Bay Street News

uniQure Highlights Pipeline Expansion and Advancements in Technology at Research & Development Day

~ Unveils New AAV Gene Therapy Approaches to Hemophilia A, Fabry Disease and Spinocerebellar Ataxia Type 3 ~

~ Introduces miQURE™ Gene Silencing Platform with Applications Across Multiple Indications ~

~ Highlights Advancements in Manufacturing and Research Technology ~

LEXINGTON, Mass. and AMSTERDAM, Netherlands, Nov. 19, 2018 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating Hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3 at the Company’s Research & Development Day held this morning in New York City.

“We are very proud of the progress the Company has made to deliver extensive preclinical data for these new gene therapy programs that expand our pipeline and further validate uniQure’s potential best-in-class vector delivery platform,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “The addition of these gene therapy candidates for indications in the liver and CNS brings us yet another step closer towards uniQure’s goal of delivering transformational medicine to patients suffering from genetic diseases. We look forward to advancing these programs closer to the clinic in 2019.”

New Gene Therapy Programs

Advances in Technology and Manufacturing

A replay of the webcast from the Company’s Research and Development Day will be available on the Investor section of the corporate website at www.uniQure.com along with a copy of the presentation.

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 liver/metabolic, central nervous system and cardiovascular 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, the achievement of any of our planned near term or other milestones, the development of our gene therapy product candidates including each of the product candidates at the pre-clinical stage of development, the ability to achieve therapeutic effects in human patients in any of our product candidates, the ability to produce a product candidate that is safe and effective, the ability to obtain regulatory approval for any of our product candidates, and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our 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 our and our collaboration activities, product development activities, 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 Annual Report on Form 10-K filed on March 14, 2018 and Quarterly Report on Form 10-Q filed on November 6, 2018. 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:    
       
  FOR INVESTORS:   FOR MEDIA:
  Maria E. Cantor Eva M. Mulder Tom Malone
  Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
  Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541
  m.cantor@uniQure.com e.mulder@uniQure.com t.malone@uniQure.com