uniQure Announces 2020 Financial Results and Highlights Recent Company Progress ~ Six patient procedures now completed in Phase I/II for Huntington's disease, including two procedures conducted in February 2021, with ful

uniQure Announces 2020 Financial Results

and Highlights Recent Company Progress

procedures now completed in Phase I/II for Huntington's disease, including two procedures conducted in February 2021,

with full enrollment of first cohort expected mid-2021 ~

in HOPE-B pivotal study to complete 52-week follow-up visit before end of the first quarter of 2021, with top-line data to be

presented in the second quarter of 2021 ~

the HOPE-B safety event expected to be completed and submitted to the FDA before end of the first quarter of 2021 ~

Development Day to be held mid-2021 to announce expanded pipeline and platform advancements ~

Lexington, MA and Amsterdam, the Netherlands,

March 1, 2021 - 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 2020 and highlighted recent progress across its

"Despite unprecedented challenges

in 2020, the uniQure team demonstrated extraordinary resiliency and dedication to the patients we serve, qualities which have

defined our company for more than two decades," stated Matt Kapusta, chief executive officer at uniQure. "I'm

very proud of uniQure's accomplishments, including completing patient dosing in the HOPE-B pivotal trial, presenting positive

26-week data on 54 patients that met the study's first primary endpoint, and announcing a landmark transaction with CSL

Behring for the global commercialization of etranacogene dezaparvovec, which is expected to close in the second quarter of 2021

following the U.S. antitrust review. We also became the first company to initiate the clinical investigation of an AAV gene therapy

for Huntington's disease and have now enrolled six patients in the study."

"Looking at 2021, we are focused

on important catalysts as we prepare the filings for regulatory approval of etranacogene dezaparvovec and advance our Phase I/II

study of AMT-130 in Huntington's disease, with early biomarker data on the initial patients expected as early as the end

of this year. We are also making investments to expand our pipeline of promising gene therapies and further strengthen our AAV

manufacturing platform. We look forward to hosting a Research and Development Day mid-year during which we will unveil new research

programs and other technology initiatives."

Recent Company Progress

Upcoming Investor Events (each to be

conducted virtually)

Financial Highlights

Cash Position: As of December 31,

2020, the Company held cash and cash equivalents of $244.9 million, compared to $377.8 million as of December 31, 2019.

Revenues: Revenue for the year

ended December 31, 2020 was $37.5 million, compared to $7.3 million during the same period in 2019. The change was

primarily related to an increase in non-cash license revenue that we recognized as of the December 1, 2020 effective date

of the amended Bristol-Myers Squibb collaboration and license agreement, as well as from revenue that we recognized in December 2020

following achievement of a research milestone for one of the four Collaboration Targets.

R&D Expenses: Research and

development expenses were $122.4 million for the year ended December 31, 2020, compared to $94.7 million during the same

period in 2019. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene

dezaparvovec and AMT-130, increased share-based compensation, increased license expenses and the recruitment of personnel to support

the development of our product candidates.

SG&A Expenses: Selling, general

and administrative expenses were $42.6 million for the year ended December 31, 2020, compared to $33.5 million during the

same period in 2019. The change was primarily related to increases in personnel and consulting expenses to support our growth,

as well as increases in professional fees.

Other non-operating items, net:

Other expenses were $16.0 million for the year ended December 31, 2020, compared to other expenses of $3.1 million during

the same period in 2019 primarily related to foreign exchange results related to changes in the exchange rates between the Euro

and the U.S. Dollar.

Net Loss: The net loss for the

year ended December 31, 2020 was $125.0 million, or $2.81 loss per share, compared to $124.2 million, or $3.11 loss per share

during the same period in 2019.

uniQure 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, Fabry disease, spinocerebellar ataxia Type 3 and other 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," "estimate," "expect," "goal,"

"intend," "look forward to", "may," "plan," "potential," "predict,"

"project," "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, uniQure's expectations about its cash runway,

the timing of submitting data to the FDA or providing an update related to the investigation of any safety event related to the

HOPE-B study, the enrollment of patients in, or Data Safety Monitoring Board review of, our Phase I/II gene therapy clinical trial

of AMT-130 in Huntington's disease, the timing of the announcement of data in our Phase I/II gene therapy clinical trial

of AMT-130, the timing of the announcement of data in our HOPE-B pivotal trial of etranacogene dezaparvovec, the timing of filing

for regulatory approval of etranacogene dezaparvovec, the timing of regulatory approval of our agreement with CSL Behring, the

potential financial compensation we may be paid pursuant to our agreement with CSL Behring, our plans to advance or expand our

pipeline, accelerate research, identify business development opportunities, invest in technology, or scale our manufacturing capabilities.

uniQure'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 ongoing COVID-19 pandemic on our Company and the wider

economy and health care system, our Commercialization and License Agreement with CSL Behring, the regulatory approval of that

transaction, 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 uniQure's periodic securities filings, including its Annual Report on Form 10-K

filed March 1, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking

statements, and uniQure assumes no obligation to update these forward-looking statements, even if new information becomes available

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