uniQure Announces Second Quarter 2021 Financial Results and Highlights Recent Company Progress ~ Expanded gene therapy pipeline with four new research programs and announced acquisition of Corlieve Therapeutics ~ ~ Prese

uniQure Announces Second Quarter 2021 Financial

and Highlights Recent Company Progress

~ Expanded gene therapy pipeline with four new

research programs and announced acquisition

~ Presented positive 52-week data from HOPE-B

pivotal study demonstrating sustained increases in

Factor IX (FIX) levels with mean FIX activity

of 41.5 percent of normal and significant reductions in bleeding events ~

its global commercialization and license transaction with CSL Behring

for hemophilia B gene therapy program ~

~ Initiated patient enrollment in second dose

cohort of Phase I/II clinical trial of AMT-130 for Huntington's disease, with initiation of a European Phase Ib/II clinical trial

expected in second half of 2021 ~

MA and Amsterdam, the Netherlands, July 26, 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 second quarter of 2021 and highlighted recent progress across its business.

a highly productive second quarter of this year marked by several notable achievements, including the closing on our global commercial

licensing agreement with CSL Behring in hemophilia B and the recent presentation of 52-week data on all patients in our HOPE-B pivotal

study showing durable and sustained increases in Factor IX levels within the non-hemophiliac range1,

as well as significant reductions in both bleeding events and usage of replacement therapy after a one-time administration of etranacogene

dezaparvovec," stated Matt Kapusta, chief executive

officer at uniQure. "Additionally, we continued to make strong progress in our U.S. Phase I/II study

of AMT-130 in Huntington's disease, including the completion of patient enrollment in the first dose cohort and the initiation of

dosing in the second cohort."

"We also recently hosted a Research and

Development Day where we disclosed four new promising research programs that complemented our expanding focus in neurological disease

and larger patient populations, including the acquisition of Corlieve Therapeutics and its gene therapy program targeting temporal lobe

epilepsy that builds on our expanding focus in serious neurological diseases," he added. "These investments deliver on our

commitment to leverage our strong cash position to invest in new research programs and enabling technologies with the potential to drive

significant value. In the second half of 2021, we expect to continue our progress across all programs, including availability of 78-week

data from the HOPE-B study, initiating patient enrollment for our open-label European study of AMT-130 in Huntington's disease,

and announcing initial data on the first four randomized Huntington's disease patients in the ongoing U.S. Phase I/II study."

1 Srivastava et al, Haemophilia.

2020;26(Suppl 6):1-158.

Recent Company Progress

In June, the Company hosted a virtual Research & Development Day to highlight new investments in its expanding gene therapy pipeline

focusing on central nervous system and rare, liver-directed disorders, as well as new advancements in platform technology and manufacturing.

Three new, internally developed pre-clinical programs were announced in Parkinson's disease (AMT-210), amyotrophic lateral sclerosis

(ALS) (AMT-160), and autosomal dominant Alzheimer's disease (AMT-240). Additional highlights from the event included updates on

the clinical development of AMT-130 in Huntington's disease and advancements in the ongoing preclinical development of AMT-150 for

spinocerebellar ataxia 3 and AMT-190 for Fabry disease.

of the leadership team

cash position to advance the Company's programs

Upcoming Investor Events (each to be conducted

Financial Highlights

Cash Position: As of June 30, 2021, the

Company held cash and cash equivalents of $677.3 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. Additionally, 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 June 30, 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. Pursuant to this agreement, as

of April 30, 2021, the Company had sold 921,730 ordinary shares for gross proceeds of approximately $29.6 million.

Revenues: Revenue for the three months

ended June 30, 2021, were $463.9 million, compared to $1.5 million during the same period in 2020. The increase is a result of $462.4

million of license revenue recognized from the CSL Behring Agreement.

Cost of Contract Revenues: Cost of contract

revenues for the three months ended June 30, 2021, was $23.2 million compared to nil for the same period in 2020. Costs incurred in 2021

are associated with the license revenue recognized under the CSL Behring Agreement.

R&D Expenses: Research and development

expenses were $32.8 million for the three months ended June 30, 2021, compared to $28.4 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 developments

of Huntington's disease, and increased activities associated with preclinical product candidates.

SG&A Expenses: Selling, general and

administrative expenses were $17.3 million for the three months ended June 30, 2021, compared to $11.5 million during the same period

in 2020. The change was primarily related to costs incurred for financial advisory fees in relation to the licensing transaction with

CSL Behring, increases in personnel and consulting expenses to support growth, and increased share-based compensation.

Other non-operating items, net: Other income,

net was $4.7 million for the three months ended June 30, 2021, compared to other expense, net of $4.3 million during the same period in

2020. The increase in income is primarily due to foreign currency gains in the current period compared to a net foreign currency loss

in the same period in 2020.

Net Loss: The net income for the three

months ended June 30, 2021, was $399.5 million, or $8.68 basic net income per share and $8.51 diluted net income per share, compared to

a loss of $42.6 million, or $0.96 basic and diluted loss per share during the same periods 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, 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 regarding 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 potential financial compensation we may be paid pursuant to our agreement with CSL Behring, 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.

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