uniQure Announces First Quarter 2022 Financial Results and Highlights Recent Company Progress ~ Partner CSL Behring submitted Marketing Authorization Application (MAA) in Europe for etranacogene dezaparvovec in hemophili

uniQure Announces First Quarter 2022 Financial

and Highlights Recent Company Progress

~ Partner CSL Behring submitted Marketing Authorization

Application (MAA) in Europe for

etranacogene dezaparvovec in hemophilia B;

MAA validated by the European Medicines Agency (EMA)

and granted accelerated assessment ~

~ Received $55 million in milestone payments

associated with global submissions

of marketing and license applications ~

~ Completed patient enrollment of higher dose

cohort of U.S. Phase I/II study of AMT-130 and lower dose cohort of European Phase Ib/II study; total of 32 patient procedures completed

~ One year safety and biomarker data from all

10 patients in lower dose cohort of U.S. Phase I/II study of AMT-130 in Huntington's disease, including mHTT and NfL biomarkers,

expected in second quarter 2022 ~

~ Multiple preclinical data presentations to

be featured at American Society of Gene and Cell Therapy

(ASGCT) Hybrid Congress ~

~ Appointed Erin Boyer as Chief People and

Culture Officer and Richard Porter, previously founder and CEO

of Corlieve as Chief Business Officer ~

Lexington, MA and Amsterdam, the Netherlands,

May 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 first quarter of 2022 and

highlighted recent progress across its business.

"uniQure is off to a strong start in 2022,

with important advancements across our key programs and earlier-stage research activities, as well as the strengthening of our executive

leadership team," stated Matt Kapusta, chief executive officer at uniQure. "In the

first quarter of the year, our partner CSL Behring made important progress with global regulatory submissions for etranacogene dezaparvovec,

including the granting of accelerated assessment by the EMA in Europe."

"We also made significant advancements enrolling

patients in our Phase I/II studies for AMT-130 in Huntington's disease," he added. "To date, we have completed 32 patient

procedures across both studies and look forward to presenting in the second quarter of 2022 one-year safety and biomarker data from the

10-patient lower dose cohort of the U.S randomized, controlled Phase I/II trial. We continue to advance our pipeline of earlier-stage

gene therapy candidates and are pleased to have recently initiated a dose-ranging IND-enabling study for AMT-260 in refractory temporal

lobe epilepsy. I am very proud of the uniQure team's unwavering commitment to progressing our ambitious goals and achieving our

mission of delivering the promise of gene therapies to patients."

Recent Key Accomplishments

Upcoming Investor Events

Financial Highlights

Cash Position: As of March 31, 2022,

the Company held cash and cash equivalents of $524.9 million, compared to $556.3 million as of December 31, 2021.

Revenues: Revenue for the three months

ended March 31, 2022, was $1.8 million, compared to $0.5 million during the same period in 2021. The increase is a result of

collaboration revenue recognized as a result of closing the CSL Behring Agreement in May 2021.

R&D Expenses: Research and development

expenses were $45.0 million for the three months ended March 31, 2022, compared to $32.7 million during the same period in 2021.

The change was primarily related to recruitment of personnel to support the development of product candidates, advancing the clinical

development of the Company's Huntington's disease gene therapy program, increased activities associated with preclinical product

candidates, and an increase of the fair value of the liability recorded for contingent consideration owed in relation to the acquisition

of Corlieve Therapeutics SAS.

SG&A Expenses: Selling, general and

administrative expenses were $11.0 million for the three months ended March 31, 2022, compared to $12.4 million during the same period

in 2021. The reduction was primarily related to incurring one-off professional fees to close the CSL Behring transaction in 2021 with

no such fees in 2022.

Other Non-operating Items, net:

Other non-operating income, net was income of

$6.8 million for the three months ended March 31, 2022, compared to other non-operating income, net of $3.1 million for the same

period in 2021. The increase in other non-operating income was primarily related to an increase in net foreign currency gains of $8.6

million in the current period compared to $4.6 million the same period in 2021, partially offset by an increase in interest expense related

to the loan facility with Hercules Capital, Inc. of $1.0 million as a result of the Company drawing down an additional $35.0 million

in January 2021 and a further $30.0 million in December 2021.

The net loss for the three months ended March 31,

2022, was $46.7 million, or $1.00 loss per ordinary share, compared to $41.6 million, or $0.91 loss per ordinary share, during the same

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, refractory temporal lobe epilepsy, Fabry disease, 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, whether we are able to enroll up to 18 additional patients or otherwise commence enrollment at any time for our third

cohort exploring the use of an alternative stereotactic navigation system to simplify placement of infusion catheters, whether we are

able to enroll up to 59 patients in the U.S. and European Phase I/II trials of AMT-130, whether we are able to disclose volumetric MRI

and functional data during the first half of 2023 or ever, and whether we are able to initiate a GLP toxicity study in non-human primates

in the second half of 2022 or submit an associated investigational new drug application in 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 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 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

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