uniQure Announces 2022 Financial Results and Highlights Recent Company Progress ~ Achieved U.S. and European Commission marketing approvals of first and only gene therapy for adults with hemophilia B ~ ~ Advancing develo

uniQure Announces 2022 Financial Results

and Highlights Recent Company Progress

~ Achieved U.S. and European Commission marketing

approvals of first and only gene therapy for adults with hemophilia B ~

~ Advancing development of AMT-130 for patients

with Huntington's disease - clinical update from U.S. Phase I/II trial expected in second quarter 2023 ~

~Hosted virtual investor event featuring AMT-260

in refractory temporal lobe epilepsy - expected to enter clinical development in second half of 2023 ~

~Expanded pipeline with in-licensing of AMT-162

for SOD1 ALS - plans to initiate a Phase I/II clinical trial in the second half of 2023 ~

Lexington, MA and Amsterdam, the Netherlands,

February 27, 2023 - 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 2022 and highlighted recent

progress across its business.

"We had a strong ending to 2022 with the

historic approval of the world's first gene therapy for hemophilia B for which we led the multi-year research and clinical development,

and through our partnership with CSL Behring, HEMGENIX will now be available to patients in the United States and European

Union," stated Matt Kapusta, chief executive officer of uniQure. "We are immensely proud

of our efforts that led to this product approval, and of the innovation and leadership in genomic medicine that it represents. At the

same time, we are working with urgency to advance AMT-130, the first AAV gene therapy being clinically investigated in Huntington's

disease, and are on track to provide a clinical update from our U.S. Phase I/II study in the second quarter of 2023. We are also preparing

for the initiation of two new Phase I/II studies in refractory temporal lobe epilepsy and SOD1-ALS in the second half of this year, and

readying for the submission of an investigational new drug application in Fabry disease in 2023."

"Throughout this progress, we have established

cutting-edge capabilities in the field of genetic medicines - from how we discover and develop our gene therapies, to how we successfully

navigate complex clinical and regulatory pathways, to how we manufacture these novel gene therapies globally for clinical and commercial

usage," said Mr. Kapusta. "We believe these capabilities position us well for continued growth and success as we look

forward to developing more life-altering gene therapy products for patients in need."

All 26 patients have been enrolled in the first two

cohorts of the randomized, controlled and double-blinded U.S. Phase I/II study of AMT-130, including 10 patients in the lower-dose cohort

(6 treated patients and 4 control patients) and 16 patients in the higher-dose cohort (10 treated patients and 6 control patients). The

6 control patients in the higher-dose cohort will have the option to cross over to treatment if they meet the study's eligibility

criteria. In the first quarter of 2023, a second control patient was crossed over to treatment and received the higher dose of AMT-130.

Upcoming Investor Events

Financial Highlights

Cash position: As of December 31,

2022, the Company held cash and cash equivalents and investment securities of $392.8 million, compared to $556.3 million as of December 31,

2021. As of December 31, 2022, the Company held investments in debt securities of $164.8 million, compared to nil as of December 31,

2021. The investments in debt securities have remaining maturities ranging from three to 14 months.

Revenue for the year ended December 31, 2022 was $106.5 million, compared to $524.0 million in the same period in 2021. In 2022,

the Company recognized $100.0 million of license revenue related to a milestone payment the Company expects to receive following the first

sale of HEMGENIX in the U.S. In 2021, the Company recognized $462.4 million of license revenue upon

closing of the CSL Behring transaction in May 2021 as well as $55.0 million of license revenue for milestone payments related to

CSL Behring's BLA and MAA submissions in the first months of 2022.

R&D expenses: Research and development

expenses were $197.6 million for the year ended December 31, 2022, compared to $143.5 million during the same period in 2021. The

increase was primarily related to advancing the clinical development of the Company's Huntington's disease gene therapy program,

the preclinical development of the temporal lobe epilepsy program (AMT-260), an increase in personnel and contractor related expenses

to support the growth of the Company, and contractual payments owed to licensors upon FDA approval of HEMGENIX and a valid patent claim

granted within the EU.

SG&A expenses: Selling, general and

administrative expenses were $55.1 million for the year ended December 31, 2022, compared to $56.3 million during the same period

in 2021. The increase in personnel and contractor related expenses to support the growth of the Company was offset by a reduction in professional

and financial advisory fees we had incurred in 2021 related to the closing of the CSL Behring transaction.

Other non-operating items, net:

Other non-operating income, net was income of

$14.9 million for the year ended December 31, 2022, compared to other non-operating income, net of $22.2 million for the same period

in 2021. The decrease in other non-operating income, net was primarily related to a decrease in net foreign currency gains of $6.4 million,

an increase of $3.8 million in interest expense related to the long-term debt with Hercules Capital, Inc. and a $2.9 million increase

in other non-operating gains as a result of releasing a financial liability.

The net loss for the year ended December 31,

2022, was $126.8 million, or $2.71 basic and diluted loss per ordinary share, compared to $329.6 million net income for the same period

in 2021, or $7.17 basic net income per ordinary share and $7.04 diluted net income per ordinary share.

HEMGENIX is a gene therapy that

reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the

deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector

carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x

more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person's

own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.

HEMGENIX is a registered trademark

uniQure is delivering on the promise of gene therapy

- single treatments with potentially curative results. The recent approvals of our gene therapy for hemophilia B - an historic

achievement based on more than a decade of research and clinical development - represents a major milestone in the field of genomic

medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology

platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's

disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. www.uniQure.com

uniQure Forward-Looking Statements

This 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, statements about whether a third cohort of patients in our

AMT-130 clinical trial for Huntington's Disease will begin enrollment in the U.S. in the second half of 2023, whether the Company

will announce one to two-years of follow up data from the U.S. Phase I/II clinical study of AMT-130 in the second quarter of 2023, whether

clinical data from the lower-dose cohort for AMT-130 is expected to be presented in the second half of 2023, whether the Company will

submit an investigational new drug application for Fabry disease in 2023, whether the company will initiate a Phase I/II clinical study

of AMT-162 for the treatment of ALS in the second half of 2023, and whether the Company will submit an investigational new drug application

or initiate a Phase I/II clinical study of AMT-260 for rTLE in the second half of 2023. 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 regulatory approval and commercial launch of HEMGENIX , 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 27,

2023. 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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