uniQure Announces Successful Readministration of Gene Therapy in Non-Human Primates

- Effective Transduction Achieved After Readministration of Gene Transfer -

- Preclinical Data Presented Today at American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Washington, D.C. -

- Investor & Analyst Breakfast and Webcast Friday, May 12 at 7:00 a.m. EDT -

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 11, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today presented data on successful repeated hepatic gene delivery with uniQure's AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results describing successful sequential transduction with the two reporter transgenes were presented today in a session at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting in Washington, D.C., and highlight the progress made in readministration protocols for AAV gene transfers.

"One of the major challenges in AAV-based gene therapy is the presence of circulating anti-AAV neutralizing antibodies, which can pre-exist in patients or develop after a single administration of gene therapy. These neutralizing antibodies may prevent successful gene transfer in patients," stated Valerie Sier-Ferreira, Ph.D., head of immunology at uniQure. "uniQure has made tremendous progress in optimizing and advancing readministration protocols that we believe will significantly impact the application of our gene therapies, potentially enabling repeated administration and making our gene therapies available to more patients around the world."

Preclinical Data Findings  

Circulating neutralizing antibodies (NABs) against AAV vectors can be present in the blood of patients prior to AAV treatment due to naturally acquired infections with the wild-type AAV virus. Additionally, anti-AAV NAB titers are raised to extremely high levels after first administration of an AAV vector in the course of a gene transfer treatment. These NABs may prevent successful transduction of the target cells. As a result, in ongoing clinical trials using AAV vectors, patients who present detectable levels of anti-AAV antibodies are excluded from treatment.

In the study conducted by uniQure, an extracorporeal immune-adsorption (IAd) procedure, similar to those currently used clinically for the treatment of auto-immune disorders, was applied to reduce the levels of anti-AAV NABs in NHPs that previously received an AAV reporter construct (AAV5-hSEAP). NAB concentrations were decreased by a mean factor of 12, permitting successful readministration of an AAV5 vector containing a second reporter gene (hFIX). The hFIX concentrations obtained after re-administration were in the range of the hFIX levels obtained after primary administration in animals undergoing IAd, while no hFIX  expression was seen in controls who did not receive IAd prior to readministration. This immune-adsorption procedure shows promising preclinical outcomes which can potentially become a successful strategy for readministration of a gene therapy in a therapeutic regimen.

The poster presented during the ASGCT Annual Meeting in Washington, D.C., can be accessed by visiting the Investor relations section of the Company's website here.

Investor/Analyst Breakfast and Webcast on Friday, May 12, 2017

An investor and analyst breakfast meeting will be held on Friday, May 12 at 7:00 a.m. EDT. Senior members of uniQure's research and development team will discuss the results of this study along with the four additional abstracts that are being presented at ASGCT. The meeting will be webcast live along with slides and can be accessed by visiting the investor relations section of the Company's website at

Date and Time:  Friday, May 12 at 7:00 am EDT
Location: Omni Shoreham Hotel, The Congressional Room, 2500 Calvert Street NW, Washington, D.C. 
The Omni Shoreham hotel is located directly across from the conference venue.

To request attendance at the meeting, please RSVP to as space is limited.

About uniQure 
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 and partnered gene therapies to treat patients with hemophilia, Huntington's disease and cardiovascular diseases.

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, the development of our gene therapy product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with corporate reorganizations and strategic shifts, collaboration arrangements, our and our collaborators' clinical development activities, 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 2016 Annual Report on Form 10-K filed on March 15, 2017. 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.

uniQure Contacts:

Maria E. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452

Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541

Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79

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Source: uniQure N.V. via Globenewswire