uniQure Announces Positive Recommendation from Data Safety Monitoring Board of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease ~ No Significant Safety Concerns Observed ~ ~ Independent Data

Announces Positive Recommendation from

Safety Monitoring Board of Phase I/II Clinical Trial of AMT-130

the Treatment of Huntington's Disease

~ No Significant Safety Concerns Observed

~ Independent Data Safety Monitoring Board

Recommends Proceeding with Study Enrollment ~

~ Full Enrollment of First Cohort Expected

MA and Amsterdam, the Netherlands, February 4, 2021 - uniQure N.V. (NASDAQ:

QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today

announced that the independent Data Safety Monitoring Board (DSMB) overseeing the Phase I/II clinical trial of AMT-130 for the

treatment of Huntington's disease has met and reviewed the six-month safety data from the first two enrolled patients

and the 90-day safety data from the next two enrolled patients in the study. No significant safety concerns were noted to prevent

further dosing, and the final six patients in the first cohort are now cleared for enrollment. uniQure expects to achieve full

patient enrollment in cohort one by mid-year 2021. The Phase I/II study is a double-blind, randomized clinical trial being conducted

in the United States. To date, two patients have been treated with AMT-130, and two patients received the imitation surgery.

"We are encouraged with

the positive recommendation to proceed with patient enrollment from this second DSMB meeting," said Ricardo Dolmetsch, president

of research and development at uniQure. "We will now focus on enrolling the last six additional patients in the first cohort

and look forward to sharing initial biomarker and imaging data towards the end of the year."

About the Phase I/II Clinical

The Phase I/II clinical trial

of AMT-130 for the treatment of Huntington's disease will explore the safety, tolerability, and efficacy signals in 26 total

patients with early manifest Huntington's disease split into a 10 patient, low-dose cohort followed by a 16 patient, higher-dose

cohort randomized to treatment with AMT-130 or an imitation (sham) surgery. The five-year, multi-center trial consists of a blinded

12-month core study period followed by unblinded long-term follow-up. Patients will receive a single administration of AMT-130

through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).

Additional details are available on www.clinicaltrails.gov (NCT04120493).

AMT-130 is uniQure's first

clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE platform.

About Huntington's Disease

Huntington's disease is

a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive

decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing

CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein

in the brain. Despite the clear etiology of Huntington's disease, there are no currently approved therapies to delay the

onset or to slow the disease's progression.

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

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 will be able to enroll the next six patients

in the clinical trial by mid-year 2021 and whether we will be able to announce initial biomarker and imaging data by the end of

2021. Our 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 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 2,

2020 and Quarterly Report on Form 10-Q filed on October 27, 2020. Given these risks, uncertainties and other factors, you should

not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements,

even if new information becomes available in the future.