09/07/2024 - uniQure NV: July 2024 Huntington's Disease Program Update AMT-130

Disclaimer

This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to," "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions and the negatives of those terms. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this presentation. Examples of these forward-looking statements include, but are not limited to, statements concerning: our plans to meet with regulatory authorities to discuss the potential for expedited clinical development; the timing of our planned meeting and discussions with regulatory authorities; our ability to continue accumulating long-termpatient data; the potential clinical and functional effects of AMT-130;our plans to continue clinical development of AMT-130;the potential for accelerated regulatory pathways; our use of a natural history cohort as a basis for comparison with respect to the efficacy of AMT-130;our enrollment plans with respect to the third cohort studying AMT-130in combination with immunosuppression and our plans to present safety data from this cohort; the utility of NfL in CSF as an effective biomarker and indicator of clinical severity; and our plans to present further interim analyses. Because these statements are subject to risks and uncertainties, our actual results could differ materially from those expressed in these forward-looking statements. These risks and uncertainties include, among others: risks related to our clinical trials of AMT-130, including the risk that such trials will be unable to demonstrate data sufficient to support further clinical development and the risk that interim data from the trials may not be predictive of later data readouts; risks related to our ability to pursue business development efforts with respect to AMT-130; risks related to our planned interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to regulatory approval; risks related to our use of propensity- weighted external controls in connection with its statistical analysis of clinical outcomes to date, and whether regulatory authorities will accept our approach as a basis for accelerated approval; risks related to our use of nominal p values as a basis for its statistical analyses; whether the measurements that we are evaluating continue to be viewed as robust and sensitive measurements of disease progression; whether RMAT designation or any accelerated pathway, if granted, will lead to regulatory approval; our ability to conduct and fund a Phase III or confirmatory study for AMT-130; our ability to continue to build and maintain the infrastructure and personnel needed to achieve our goals; our effectiveness in managing current and future clinical trials and regulatory processes; our ability to demonstrate the therapeutic benefits of our gene therapy candidates in clinical trials; the continued development and acceptance of gene therapies; our ability to obtain, maintain and protect our intellectual property; and our ability to fund our operations and to raise additional capital as needed and on acceptable terms. These and other risks and uncertainties are described more fully under the heading "Risk Factors" in our periodic filings with the U.S. Securities and Exchange Commission ("SEC"), including in our Annual Report on Form 10-K filed with the SEC on February 28, 2024, and other filings that we make with the SEC from time to time.

Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements and, except as required by law, we assume no obligation to update these forward-looking statements to reflect events that occur or circumstances that exist after the date on which they were made.

Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party sources to be reliable as of the date of this presentation, we have not independently verified, and makes no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third -party sources. Finally, while we believe our own internal research is reliable, such research has not been verified by any independent source.

Opening Remarks

Matt Kapusta

Chief Executive Officer

A promising approach to treat Huntington's disease

AMT-130 is a gene therapy candidate in Phase I/II clinical trials in the US and Europe to investigate the

slowing of disease progression in HD patients with early-to-moderate disease.

Key AMT-130 differentiators:

• One-timeadministration with potentially long-term effects

1. Potential for early therapeutic intervention

• 1. Ability to accumulate long-term clinical data to support efficacy
• Direct delivery and spread across diseased areas of brain
• 1. MoA demonstrated across multiple animal species

• 1. Leverages proprietary miQURE gene silencing platform
• Suppression of highly toxic exon-1 splice isoform in addition to full-lengthmHTT

Abbreviations: HD, Huntington's Disease; miRNA, microRNA.

References: https://www.neuroscientificallychallenged.com/blog/know-your-brain-striatum.Accessed June 2024. Data on file. June 2024.

Key Updates

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

External Comparator & Clinical Measures