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uniQure Inc : uniQure Announces Positive Recommendation to Advance Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington s Disease

(1) Independent Data Safety Monitoring Board Recommends Proceeding with Second, Higher-Dose Cohort LEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 27, 2021has met and reviewed safety data for the fully enrolled first cohort of ten patients. This data set included nine-month safety data from the first two enrolled patients, six-month safety data from the next two enrolled patients, and 30-day safety data on remaining six patients in the trial. The DSMB recommended continued dosing in the study, and uniQure will now begin to enroll patients in the higher-dose cohort of the trial. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States. To date, six patients have been treated with AMT-130, and four patients received the imitation surgery.

uniQure Inc : uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector

uniQure Inc.: uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector ~ Further Supports Potential for AAV5 Gene Therapies to be Viable Treatments in Nearly All Patients ~ Data Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting ~ LEXINGTON, Mass. and AMSTERDAM, May 13, 2021showing clinical benefit in hemophilia B patients with pre-existing neutralizing antibodies (NAbs) to AAV5, and no relationship between AAV5 NAbs and the tolerability of the therapy. The data were featured on Wednesday, May 12, 2021 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in an oral presentation by Michael Recht, M.D., Ph.D., M.B.A., professor of pediatrics, division of hematology and oncology at the Oregon Health & Science University School of Medicine. These data were previously presented at a medical meeting late last year.

uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector

uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector
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uniQure Presents HOPE-B Clinical Data Demonstrating

~ Further Supports Potential for AAV5 Gene Therapies to be Viable Treatments in Nearly All Patients ~ Data Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting ~ LEXINGTON, Mass. and AMSTERDAM, May 13, 2021 (GLOBE NEWSWIRE) uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, yesterday presented 26-week clinical data from the pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec showing clinical benefit in hemophilia B patients with pre-existing neutralizing antibodies (NAbs) to AAV5, and no relationship between AAV5 NAbs and the tolerability of the therapy. The data were featured on Wednesday, May 12, 2021 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in an oral presentation by Michael Recht, M.D., Ph.D., M.B.A., professor of pediatrics, division of hematology and oncology at the Oregon Health & Science University School

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