"The data to be featured in an oral presentation at ISTH now demonstrate long-term safety, efficacy and the potential for broad application of AAV5 in hemophilia B," stated Matthew Kapusta, chief executive officer of uniQure. "We also look forward to the presentation of clinical data demonstrating successful and effective transduction in patients with pre-existing neutralizing antibodies to AAV5, further supporting our belief that our AAV5-based gene therapies may provide clinical benefits to nearly all patients suffering from hemophilia B and other severe diseases."
Specific details on uniQure's presentations at ISTH include:
Updated data on AMT-060 will be presented by study investigator Professor Frank W. Miesbach, M.D. Ph.D. of the Universitäts Klinikum Frankfurt, Germany. AMT-060 is an AAV5-FIX gene therapy developed by uniQure using its proprietary insect-cell based technology platform. The updated data will include up to 1.5 years of follow-up on the first patient cohort receiving 5x1012 gc/kg and up to a year of follow-up on the second patient cohort receiving a higher dose of 2x1013 gc/kg.
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, statements regarding 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 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 with the Securities and Exchange Commission 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.
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