uniQure Announces Third Quarter 2022 Financial Results and Highlights Recent Company Progress ~ Patient enrollment at the higher dose of AMT-130 to resume in European Phase Ib/II study following Data Safety Monitoring Bo

Announces Third Quarter 2022 Financial Results

Highlights Recent Company Progress

Patient enrollment at the higher dose of AMT-130 to resume in European Phase Ib/II study following

Data Safety Monitoring Board recommendation

and European pre-approval inspections of Company's Lexington, MA manufacturing site completed with

EMA issuing Good Manufacturing

Virtual investor event focused on next pipeline program, AMT-260 investigational gene therapy for

refractory temporal lobe epilepsy,

to be held on Tuesday, November 29, 2022 ~

MA and Amsterdam, the Netherlands, November 2, 2022 - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing

transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2022 and

highlighted recent progress across its business.

are pleased that following a comprehensive review of all available safety, biomarker and imaging data, the Data Safety Monitoring Board

(DSMB) has recommended that we resume patient enrollment at the higher dose in the ongoing Phase Ib/II study evaluating AMT-130 in Huntington's

disease, " stated Matt Kapusta, chief executive officer of uniQure. "Patient safety will always be our utmost priority,

and we are grateful for the close collaboration between our clinical team, study investigators and most importantly, the patients, all

of whom were instrumental in completing our comprehensive investigations. We believe that AMT-130 has the potential to provide a positive

impact for patients with this devastating disease for which there is no currently approved treatment. We look forward to finishing patient

enrollment in the higher-dose cohort of the European study in the first half of 2023 and remain on track to announce data from the U.S.

Phase I/II study in the second quarter of 2023."

the third quarter, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) completed site inspections of

our Lexington, Massachusetts gene therapy manufacturing facility, and we have since received good manufacturing practice (GMP) certification

from the European authorities. This is an important milestone as we seek to establish industry-leading commercial gene therapy manufacturing

capabilities. Our partner, CSL Behring, continues to interact with U.S. and European health authorities as they complete their review

of the marketing applications for etranacogene dezaparvovec and, assuming approval, we look forward to partnering with our colleagues

at CSL Behring to bring this potentially transformational treatment option to people living with hemophilia B."

position: As of September 30, 2022, the Company held cash and cash equivalents of $440.3 million, compared to $556.3 million as of

Collaboration revenue for the three months ended September 30, 2022, was $1.4 million, compared to collaboration revenue of $2.0

million for the same period in 2021.

of contract manufacturing: Cost of contract manufacturing for the three months ended September 30, 2022, was $0.9 million compared

to nil for the same period in 2021. Costs incurred in 2022 related to the manufacture of etranacogene dezaparvovec for CSL Behring.

expenses: Research and development expenses were $48.1 million for the three months ended September 30, 2022, compared to $36.4 million

during the same period in 2021. The increase was primarily related to an increase in the fair value of contingent consideration associated

with the acquisition of Corlieve Therapeutics SAS, the preclinical development of temporal lobe epilepsy (AMT-260), an increase in facility-related

expenses and an increase in disposable costs.

expenses: Selling, general and administrative expenses were $13.3 million for the three months ended September 30, 2022, compared

to $12.0 million during the same period in 2021. The increase was primarily related to the recruitment of personnel and contractor-related

non-operating items, net:

non-operating income, net was income of $11.3 million for the three months ended September 30, 2022, compared to other non-operating

income, net of $8.6 million for the same period in 2021. The increase in other non-operating income, net was primarily related to an

increase in net foreign currency gains of $3.9 million partially offset by an increase in interest expense related to the long-term debt

with Hercules Capital, Inc.

net loss for the three months ended September 30, 2022, was $47.9 million, or $1.02 basic and diluted loss per ordinary share, compared

to $36.5 million net loss for the same period in 2021, or $0.79 basic and diluted loss per ordinary share.

is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular

and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia

B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases. www.uniQure.com

Forward-Looking Statements

press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements,

which are often indicated by terms such as "anticipate," "believe," "could," "establish," "estimate,"

"expect," "goal," "intend," "look forward to", "may," "plan," "potential,"

"predict," "project," "seek," "should," "will," "would" and similar expressions.

Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the

date of this press release. These forward-looking statements include, but are not limited to, whether we will announce data from the

U.S. Phase I/II study in the second quarter of 2023, whether we are able to bring AMT-061 to people living with hemophilia B and whether

the treatment will be transformational, whether we will resume patient dosing in the open-label European study [in the fourth quarter

of 2022] or complete enrollment in the first half of 2023, whether we will announce one to two-years of follow up data from the U.S.

Phase I/II study in the second quarter of 2023, whether we will file an IND in 2023 for our rTLE program or for our Fabry program. The

Company's actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including,

without limitation, risks associated with the impact of the postponement in our clinical trial for Huntington's disease, the impact

of financial and geopolitical events on our Company and the wider economy and health care system, our Commercialization and License Agreement

with CSL Behring, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization

and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors"

in the Company's periodic securities filings, including its Annual Report on Form 10-K filed February 25, 2022. Given these risks,

uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no

obligation to update these forward-looking statements, even if new information becomes available in the future.

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