more than a decade of investment banking experience focused on emerging life-sciences companies, including serving as Managing Director, Healthcare Investment Banking at Collins Stewart, and various roles at Wells Fargo Securities, Robertson Stephens, and PaineWebber.

Christian Klemt, Chief Financial Officer
Christian Klemt was appointed Chief Financial Officer of uniQure in June 2021. Prior to this role, he served as uniQure's Chief Accounting Officer from August 2017 to June 2021. Mr. Klemt also continues to serve as the General Manager of uniQure's Amsterdam site.

Walid Abi-Saab, M.D., Chief Medical Officer
Walid Abi-Saab joined uniQure as Chief Medical Officer in June 2023. In this role, he is responsible for leading clinical research and development, regulatory affairs, medical affairs, and program management. Before uniQure, Dr. Abi-Saab served as Chief Medical Officer at Galapagos from 2017 through 2022, where he expanded the company's development organization and led the successful completion of Phase 3 studies for filgotinib. He has also held leadership positions at companies such as Novartis, Abbott Laboratories, Pfizer, and served as Group Vice President, Global Clinical Development at Shire.

Richard Porter, Ph.D., Chief Business and Scientific Officer
Richard Porter assumed responsibilities for research, non-clinical, and vector development in his role as Chief Business and Scientific Officer following a strategic reorganization in October 2023. He also continues to oversee business development and product planning. Dr. Porter joined uniQure in June 2021 through the acquisition of Corlieve Therapeutics, where he was the founder and Chief Executive Officer. He possesses over 25 years of neuroscience leadership experience in the biopharma industry.

Kylie O'Keefe, Chief Customer and Strategy Officer
Kylie O'Keefe was appointed Chief Customer and Strategy Officer in June 2025, where she is responsible for leading the development and execution of uniQure's global commercialization strategy for AMT-130, including all commercial functions and medical affairs. Prior to joining uniQure, Ms. O'Keefe served as Chief Commercial Officer at PTC Therapeutics, overseeing global commercial strategy, operations, and portfolio management for multiple rare neurology and metabolic commercial products across more than 50 countries. Her experience includes leading several strategically significant commercial launches and supporting corporate strategy and pipeline development for both small molecules and gene therapies.

AI Analysis | Feedback

uniQure (NASDAQ: QURE) faces several significant business risks, primarily stemming from the challenging regulatory landscape and the inherent uncertainties of gene therapy development. The most prominent risk centers on **regulatory and legal challenges for its Huntington's disease gene therapy candidate, AMT-130**. The U.S. Food and Drug Administration (FDA) has "strongly recommended" that uniQure conduct a new, costly, and lengthy Phase III sham-controlled study for AMT-130, rejecting the company's current data package for accelerated approval. This decision significantly alters the drug's path to market, increasing both development risks and capital requirements. Further exacerbating this risk, an unnamed senior FDA official has publicly criticized uniQure, accusing the company of presenting "distorted" and "manipulated" clinical data for a "failed" therapy and of misleading behavior in its interactions with federal health authorities. These accusations have led to a substantial drop in uniQure's share price and have prompted multiple law firms to file securities fraud class-action lawsuits against the company, alleging misleading statements about FDA alignment and timelines for AMT-130. Such legal actions expose uniQure to significant financial penalties and reputational damage. A second key risk arises from the **inherent complexities and uncertainties of gene therapy development**. Developing gene therapies is a costly endeavor with a high risk of clinical trial failure. While uniQure has programs in various clinical phases, there are ongoing concerns regarding long-term safety, including potential delayed adverse effects, immune responses, and unintended off-target effects. Manufacturing these advanced therapeutic medicinal products also presents significant challenges related to quality control, scalability, and standardization. Even if a therapy gains regulatory approval, commercialization hurdles such as high treatment costs and challenges in market acceptance and reimbursement pose additional risks. Finally, uniQure's **financial health and cash burn** present a significant business risk. The company has experienced a substantial revenue growth decline and a deeply negative net margin over the past three years. Although uniQure currently holds a strong cash position, its considerable annual cash burn rate, estimated at nearly $200 million, means that the newly recommended, extended Phase III trial for AMT-130 will severely test its financial resilience. The company's Altman Z-Score, which indicates financial distress, is also low, implying a potential risk of bankruptcy in the near future.

AI Analysis | Feedback

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AI Analysis | Feedback

uniQure N.V. (symbol: QURE) operates in the gene therapy space, developing treatments for genetic and other devastating diseases. The addressable markets for their main products are as follows:

Etranacogene dezaparvovec (HEMGENIX) for Hemophilia B

HEMGENIX (etranacogene dezaparvovec) is uniQure's approved gene therapy for hemophilia B. The global market for hemophilia B gene therapy was valued at approximately USD 0.51 billion in 2023 and is projected to reach USD 3.43 billion by 2032, growing at a compound annual growth rate (CAGR) of roughly 23.60% between 2024 and 2032. The broader global hemophilia B market was valued at US$ 2,956.0 million in 2024 and is estimated to grow to US$ 4,362.0 million by 2030. In the United States, the hemophilia B market size was approximately USD 2 billion in 2023.

AMT-130 for Huntington's Disease

Huntington's disease impacts approximately 75,000 people across the U.S., Europe, and the UK. The global Huntington's disease treatment market was valued at USD 1.2 billion in 2024 and is projected to reach USD 3.7 billion by 2034, exhibiting an 8.9% CAGR. Another estimate places the global market at USD 578.9 million in 2024, expected to reach USD 2,443 million by 2033 with a CAGR of 17.4% from 2025-2033. The North American market for Huntington's disease treatment held the leading regional position in 2024, with an estimated value of approximately USD 0.5 billion.

AMT-210 for Parkinson's Disease

uniQure announced in October 2023 its decision to discontinue the AMT-210 program for Parkinson's disease as part of a strategic reorganization. Therefore, there is no addressable market size for this specific product from uniQure's portfolio.

AI Analysis | Feedback

uniQure N.V. (QURE) is expected to drive future revenue growth over the next 2-3 years primarily through the commercialization of its gene therapies. The key drivers include:
  • Commercialization and Market Penetration of Hemgenix (etranacogene dezaparvovec): uniQure's approved gene therapy for hemophilia B, Hemgenix, is a current source of revenue, and its continued rollout and increasing adoption in the market are anticipated to contribute to revenue growth.
  • Potential Regulatory Approval and Commercial Launch of AMT-130 for Huntington's Disease: Despite recent regulatory setbacks and the U.S. FDA recommending a Phase III sham-controlled study, AMT-130 remains a significant potential revenue driver. Analysts project substantial annual revenue if approved, with a potential commercial launch in 2027 or 2028.
  • Expansion into International Markets: The company is actively engaging with regulatory authorities outside the U.S. for its pipeline assets, including AMT-130. This strategic focus on international markets presents an opportunity for geographical expansion and increased revenue from existing or newly approved products.

AI Analysis | Feedback

Share Issuance

  • In 2025, uniQure received aggregate net proceeds of $404.2 million from follow-on public offerings, which included the issuance of 11.8 million ordinary shares and pre-funded warrants to purchase 0.5 million ordinary shares.
  • An underwritten public offering in September 2025 generated approximately $345 million in gross proceeds from the sale of 6,736,841 ordinary shares and 526,316 pre-funded warrants.
  • Net proceeds of $80.5 million were raised in January and February 2025 through a follow-on offering.

Inbound Investments

  • uniQure refinanced its existing $50 million debt in the third quarter of 2025, extending the term to October 2030 and reducing its cost of capital.
  • An additional term loan tranche of $100 million is available, subject to a pre-defined regulatory milestone for AMT-130, with a third tranche of $25 million also available upon lender approval.
  • The company's cash and investments grew to $622.5 million as of December 31, 2025, primarily due to proceeds from public offerings, up from $367.5 million at the end of 2024.

Capital Expenditures

  • Research and development expenses totaled $140.7 million in 2025, a decrease from $143.8 million in 2024.
  • Selling, general and administrative expenses were $65.5 million in 2025, increasing from $52.7 million in 2024, partly due to $6.5 million incurred for the planned commercialization of AMT-130 in the United States.
  • In the third quarter of 2025, direct research and development expenses increased by $10.1 million, including $6.6 million related to the preparation for the Biologics License Application submission for AMT-130.

Better Bets vs. uniQure (QURE)

Latest Trefis Analyses

Recent Active Movers

Peer Comparisons

Peers to compare with:

Financials

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Mkt Price41.4160.3519.7986.33522.2515.0250.88
Mkt Cap2.611.62.114.2132.70.87.1
Rev LTM183,2422,1841,05812,218881,621
Op Inc LTM-1883151-3524,662-257-94
FCF LTM-173759108-9783,710-236-32
FCF 3Y Avg-161514-414-6291,977-174-168
CFO LTM-172874175-9114,241-2342
CFO 3Y Avg-156618-302-5792,401-170-163

Growth & Margins

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Rev Chg LTM-10.4%9.9%-2.2%47.5%10.1%-44.0%3.8%
Rev Chg 3Y Avg-29.0%14.3%33.6%24.9%9.9%5.3%12.1%
Rev Chg Q127.3%2.8%-1.9%87.0%7.8%-92.8%5.3%
QoQ Delta Rev Chg LTM12.4%0.7%-0.6%12.1%1.8%-48.5%1.2%
Op Inc Chg LTM-13.8%-49.1%100.9%25.4%1,214.0%-63.1%5.8%
Op Inc Chg 3Y Avg-12.2%93.5%53.7%6.9%364.1%-8.4%30.3%
Op Mgn LTM-1,039.8%9.7%0.0%-33.3%38.2%-292.3%-16.6%
Op Mgn 3Y Avg-1,079.8%13.5%-4.0%-49.8%24.1%-232.3%-26.9%
QoQ Delta Op Mgn LTM86.0%-3.0%30.0%7.2%0.2%-197.7%3.7%
CFO/Rev LTM-951.2%27.0%8.0%-86.1%34.7%-266.2%-39.0%
CFO/Rev 3Y Avg-816.3%20.7%-18.2%-65.9%21.5%-180.8%-42.0%
FCF/Rev LTM-954.1%23.4%5.0%-92.4%30.4%-269.2%-43.7%
FCF/Rev 3Y Avg-845.4%17.0%-24.3%-71.8%17.7%-184.6%-48.0%

Valuation

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Mkt Cap2.611.62.114.2132.70.87.1
P/S143.63.61.013.510.99.09.9
P/Op Inc-13.836.91,984.4-40.428.5-3.112.7
P/EBIT-18.328.426.0-58.826.2-3.211.4
P/E-12.443.231.9-43.530.6-2.713.9
P/CFO-15.113.311.9-15.631.3-3.44.2
Total Yield-8.0%2.3%3.1%-2.3%3.3%-36.8%0.0%
Dividend Yield0.0%0.0%0.0%0.0%0.0%0.0%0.0%
FCF Yield 3Y Avg-33.9%4.2%-4.0%-8.4%1.8%-32.7%-6.2%
D/E0.00.10.50.10.00.10.1
Net D/E-0.2-0.10.20.0-0.0-0.1-0.1

Returns

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
1M Rtn56.6%6.3%25.7%15.9%16.9%147.0%21.3%
3M Rtn141.6%7.3%-14.3%16.8%20.9%70.7%18.9%
6M Rtn85.6%0.5%-11.5%5.9%11.5%5.9%5.9%
12M Rtn189.4%5.1%14.3%106.6%13.7%78.2%46.2%
3Y Rtn264.8%-29.1%-81.8%106.6%54.4%-22.9%15.8%
1M Excs Rtn49.0%7.9%19.8%16.0%19.3%119.4%19.6%
3M Excs Rtn124.6%-6.1%-26.7%2.7%6.8%57.2%4.7%
6M Excs Rtn67.6%-7.9%-16.5%-1.1%6.1%1.3%0.1%
12M Excs Rtn170.5%-13.9%-11.0%78.5%-5.2%54.7%24.8%
3Y Excs Rtn201.7%-102.8%-153.0%39.3%-20.7%-94.9%-57.8%

Comparison Analyses

null

Financials

Segment Financials

Revenue by Segment
$ Mil20252024202320222021
Discovery and development of innovative gene therapies162716  
Collaboration revenues   57
Contract manufacturing revenues   2 
License revenues   100517
Total162716106524


Price Behavior

Price Behavior
Market Price$41.41 
Market Cap ($ Bil)2.6 
First Trading Date02/05/2014 
Distance from 52W High-41.3% 
   50 Days200 Days
DMA Price$29.85$28.98
DMA Trendupup
Distance from DMA38.7%42.9%
 3M1YR
Volatility182.2%284.6%
Downside Capture-299.28-150.63
Upside Capture159.8910.79
Correlation (SPY)-12.6%-0.8%
QURE Betas & Captures as of 6/30/2026

 1M2M3M6M1Y3Y
Beta-4.20-2.90-1.220.27-0.241.11
Up Beta1.171.181.512.783.081.36
Down Beta-4.45-5.03-6.29-2.880.341.68
Up Capture-147%93%228%160%10%155%
Bmk +ve Days11244067140429
Stock +ve Days8193260121340
Down Capture-927%-843%-538%12%-402%93%
Bmk -ve Days10172358112321
Stock -ve Days12213064129399

[1] Upside and downside betas calculated using positive and negative benchmark daily returns respectively
Based On 1-Year Data
Annualized
Return
Annualized
Volatility
Sharpe
Ratio
Correlation
with QURE
QURE190.6%284.6%1.13-
Sector ETF (XLV)23.0%15.7%1.13-2.3%
Equity (SPY)20.7%12.5%1.22-1.0%
Gold (GLD)23.0%27.8%0.73-3.2%
Commodities (DBC)22.9%18.6%0.971.2%
Real Estate (VNQ)13.6%13.8%0.68-12.8%
Bitcoin (BTCUSD)-41.8%42.8%-1.142.4%

Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 5-Year Data
Annualized
Return
Annualized
Volatility
Sharpe
Ratio
Correlation
with QURE
QURE5.8%157.9%0.52-
Sector ETF (XLV)7.1%14.9%0.299.4%
Equity (SPY)13.3%17.1%0.6013.9%
Gold (GLD)17.8%18.3%0.79-0.9%
Commodities (DBC)7.6%19.5%0.290.7%
Real Estate (VNQ)3.1%18.9%0.066.7%
Bitcoin (BTCUSD)13.2%53.5%0.438.0%

Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 10-Year Data
Annualized
Return
Annualized
Volatility
Sharpe
Ratio
Correlation
with QURE
QURE18.8%122.2%0.54-
Sector ETF (XLV)10.9%16.6%0.5414.8%
Equity (SPY)15.7%17.9%0.7517.1%
Gold (GLD)11.6%16.1%0.590.5%
Commodities (DBC)6.2%18.0%0.273.4%
Real Estate (VNQ)5.6%20.7%0.239.2%
Bitcoin (BTCUSD)57.9%66.2%0.987.7%

Smart multi-asset allocation framework can stack odds in your favor. Learn How

Short Interest

Short Interest: As Of Date6152026
Short Interest: Shares Quantity11.8 Mil
Short Interest: % Change Since 53120266.4%
Average Daily Volume1.4 Mil
Days-to-Cover Short Interest8.2 days
Basic Shares Quantity62.7 Mil
Short % of Basic Shares18.8%

Earnings Returns History

Updated 6/17/2026
Expand for More
 Forward Returns
Earnings Date1D Returns5D Returns21D Returns
5/5/20262.3%33.7%44.5%
3/2/2026-32.8%-8.7%-5.8%
11/10/2025-5.9%7.5%-27.5%
7/29/2025-8.7%-8.1%5.4%
5/9/20254.4%8.2%38.0%
2/27/20253.4%3.6%12.5%
11/5/20249.0%37.8%13.1%
8/1/2024-2.3%-7.0%-18.8%
...
SUMMARY STATS   
# Positive141012
# Negative101412
Median Positive3.2%7.7%14.2%
Median Negative-4.3%-8.4%-14.0%
Max Positive12.7%37.8%44.5%
Max Negative-32.8%-25.3%-27.5%
Collapse to Preview
 Forward Returns
Earnings Date1D Returns5D Returns21D Returns
5/5/20262.3%33.7%44.5%
3/2/2026-32.8%-8.7%-5.8%
11/10/2025-5.9%7.5%-27.5%
7/29/2025-8.7%-8.1%5.4%
5/9/20254.4%8.2%38.0%
2/27/20253.4%3.6%12.5%
11/5/20249.0%37.8%13.1%
8/1/2024-2.3%-7.0%-18.8%
5/7/20242.1%3.4%15.0%
2/28/2024-0.9%-25.3%-19.9%
11/7/202312.7%-1.8%15.4%
8/1/2023-5.3%-19.2%-17.8%
5/9/20230.8%0.2%-5.6%
2/27/2023-0.2%-0.7%-4.4%
11/2/2022-2.7%-8.3%28.8%
8/8/2022-27.0%-20.2%-23.6%
5/2/20221.0%-14.5%-10.2%
2/28/20222.9%-8.5%4.8%
10/25/2021-3.3%8.0%-4.2%
7/27/20214.5%7.2%4.2%
5/10/20211.7%-1.7%13.3%
3/1/20213.7%-11.5%-17.8%
10/28/20203.6%7.8%21.6%
7/31/20202.1%-1.0%-2.3%
SUMMARY STATS   
# Positive141012
# Negative101412
Median Positive3.2%7.7%14.2%
Median Negative-4.3%-8.4%-14.0%
Max Positive12.7%37.8%44.5%
Max Negative-32.8%-25.3%-27.5%

SEC Filings

Expand for More
Report DateFiling DateFiling
03/31/202605/05/202610-Q
12/31/202503/02/202610-K
09/30/202511/10/202510-Q
06/30/202507/29/202510-Q
03/31/202505/09/202510-Q
12/31/202402/27/202510-K
09/30/202411/05/202410-Q
06/30/202408/01/202410-Q
03/31/202405/07/202410-Q
12/31/202302/28/202410-K
09/30/202311/07/202310-Q
06/30/202308/01/202310-Q
03/31/202305/09/202310-Q
12/31/202202/27/202310-K
09/30/202211/02/202210-Q
06/30/202208/08/202210-Q
Collapse to Preview
Report DateFiling DateFiling
03/31/202605/05/202610-Q
12/31/202503/02/202610-K
09/30/202511/10/202510-Q
06/30/202507/29/202510-Q
03/31/202505/09/202510-Q
12/31/202402/27/202510-K
09/30/202411/05/202410-Q
06/30/202408/01/202410-Q
03/31/202405/07/202410-Q
12/31/202302/28/202410-K
09/30/202311/07/202310-Q
06/30/202308/01/202310-Q
03/31/202305/09/202310-Q
12/31/202202/27/202310-K
09/30/202211/02/202210-Q
06/30/202208/08/202210-Q
03/31/202205/02/202210-Q
12/31/202102/25/202210-K
09/30/202110/25/202110-Q
06/30/202107/26/202110-Q
03/31/202105/10/202110-Q
12/31/202003/01/202110-K
09/30/202010/27/202010-Q
06/30/202007/30/202010-Q
03/31/202004/29/202010-Q
12/31/201903/02/202010-K
09/30/201910/28/201910-Q
06/30/201907/29/201910-Q

Recent Forward Guidance

Updated 7/1/2026

Latest: Q1 2026 Earnings Reported 5/5/2026

Forward GuidanceGuidance Change
MetricLowMidHigh% Chg% DeltaChangePrior
2029 Cash Runway 2,030    

Prior: Q4 2025 Earnings Reported 3/2/2026

Forward GuidanceGuidance Change
MetricLowMidHigh% Chg% DeltaChangePrior
2029 Cash Runway     AffirmedGuidance: 2,029 for 2029

Q3 2025 Earnings Reported 11/10/2025

Forward GuidanceGuidance Change
MetricLowMidHigh% Chg% DeltaChangePrior
2025 Patient Dosing Completion 2,025    
2029 Cash Runway 2,029 0.7% RaisedGuidance: 2,027 for 2027

Insider Activity

Updated 7/7/2026
Expand for More
#OwnerTitleHoldingActionFiling DatePriceSharesTransacted
Value
Value of
Held Shares
Form
1Kapusta, Matthew CCEO, Managing DirectorDirectSell707202645.1621,082952,05123,447,999Form
2Abi-Saab, WalidChief Medical OfficerDirectSell702202646.4230,0001,392,6006,881,069Form
3Kaye, JackDirectSell702202646.4421,167983,0301,207,297Form
4Abi-Saab, WalidChief Medical OfficerDirectSell630202646.861,43467,1977,883,492Form
5Kapusta, Matthew CCEO, Managing DirectorDirectSell626202650.0228,7161,436,37425,971,735Form
Collapse to Preview
#OwnerTitleHoldingActionFiling DatePriceSharesTransacted
Value
Value of
Held Shares
Form
1Kapusta, Matthew CCEO, Managing DirectorDirectSell707202645.1621,082952,05123,447,999Form
2Abi-Saab, WalidChief Medical OfficerDirectSell702202646.4230,0001,392,6006,881,069Form
3Kaye, JackDirectSell702202646.4421,167983,0301,207,297Form
4Abi-Saab, WalidChief Medical OfficerDirectSell630202646.861,43467,1977,883,492Form
5Kapusta, Matthew CCEO, Managing DirectorDirectSell626202650.0228,7161,436,37425,971,735Form
6Kapusta, Matthew CCEO, Managing DirectorDirectSell626202650.006,696334,80025,961,350Form
7Kapusta, Matthew CCEO, Managing DirectorDirectSell622202650.0041,6712,083,55026,089,750Form
8Kapusta, Matthew CCEO, Managing DirectorDirectSell622202644.32164,4297,288,06224,768,391Form
9Balachandran, MadhavanDirectSell622202645.0611,685526,4851,948,872Form
10Kaye, JackDirectSell622202642.575,295225,4081,106,650Form
11Balachandran, MadhavanDirectSell617202627.8510,000278,5001,204,624Form
12Potts, JeannetteChief Legal OfficerDirectSell617202627.675,237144,9083,686,917Form
13Meek, David DDirectSell612202627.061,99353,9311,075,554Form
14Gut, RobertDirectSell612202627.062,72673,766872,929Form
15O'Keefe, KylieChief Customer & Strat OfficerDirectSell612202627.0615,936431,2282,945,860Form
16Springhorn, Jeremy PDirectSell612202627.061,99353,9311,170,372Form
17Post, Leonard EDirectSell612202627.061,99353,931960,468Form
18Kaye, JackDirectSell612202627.061,99353,931703,452Form
19Jacques, Rachelle SuzanneDirectSell612202627.061,99353,931917,415Form
20Balachandran, MadhavanDirectSell612202627.061,99353,9311,170,453Form
21Gut, RobertDirectSell612202626.057,552196,720714,647Form
22Kaye, JackDirectSell605202630.012,64579,376613,374Form
23Abi-Saab, WalidChief Medical OfficerDirectSell512202625.0645,0001,127,5004,251,151Form
24Kapusta, Matthew CCEO, Managing DirectorDirectSell30620269.0614,581132,1045,985,561Form
25Abi-Saab, WalidChief Medical OfficerDirectSell30620269.068087,3201,718,401Form
26Potts, JeannetteChief Legal OfficerDirectSell30620269.063,41230,9131,254,656Form
27Klemt, ChristianChief Financial OfficerDirectSell30620269.0612,000108,7202,052,824Form
28Kapusta, Matthew CCEO, Managing DirectorDirectSell30420269.9534,437342,6486,016,158Form
29Klemt, ChristianChief Financial OfficerDirectSell30420269.9512,532124,6931,979,861Form
30Potts, JeannetteChief Legal OfficerDirectSell30420269.958,57885,3511,059,625Form
31Abi-Saab, WalidChief Medical OfficerDirectSell30420269.951,66016,5171,480,331Form
32Kapusta, Matthew CCEO, Managing DirectorDirectSell227202623.8612,378295,33915,248,353Form
33Klemt, ChristianChief Financial OfficerDirectSell227202623.866,217148,3385,046,700Form
34Kaye, JackDirectSell112202627.286,390174,319557,576Form
35Gut, RobertDirectSell1110202527.2631,434856,8811,094,340Form
36Kaye, JackDirectSell1106202530.3438,8101,177,517620,131Form
37Klemt, ChristianChief Financial OfficerDirectSell926202555.0015,000825,00011,975,150Form
38Kapusta, Matthew CCEO, Managing DirectorDirectSell926202541.46226,3169,382,47327,007,588Form
39Klemt, ChristianChief Financial OfficerDirectSell926202540.043,000120,1208,717,909Form
40Abi-Saab, WalidChief Medical OfficerDirectSell701202513.921,46620,4072,094,083Form
Core Cache Last Updated: 7/7/2026