uniQure Announces 2021 Financial Results and Highlights Recent Company Progress ~ Data from largest gene therapy study in hemophilia B showed sustained therapeutic effect at 18-months and statistical superiority in reduc

uniQure Announces 2021 Financial Results and

Highlights Recent Company Progress

~ Data from largest gene therapy study in hemophilia

B showed sustained therapeutic effect at 18-months and statistical superiority in reducing annualized bleeding rate compared to baseline

FIX prophylactic therapy; Submissions of marketing applications on track for the first half of 2022 ~

~ Data from first four patients in ongoing

U.S. Phase I/II clinical trial in Huntington's Disease showed AMT-130 was well tolerated, with no significant safety concerns observed

~ Additional clinical data from all patients

in lower dose cohort of U.S. Phase I/II study of AMT-130, including mHTT and NfL biomarkers, expected to be presented in second quarter

~ Initiated enrollment of European Phase Ib/II

study of AMT-130 with first two patients dosed ~

~ Refractory temporal lobe epilepsy and Fabry

programs advancing into IND-enabling studies in 2022 ~

~ Ended 2021 in strong financial position with

$556 million in cash ~

Lexington, MA and Amsterdam, the Netherlands,

February 25, 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 2021 and highlighted recent progress across its business.

"We made tremendous progress in 2021 across

all our strategic imperatives, including advancing our clinical-stage programs, expanding our research pipeline, and preparing for the

submission of marketing applications planned for etranacogene dezaparvovec during the first half of 2022," stated Matt

Kapusta, chief executive officer at uniQure. "With positive data from the HOPE-B pivotal study we look forward to working closely

with our partner, CSL Behring to bring this potentially life-changing gene therapy to patients living with hemophilia B."

"During 2022, we are also keenly focused

on maintaining our strong momentum in enrolling two ongoing Phase I/II studies in Huntington's disease. Importantly, we look forward

to sharing additional safety and biomarker data from all ten patients in the lower dose cohort, including mutant HTT protein (mHTT) and

neurofilament light chain (NfL), during the second quarter of 2022" he added. "We also are poised to advance our gene therapy

product candidates for Fabry disease and refractory temporal lobe epilepsy into IND-enabling toxicology studies, and we expect to initiate

at least two new gene therapy programs targeting the liver and CNS during the year. As we continue to advance and grow our pipeline,

we are also expanding our manufacturing footprint with a second cGMP facility in Amsterdam, which we expect will come online in 2022."

Recent Key Accomplishments

Upcoming Investor Events (each to be conducted

Financial Highlights

Cash Position: As of December 31,

2021, the Company held cash and cash equivalents of $556.3 million, compared to $244.9 million as of December 31, 2020. Upon the

CSL Behring Agreement becoming fully effective on May 6, 2021, the Company received $462.4 million of payments. In January and

December 2021, the Company and Hercules amended the debt facility agreement, under which the Company drew down an additional $35.0

million in January 2021 and a further $30.0 million for a total of $100.0 million outstanding under the facility as of December 31,

2021. The Company also extended the maturity from June 2023 to December 2025. In March and April 2021, the Company

sold 921,730 ordinary shares for gross proceeds of approximately $29.6 million under an Open Market Sale Agreement with SVB Leerink LLC.

In July 2021, the Company paid a net EUR 42.1 million ($49.9 million) related to its acquisition of Corlieve.

Revenues: Revenue for the year ended December 31,

2021, was $524.0 million, compared to $37.5 million during the same period in 2020. The increase is a result of $462.4 million of

license revenue recognized upon closing of the CSL Behring transaction in May 2021 as well as $55.0 million of license revenue related

to milestone payments the Company expects to collect following the submissions of a biologics license application ("BLA")

and market authorization application ("MAA") by CSL Behring in the first half of 2022. In 2020, the Company had recognized

$27.8 million in non-cash license revenue as of the December 1, 2020, effective date of the amended Bristol-Myers Squibb collaboration

and license agreement as well as revenue of $4.4 million in December 2020 following achievement of a research milestone for one

of the four Collaboration Targets.

R&D Expenses: Research and development

expenses were $143.5 million for the year ended December 31, 2021, compared to $122.4 million during the same period in 2020. 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 as well as increased activities associated with preclinical product

candidates, and an increase from fair value changes related to liability recorded for contingent consideration owed in relation to the

acquisition of Corlieve.

SG&A Expenses: Selling, general and

administrative expenses were $56.3 million for the year ended December 31, 2021, compared to $42.6 million during the same period

in 2020. The change was primarily related to recruitment of personnel, increased share-based compensation expenses, and as a result of

financial advisory payments made in relation to our licensing transaction with CSL Behring.

Other Income, net: Other income, net was

$11.4 million for the year ended December 31, 2021, compared to other income, net of $2.0 million during the same period in 2020.

The increase in other income, net was primarily related to the receipts of employee retention credits under the U.S. CARES Act, income

related to payments received from European authorities to subsidize research and development efforts in the Netherlands and income related

to a settlement agreement that the Company and VectorY B.V. entered into in April 2021.

Other Non-operating Items, net:

Other non-operating income, net was income of

$22.2 million for the year ended December 31, 2021, compared to other non-operating loss of $16.0 million for the same period in

2020. The increase in other non-operating income was primarily related to net foreign currency gains in the current period compared to

net foreign currency losses in the same period in 2020.

The net income for the year ended December 31,

2021, was $329.6 million, or $7.17 basic net income per ordinary share and $7.04 diluted net income per ordinary share, compared to a

loss of $125.0 million, or $2.81 basic and diluted loss per ordinary share during the same period in 2020.

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 re

not limited to, whether we will bring etranacogene dezaparvovec to patients, whether we will share mutant HTT protein (mHTT) and neurofilament

light chain (NfL) or other safety and biomarker data from any patients in our AMT-130 clinical trials, during the second quarter of 2022,

the first half of 2023 or ever, whether we will be able to advance our gene therapy product candidates for Fabry disease or refractory

temporal lobe epilepsy into IND-enabling toxicology studies, whether we will initiate any new gene therapy programs targeting the liver

or CNS during the year, whether our second cGMP facility in Amsterdam will come online in 2022 or ever, whether marketing applications

for etranacogene dezaparvovec, will be submitted in the U.S. or the European Union during the first half of 2022 or ever.. 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 in the future.

UNAUDITED CONSOLIDATED BALANCE SHEETS

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS