uniQure Announces First Quarter 2021 Financial Results and Highlights Recent Company Progress ~ 52-week follow-up data from HOPE-B pivotal study expected to be presented later this quarter ~ ~ Announced closing of the gl

uniQure Announces First Quarter 2021 Financial

Results and Highlights Recent Company Progress

~ 52-week follow-up data from HOPE-B pivotal

study expected to be presented later this quarter ~

closing of the global commercialization and license agreement with CSL Behring for hemophilia B gene therapy ~

~ Completed enrollment of first dose cohort

of U.S. Phase I/II clinical trial of AMT-130 for Huntington's disease, with initiation of second dose cohort and European Phase

Ib/II expected in second half of 2021 ~

~ Pierre Caloz to be appointed Chief Operating

~ Research & Development Day to be held

Tuesday, June 22, 2021 ~

MA and Amsterdam, the Netherlands, May 10, 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 the first quarter of 2021 and highlighted recent progress across its business.

made great strides with our clinical trial execution across both our hemophilia and Huntington's disease programs, with all patients

in the HOPE-B pivotal trial completing their 52-week follow-up visits and all ten patients in the first dose cohort of our U.S. clinical

trial in Huntington's disease completing their procedures," stated Matt

Kapusta, chief executive officer at uniQure. "Looking at important near-terms catalysts, we

look forward to announcing top-line, 52-week follow-up data from the HOPE-B pivotal trial, initiating our collaboration with CSL Behring

and holding an R&D Day to provide updates on the research pipeline, including new product candidates," he added. "In

the second half of the year, we expect to begin patient enrollment in the second cohort of AMT-130 and to initiate our open-label European

study of AMT-130. Later this year, we expect to submit the BLA for etranacogene dezaparvovec and announce early data on the first four

patients in the Phase I/II study of AMT-130."

Recent Company Progress

Upcoming Investor Events (each to be conducted

Financial Highlights

Cash Position: As of March 31, 2021, the

Company held cash and cash equivalents of $260.8 million, compared to $244.9 million as of December 31, 2020. In January 2021, the Company

and Hercules entered into an amended debt facility agreement increasing the aggregate principle to up to $135.0 million, of which the

Company drew down an additional $35.0 million for a total of $70.0 million outstanding under the facility as of March 31, 2021. In March

2021, the Company entered into an Open Market Sale Agreement with SVB Leerink LLC providing for the sale of up to $200.0 million of its

ordinary shares from time to time in at-the-market' offerings under a shelf registration statement. Through March 31, 2021,

the Company sold 859,885 ordinary shares pursuant to this agreement for gross proceeds of approximately $28.7 million.

Revenues: Revenue for the three months

ended March 31, 2021 was $0.5 million, compared to $0.1 million during the same period in 2020.

R&D Expenses: Research and development

expenses were $32.7 million for the three months ended March 31, 2021, compared to $26.0 million during the same period in 2020. The change

was primarily related to increased activities associated with our ongoing clinical studies of Huntington's disease, increased activity

in programs in preclinical development and the recruitment of personnel to support the development of our product candidates.

SG&A Expenses: Selling, general and

administrative expenses were $12.4 million for the three months ended March 31, 2021, compared to $9.1 million during the same period

in 2020. The change was primarily related to increases in personnel and consulting expenses to support our growth, increases in professional

fees, and increased share-based compensation.

Other non-operating items, net: Other income,

net was $3.1 million for the three months ended March 31, 2021, compared to other income, net of $6.5 million during the same period in

2020. The decrease is primarily due to the termination of the Bristol-Myers Squibb warrants in December 2020, resulting in no recognition

of changes in fair value of the derivative financial liability in the current period compared to a recognized in the same period in 2020.

Net Loss: The net loss for the three months

ended March 31, 2021 was $41.6 million, or $0.91 loss per share, compared to $28.0 million, or $0.63 loss per share during the same period

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

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 its plans to announce data from the HOPE-B pivotal trial, patient

enrollment in the second cohort of AMT-130, the initiation of an open-label European study of AMT-130, the announcement of data on the

first four patients in the Phase I/II study of AMT-130, the submission of the regulatory filing for marketing approval of etranacogene

dezaparvovec, the employment of a Chief Operating Officer, the potential financial compensation we may be paid pursuant to our agreement

with CSL Behring, the planned R&D Day or other updates on the research pipeline, and 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, 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 in the future.

UNAUDITED CONSOLIDATED BALANCE SHEETS

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS