uniQure Announces Second Quarter 2023 Financial Results and Highlights Recent Company Progress ~ Announced encouraging interim analysis data from ongoing Phase I/II clinical trial of AMT-130 in Huntington's disease showi

Second Quarter 2023 Financial Results

Recent Company Progress

~ Announced encouraging interim analysis data

from ongoing Phase I/II clinical trial of AMT-130 in Huntington's disease showing preservation of function compared to baseline

and clinical benefits relative to natural history; both dose cohorts of AMT-130 continue to be generally well-tolerated ~

underway to initiate two Phase I/II clinical trials in refractory temporal lobe epilepsy and SOD1-ALS later this year; IND for Fabry

disease expected to be submitted in the second half of 2023 ~

~ Strong cash position of $628.6 million as

of June 30, 2023, which excludes a $100 million milestone related to first commercial sale of HEMGENIX in the U.S.

received in July 2023 ~

~ Announced sale of royalty interest in HEMGENIX

for up to $400 million ~

~ Appointed Walid Abi-Saab, M.D., as Chief Medical

Officer; Jeannette Potts as Chief Legal Officer ~

Lexington, MA and Amsterdam, the Netherlands,

August 1, 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 the second quarter

of 2023 and highlighted recent progress across its business.

"In the second quarter of the year, we

continued to make strong progress advancing our programs, including announcing encouraging data from the interim analysis of our Phase

I/II study of AMT-130 in Huntington's disease," stated Matt Kapusta, chief executive officer of uniQure. "These

data show that patients treated with AMT-130 appear to have generally preserved clinical and motor function and neurofilament light chain

(NfL) levels suggesting a stable to improving neurodegenerative profile. We believe that, collectively, the data suggest early and promising

evidence of clinical benefit in patients treated with AMT-130 compared to a patient-matched natural history data set. We look forward

to providing another update in the fourth quarter of 2023 that will include data from our European study, as well as preparing for regulatory

interactions in the first quarter of 2024 to discuss the data and potential late-stage development of AMT-130."

"We are also working towards the initiation

of clinical trials for our gene therapy candidates in refractory temporal lobe epilepsy and SOD1-ALS later this year, as well as the submission

of an IND for our Fabry disease gene therapy," he added. "With a strong cash position following achievement of the $100 million

milestone for first U.S. commercial sale of HEMGENIX and completion of a royalty financing transaction, we are

focused on rapidly moving programs into clinical development and creating near-term value to shareholders."

Upcoming Investor Events

Financial Highlights

Cash position: As of June 30, 2023,

the Company held cash and cash equivalents and investment securities of $628.6 million, compared to $392.8 million as of December 31,

2022. The Company entered into a royalty agreement in May 2023 and received an upfront payment of $375.0 million.

Revenues: Revenue for the three months

ended June 30, 2023 was $2.4 million, compared to $0.5 million in the same period in 2022. The increase is primarily a result of

contract manufacturing revenues of $1.3 million recognized in the current period related to contract manufacturing HEMGENIX

for CSL Behring and an increase in license revenues of $0.8 million.

R&D expenses: Research and development

expenses were $46.0 million for the three months ended June 30, 2023, compared to $46.2 million in the same period in 2022. The decrease

in external program expense was offset by an increase in various other expenses, including employee and contractor-related expenses, facility

expenses and research and development disposable costs.

SG&A expenses: Selling, general and

administrative expenses were $21.2 million for the three months ended June 30, 2023, compared to $12.5 million in the same period

in 2022. The increase was primarily related to an increase in financial advisory fees, an increase in various other professional fees

and an increase in personnel and contractor-related expenses.

Other non-operating items, net:

Other non-operating items net was an expense of

$3.2 million for the three months ended June 30, 2023, compared to net income of $16.7 million for the same period in 2022. The decrease

in other non-operating items, net was primarily related to a decrease in foreign currency gains of $19.0 million and an increase in interest

The net loss for the three months ended June 30,

2023, was $68.5 million, or $1.44 basic and diluted loss per ordinary share, compared to $39.1 million net loss for the same period in

2022, or $0.84 basic and diluted loss 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 - represent 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, the Company's ability to fund operations into 2026, whether

there will be continued progress on the commercialization of HEMGENIX and the Company's ability to meet certain

milestones, statements about the clinical benefits for patients treated with AMT-130, the timing of patient enrollment in our AMT-130

clinical trial for Huntington's Disease, whether the Company will announce follow up data from the U.S. Phase I/II clinical study

of AMT-130 and whether such announcement will include data from the Company's European study, whether that data will help to further

guide our ongoing clinical development of AMT-130, 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,

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, and whether the Company will begin interactions with regulatory agencies in the first quarter of 2024. 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 , material changes to our interim

or preliminary data, 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 and ongoing

litigation, the 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 and the Quarterly Report on Form 10-Q

filed August 1, 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

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