uniQure Announces Positive Top-Line Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B ~ Phase III study in 54 patients met primary endpoint with mean Factor IX act

uniQure Announces Positive Top-Line

Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B

~ Phase III study in 54 patients met primary

endpoint with mean Factor IX activity of 37% of normal at 26 weeks ~

~ Patients achieved significant increases

in Factor IX activity irrespective of pre-existing neutralizing antibodies, potentially supporting broad patient access ~

~ Increases in Factor IX activity were

sustained for up to 18 months with near elimination of bleeding ~

~ Mean annualized usage of FIX replacement

therapy declined by 96 percent after dosing compared to the observational lead-in period ~

~ Etranacogene dezaparvovec was well-tolerated

with no treatment-related serious adverse events ~

~ Data selected for late-breaker oral presentation

at the Annual Meeting of the American Society

of Hematology (ASH) ~

Lexington, MA and Amsterdam, the Netherlands,

November 19, 2020 - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative

therapies for patients with severe medical needs, today announced positive top-line data from its pivotal, Phase III HOPE-B gene

therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy

for the treatment of patients with severe and moderately severe hemophilia B. This is the first data set to be reported from a

Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of patients receiving a single gene therapy

investigational product to be reported to date. These clinical data were published today as a late-breaking abstract, one of only

six accepted for presentation at the 62nd Annual Meeting of the American Society of Hematology (ASH) and will be featured

as an oral presentation in the conference on December 8, 2020. The abstract is available here.

"We are extremely pleased that these

top-line pivotal data show that a single administration of etranacogene dezaparvovec gene therapy led to sustained increases of

Factor IX (FIX) to functionally-curative levels capable of eliminating the need for regular infusions to control and prevent bleeding

episodes," stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. "Most impressively,

these data also demonstrate the potential to achieve clinical benefit in patients with a range of pre-existing neutralizing antibodies

representative of the general population. The ability to dose a gene therapy in patients with pre-existing neutralizing antibodies

has not been demonstrated for any other gene therapy and illustrates the potentially unique ability of our AAV5 platform to address

the needs of a broad set of patients living with hemophilia B and other disorders."

The pivotal, Phase III HOPE-B clinical

trial of etranacogene dezaparvovec is an open-label, single-dose, single-arm, multi-national trial in adult males with severe

or moderately severe hemophilia. All patients required prophylactic routine FIX replacement prior to entering the clinical trial,

and patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5.

Patients in the HOPE-B clinical study

were initially enrolled into a prospective, observational lead-in period of at least six months during which bleeding events and

FIX replacement therapy usage were monitored. Fifty-four patients received a single intravenous infusion of etranacogene dezaparvovec

gene therapy at 2x1013 gc/kg, including 23 patients who had pre-existing NAbs to AAV5. Patients are then evaluated

to assess FIX activity determined by a one-stage assay performed at a central laboratory, annualized bleeding rates and usage

of Factor IX replacement therapy. Patients will be monitored for five years to evaluate the safety of etranacogene dezaparvovec.

HOPE-B Primary Endpoint of FIX Activity

at 26 Weeks Achieved, Irrespective of Pre-Existing NAbs

FIX activity in the 54 patients increased

rapidly after dosing from 2% to a mean of 37.2 percent at 26 weeks, meeting the first primary endpoint. No correlation between

pre-existing NAbs and FIX activity was found in patients with NAb titers up to 678.2, a range expected to include more than 95%

of the general population; one patient with a NAb titer of 3,212.3 did not show increase in FIX activity.

During the 26-week period after dosing,

72 percent of patients (39/54) reported no bleeding events. Fifteen patients reported a total of 21 bleeds1. Mean

annualized usage of FIX replacement therapy, a secondary endpoint in the clinical trial, declined by 96 percent.

Etranacogene dezaparvovec was generally

well-tolerated with no treatment-related serious adverse events. Most adverse events were classified as mild (81.5 percent). Most

common events included transaminase elevation treated with steroids per protocol (9 pts; 17%), infusion-related reactions (7 pts;

13%), headache (7 pts; 13%) and influenza-like symptoms (7 pts; 13%). Liver enzyme elevations resolved with a tapering course

of corticosteroids and FIX activity remained in the mild range in the steroid treated patients. No relationship between safety

and NAbs titers was observed.

"We believe that etranacogene dezaparvovec

has the potential to be a first- and best-in-class gene therapy for patients with hemophilia B," stated Matt Kapusta, chief

executive officer of uniQure. "We are very pleased to have met the 26-week FIX primary endpoint and to feature these promising

data at the upcoming ASH conference. Based on interactions with the FDA and EMA, we plan to incorporate FIX activity and bleeding

rates at 52 weeks as additional co-primary endpoints in the study. We look forward to holding our pre-BLA meeting with the FDA

and completing the last patient's 52-week follow-up visit in the first quarter of 2021."

About Etranacogene Dezaparvovec

Etranacogene dezaparvovec consists of

an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). uniQure holds

multiple issued patents in the United States and Canada broadly covering methods of treating bleeding disorders, including hemophilia

B, using AAV gene therapy with the FIX-Padua variant. Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation

by the United States Food and Drug Administration and access to Priority Medicine (PRIME) regulatory initiative by the European

Medicines Agency. In June 2020, the Company and CSL Behring entered into a licensing agreement providing CSL Behring with

exclusive global rights to etranacogene dezaparvovec. This licensing agreement is subject to antitrust regulatory review in the

United States, Australia and the United Kingdom that is currently ongoing.

include any bleeding event reported after the treatment of etranacogene dezaparvovec, including spontaneous, traumatic, and those

associated with unrelated medical procedures, whether or not FIX treatment was required.

AAV5-based gene therapies have been demonstrated

to be safe and well tolerated in a multitude of clinical trials, including five uniQure trials conducted in nearly 80 patients

in hemophilia B and other indications. No patient treated in clinical trials with the uniQure's AAV5 gene therapies has

experienced any confirmed cytotoxic T-cell-mediated immune response to the capsid. Additionally, pre-clinical and clinical data

show that AAV5-based gene therapies may be viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially

increasing patient eligibility for treatment compared to other gene therapy product candidates.

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 etranacogene dezaparvovec will be the first-in-class

or best-in-class gene therapy for patients with hemophilia B, whether AAV5-based gene therapies can provide clinical benefit to

patients with pre-existing neutralizing antibodies, and whether uniQure will conduct the 52-week follow-up visit of the last patient

and its pre-BLA meeting with the FDA in the first quarter of 2021 or ever. Our actual results could differ materially from those

anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and

our collaborators' clinical development activities, clinical results, collaboration arrangements, corporate reorganizations

and strategic shifts, 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 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