Here are 1-3 brief analogies for uniQure (QURE):

• Like Moderna, but focused on developing one-time gene therapies for genetic diseases rather than mRNA vaccines.
• Like Vertex Pharmaceuticals, but specifically using gene therapy to provide potentially curative treatments for genetic diseases like hemophilia.

• HEMGENIX® (etranacogene dezaparvovec): A one-time gene therapy approved for the treatment of adults with severe to moderately severe hemophilia B.
• AMT-130: An investigational gene therapy currently in clinical trials for the treatment of Huntington's disease.

uniQure (QURE) is a biotechnology company focused on gene therapies. Its business model primarily involves the development and licensing of its therapeutic products to other pharmaceutical companies for commercialization.

Therefore, uniQure's major customer is another company:

• CSL Behring (a subsidiary of CSL Limited, ASX: CSL)

CSL Behring is uniQure's partner for HEMGENIX® (etranacogene dezaparvovec), a gene therapy for severe hemophilia B. uniQure licensed the global commercialization rights for HEMGENIX to CSL Behring, and its revenue from this product largely consists of milestone payments and royalties received from CSL Behring.

• Lonza Group AG (SIX: LONN)

Matthew Kapusta, Chief Executive Officer

Mr. Kapusta has served as uniQure's Chief Executive Officer since December 2016 and is also a member of the company's Board of Directors. He initially joined uniQure as Chief Financial Officer in January 2015. Prior to uniQure, he was Senior Vice President at AngioDynamics from 2011 to 2014, where he was responsible for corporate development, strategic planning, and national accounts. Before AngioDynamics, he served as Vice President, Finance for Smith & Nephew Orthopaedics. Mr. Kapusta's career also includes over a decade of investment banking experience focused on emerging life sciences companies, having held positions at Collins Stewart as Managing Director, Healthcare Investment Banking, and at Wells Fargo Securities, Robertson Stephens, and PaineWebber. He has served as a director of Decibel Therapeutics (Nasdaq: DBTX) since March 2023.

Christian Klemt, Chief Financial Officer

Mr. Klemt was appointed Chief Financial Officer of uniQure in June 2021. He has been with uniQure since 2015 and previously served as Chief Accounting Officer starting in August 2017 and as General Manager of the Amsterdam site in 2020. During his tenure, he has transformed the finance and accounting functions, established financial planning and analysis, led tax and treasury initiatives, and overseen IT development. Before joining uniQure, Mr. Klemt was Finance Director at CGGVeritas from 2009 to 2015. He also served as Group Finance Manager of Basell Polyolefins N.V. from 2005 to 2007 and played a role in acquisition projects following its merger with Lyondell Inc. in 2007. Mr. Klemt qualified as a German Certified Public Accountant and Tax Advisor while working at KPMG from 1999 to 2005.

Walid Abi-Saab, M.D., Chief Medical Officer

Dr. Abi-Saab joined uniQure as Chief Medical Officer in June 2023, overseeing clinical research and development, regulatory affairs, and program management. Prior to uniQure, he was Chief Medical Officer at Galapagos from 2017 to 2022, where he expanded the development organization and led successful Phase 3 studies for filgotinib. He has also held leadership positions at Shire (now part of Takeda Pharmaceutical Co.), Novartis, Abbott Laboratories, and Pfizer. Dr. Abi-Saab also served as a resident, research fellow, and assistant professor at Yale University School of Medicine.

Richard Porter, Ph.D., Chief Business and Scientific Officer

Dr. Porter was named Chief Business and Scientific Officer in October 2023. In this role, he is responsible for research, non-clinical and vector development, business development, and product planning. Dr. Porter joined uniQure as General Manager of the Corlieve subsidiary following uniQure's acquisition of Corlieve Therapeutics in July 2021. He was the founder and CEO of Corlieve Therapeutics, which was founded in November 2019. He previously served as Chief Operating Officer of Therachon until its acquisition by Pfizer. Dr. Porter's experience also includes roles at Roche as Global Head of Scientific Business Strategy and Operations for Neuroscience Ophthalmology and Rare Diseases, and as Product General Manager of the Emerging Business Unit at Shire Pharmaceuticals.

Jeannette Potts, Ph.D., J.D., Chief Legal & Compliance Officer

Dr. Potts joined uniQure as Chief Legal and Compliance Officer in May 2023. She brings over 25 years of global legal, management, and transactional experience in the biopharmaceutical industry. Most recently, she was Senior Vice President, General Counsel & Corporate Secretary at Forma Therapeutics, which was acquired by Novo Nordisk in 2022. At Forma, she built and led the legal, IP, and compliance team and played key roles in corporate financings and business development transactions. Before Forma, Dr. Potts served as Vice President, Legal and Head Counsel, Research and Development, at Takeda Pharmaceuticals, and prior to that, Vice President, Legal at Millennium Pharmaceuticals before its acquisition by Takeda.

1. Regulatory Uncertainty for AMT-130: The most significant risk stems from the regulatory pathway for AMT-130, its investigational gene therapy for Huntington's disease. The U.S. Food and Drug Administration (FDA) recently indicated that current Phase I/II study data for AMT-130 may not be sufficient to support a Biologics License Application (BLA) submission for accelerated approval, creating uncertainty around the timing of a potential submission and approval. This regulatory setback has led to a substantial decline in uniQure's stock price and is considered a critical "binary event" given the importance of AMT-130 to the company's future.
2. Financial Health and Funding: uniQure continues to face significant financial challenges, reporting deepening losses, high research and development (R&D) costs, and negative operating and net margins. Despite recent financing activities that have improved its cash position, the company's Altman Z-Score indicates financial distress and a potential risk of bankruptcy within two years. The ability to fund ongoing operations and potentially raise additional capital if regulatory timelines are delayed is a persistent concern.
3. Intense Competition and Market Volatility: The biotechnology sector, particularly gene therapy, is characterized by rapid technological advancements, intense competition, and high market volatility. uniQure operates in a highly dynamic environment where new players and innovations can quickly alter market dynamics, requiring continuous innovation to maintain a competitive edge. The company's stock price has experienced significant fluctuations, which can impact investor confidence and its ability to raise capital in the future.

uniQure's main products target Hemophilia B and Huntington's disease.

HEMGENIX (etranacogene dezaparvovec) for Hemophilia B

The addressable market for Hemophilia B gene therapy is substantial. The global Hemophilia B gene therapy market size was valued at approximately USD 0.51 billion in 2023 and is projected to reach USD 3.43 billion by 2032, with a compound annual growth rate (CAGR) of around 23.60% from 2024 to 2032. Another estimate for the global Hemophilia B gene therapy market projects a valuation of USD 5.94 billion by 2032, with a CAGR of 34.1% from 2023. The broader Hemophilia B treatment market across the 7 Major Markets (7MM), which includes the United States, Germany, Spain, Italy, France, the United Kingdom, and Japan, was approximately USD 3.4 billion in 2023, with the United States being the largest contributor. The global Hemophilia B treatment market is estimated to reach a valuation of approximately USD 4.29 billion in 2024 and is expected to grow at a CAGR of 7.6% during the forecast period of 2024 to 2034.

AMT-130 for Huntington's Disease

The addressable market for Huntington's disease treatments is also significant. The global Huntington's disease treatment market size was estimated at USD 500 million in 2024 and is projected to reach USD 1.87 billion by 2030, growing at a CAGR of 23.8% from 2025 to 2030. Another source states the global market size was USD 578.9 million in 2024 and is estimated to reach USD 2.44 billion by 2033, exhibiting a CAGR of 17.4% from 2025 to 2033. North America currently holds the largest share of this market, accounting for approximately 40.0% in 2024. The Huntington's disease treatment market size was valued at USD 658.31 million in 2025 and is likely to surpass USD 1.69 billion by 2035, growing at a CAGR of more than 9.9% between 2026 and 2035.

Other Products

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uniQure (QURE) is expected to drive future revenue growth over the next 2-3 years through several key initiatives:

1. Commercial Launch and Sales of AMT-130 for Huntington's Disease: The most significant anticipated driver is the potential commercial launch of AMT-130, a gene therapy for Huntington's disease (HD). uniQure plans to submit a Biologics License Application (BLA) to the FDA in the first quarter of 2026, following positive top-line data from its Phase 1/2 study, which demonstrated a statistically significant slowing of disease progression. Analysts project peak revenues for AMT-130 could reach approximately $3 billion, positioning it as a potentially first-in-class disease-modifying therapy for HD, addressing a high unmet medical need.
2. Advancement and Potential Commercialization of Other Pipeline Assets: uniQure is progressing a focused pipeline of innovative gene therapies beyond Huntington's disease. Key programs include AMT-260 for refractory mesial temporal lobe epilepsy, which has shown encouraging early clinical signals, and AMT-191 for Fabry disease, for which initial safety and efficacy data are expected. Further clinical development, potential regulatory approvals, and eventual commercialization of these and other early-stage candidates like AMT-162 for SOD1-ALS and AMT-240 for autosomal dominant Alzheimer's disease would contribute to future revenue diversification.
3. Milestone Payments from Hemgenix: Although uniQure previously sold a portion of its royalty rights from Hemgenix (etranacogene dezaparvovec-drbl) net sales for hemophilia B, the company retains eligibility for substantial contractual milestone payments from CSL Behring, the licensee. These milestones, potentially totaling up to $1.5 billion, are contingent on the ongoing commercial and regulatory progress of Hemgenix in various territories.

Share Issuance

• In September 2025, uniQure completed an upsized public offering, generating approximately $345 million in gross proceeds from the sale of 6,736,841 ordinary shares and pre-funded warrants for 526,316 shares.
• In September 2025, the company announced the pricing of a $300 million public offering, which included 5,789,473 ordinary shares and pre-funded warrants for 526,316 shares.
• During January and February 2025, uniQure raised $75 million through a public offering of 4.4 million shares at $17 per share, and an additional $10.6 million in net proceeds from underwriters' option exercise for 0.7 million shares.

Inbound Investments

• In September 2025, uniQure secured a $175 million non-dilutive senior secured term loan facility from Hercules Capital, Inc., which included refinancing an existing $50 million debt and providing access to an additional $125 million.
• The additional $125 million in non-dilutive funding consists of a $100 million tranche contingent on regulatory and financial milestones for AMT-130, and a $25 million tranche subject to Hercules' approval.
• As of November 10, 2025, the liability from the HEMGENIX royalty financing stood at $469.1 million.

Capital Expenditures

• For the trailing twelve months ended June 2025, uniQure reported capital expenditures of approximately $-9.13 million.
• For the three months ended June 2025, capital expenditures were approximately $-0.26 million.
• These expenditures are primarily focused on acquiring or upgrading physical assets like property, industrial buildings, or equipment.