uniQure (QURE) risk factors, FY2025 10-K

Risks Related to Our Business and the Development of Our Clinical Product Candidates

1. 01We have invested a significant portion of our development efforts and financial resources in the development of our lead clinical product candidate, AMT-130 for the treatment of Huntington’s diseaseNew
2. 02We have encountered and may encounter future delays in and impediments to the progress of our clinical trials or fail to demonstrate the safety and efficacy of our product candidates29% rewritten
3. 03Our progress in early-stage clinical trials may not be predictive of long-term efficacy in late-stage clinical trials, and our progress in trials for one product candidate may not be predictive of progress in trials for other product candidates
4. 04Interim, topline or preliminary results from our clinical trials may change as more data become available, as such data are subject to regulatory audit and verification procedures, and/or regulatory review, which could result in material changes in the final results and conclusions59% rewritten
5. 05Data analyses conducted either on a post-hoc basis or with a pre-specified statistical analysis plan using external, historical controls may not be accepted as a basis for regulatory approval50% rewritten
6. 06We are making use of exploratory biomarkers and other data that are not scientifically validated, and our reliance on these data may lead us to direct our resources inefficiently
7. 07We may not be successful in our efforts to use our gene therapy technology platform to build a pipeline of additional product candidates or otherwise leverage our research and technology to remain competitive
8. 08We may not be successful in obtaining rights from external parties to new product candidates and key technologies, or in securing partnerships to support the development or commercialization of our product candidates
9. 09Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may damage the public perception of our product candidates or adversely affect our ability to conduct our business or obtain marketing approvals for our product candidates
10. 10We face substantial competition, and others may discover, develop, or commercialize competing products before or more successfully than we do

Risks Related to Regulatory Approval of Our Products

1. 11We cannot predict when or if we will obtain marketing approval to commercialize our product candidates66% rewritten
2. 12The risks associated with the marketing approval process are heightened by the status of our products as gene therapies24% rewritten
3. 13As appropriate, we intend to seek available periods of regulatory exclusivity for our product candidates. However, there is no guarantee that we will be granted these periods of regulatory exclusivity or that we will be able to maintain these periods of exclusivity
4. 14We face uncertainty related to insurance coverage of, and pricing and reimbursement for, our product candidates for which we may receive marketing approval
5. 15Due to the generally limited addressable market for our target indications and the potential for our therapies to offer therapeutic benefit in a single administration, we face uncertainty related to pricing and reimbursement of our product candidates
6. 16If any of our product candidates receive regulatory approval, we and/or our partners will be subject to extensive regulatory requirements. Failure to fulfill and comply with the applicable regulatory requirements could result in regulatory enforcement actions that would be detrimental to our business27% rewritten

Risks Related to Commercialization

1. 17If we are unable to successfully commercialize our product candidates or experience significant delays in doing so, including as a result of a BLA or other key regulatory filings being delayed or rejected, our business could be materially harmed
2. 18Any approved gene therapy we seek to commercialize may fail to achieve the degree of market acceptance by physicians, patients, third-party payers and others in the medical community necessary for commercial success
3. 19The affected populations for our gene therapies may be smaller than we or third parties currently project, which may affect the size of our addressable markets
4. 20If the market opportunities for our product candidates are smaller than we believe they are, our product revenues may be adversely affected, and our business may suffer
5. 21Ethical, legal, and social issues associated with genetic testing may reduce demand for any gene therapy products for which we obtain marketing approval
6. 22If we obtain approval to commercialize any of our product candidates outside of the U.S., a variety of risks associated with international operations could materially adversely affect our business

Risks Related to Manufacturing and Our Dependence on Third Parties

1. 23Gene therapies are complex, expensive and difficult to manufacture. Genezen or any third-party manufacturer that we engage could experience capacity, production or technology transfer challenges that could result in delays in our development or commercialization schedules or otherwise adversely affect our business
2. 24Our use of viruses, chemicals and other potentially hazardous materials requires us and our contract manufacturers to comply with regulatory requirements and exposes us to significant potential liabilities
3. 25Our business may be adversely affected if we or third-party manufacturers are unable to validate our manufacturing processes and methods or develop new processes and methods to meet our product supply needs and obligations
4. 26We rely on third parties for important aspects of our development programs. If these parties do not perform successfully or if we are unable to enter into or maintain key collaborations or other contractual arrangements, our business could be adversely affected

Risks Related to Our Intellectual Property

1. 27If we fail to comply with our obligations in our intellectual property licenses with third parties, we could lose rights that are important to our business
2. 28If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, our ability to successfully commercialize our products may be impaired
3. 29We may become involved in lawsuits to protect or enforce our patents or other intellectual property, or third parties may assert their intellectual property rights against us, which could be expensive, time consuming and unsuccessful
4. 30If we are unable to protect the confidentiality of our proprietary information and know-how, the value of our technology and products could be adversely affected
5. 31Our reliance on third parties may require us to share our trade secrets, which could increase the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed
6. 32Intellectual property rights do not necessarily address all potential threats to our competitive advantage

Risks Related to Our Financial Position and Need for Additional Capital

1. 33We had net losses in the years ended December 31, 2025 and 2024, have incurred significant losses in previous years and expect to incur losses in the future, and may never achieve or maintain profitability39% rewritten
2. 34Our business development and strategic initiatives, acquisitions, partnerships, collaborations or other transactions, may not achieve their intended benefits or goals and may result in additional risks to our business
3. 35Actions that we have taken or may take in the future to restructure our business in alignment with our strategic priorities may not be as effective as anticipated, may not result in cost savings to us and could disrupt our business62% rewritten
4. 36Our existing and any future indebtedness could adversely affect our ability to operate our business29% rewritten

Risks Related to Other Legal Compliance Matters

1. 37Our relationships with employees, customers and third parties are subject to applicable laws and regulations, the non-compliance of any of which could have a material adverse effect on our business, financial condition, and results of operations29% rewritten
2. 38Product liability lawsuits could cause us to incur substantial liabilities and to limit commercialization of our therapies
3. 39Healthcare legislative and regulatory reform measures may have a material adverse effect on our financial operations
4. 40Our future growth may depend, in part, on our ability to penetrate markets outside of the U.S. and Europe where we would be subject to additional regulatory burdens and other risks and uncertainties
5. 41There may be future changes in legal and regulatory requirements or standards that may materially impact our results of operations23% rewritten

Risks Related to Our Ordinary Shares

1. 42The market price of our ordinary shares has been and may in the future be volatile and fluctuate substantially28% rewritten
2. 43Our directors, executive officers, and major shareholders, if they choose to act together, will continue to have a significant degree of control with respect to matters submitted to shareholders for approval
3. 44The rights and responsibilities of our shareholders and directors are governed by Dutch law and differ in some important respects from the rights and responsibilities of shareholders under U.S. law50% rewritten
4. 45Provisions of our articles of association or Dutch corporate law might deter acquisition bids for us that might otherwise be considered favorable and could make it more difficult to replace our board, ensuring continuity and stability in corporate governance in line with Dutch law and case law requirements65% rewritten
5. 46We do not expect to pay dividends in the foreseeable future. Accordingly, any returns on an investment in our ordinary shares will likely depend entirely upon any future appreciation in the price of our ordinary shares, which is uncertain
6. 47Any U.S. or other foreign judgments may be difficult to enforce against us in the Netherlands84% rewritten
7. 48Also, service of process against non-residents of the U.S. can in principle (absent, for example, a valid choice of domicile) not be affected in the U.SNew
8. 49We are not obligated to, and do not, comply with all best practice provisions of the Dutch Corporate Governance Code
9. 50We expect to become subject to the Dutch large company regime, which we expect will impact our governance structure, including how the members of our board are appointed and dismissedNew
10. 51Shareholders may not be able to exercise pre-emption rights and, as a result, may experience substantial dilution upon future issuances of ordinary shares or grants of rights to subscribe for sharesNew

General Risks

1. 52Our future success depends on our ability to retain key executives, technical staff, and other employees and to attract, retain and motivate qualified personnel
2. 53We may be adversely affected by unstable market and economic conditions and potential macroeconomic effects, such as inflation, new or increased tariffs and higher interest rates, which may negatively impact our business, financial condition and share price
3. 54If we fail to maintain an effective system of internal controls, we may be unable to accurately report our results of operations or prevent fraud or fail to meet our reporting obligations, and investor confidence and the market price of our ordinary shares may be materially and adversely affected
4. 55Our internal computer systems, or those of our collaborators, third-party vendors, contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our business and development programs
5. 56If securities or industry analysts do not publish, cease to publish or publish inaccurate or unfavorable research about our business, our share price and trading volume could decline
6. 57If we do not achieve our projected development and financial goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and, as a result, our share price may decline
7. 58Environmental sustainability and social initiatives that we undertake may impose additional costs on our business and expose us to new risks