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uniQure Announces Positive Data from the HOPE-B Pivotal Trial Gene Therapy in Patients with Hemophilia B

November 25, 2020

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V.,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, 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 […]

uniQure Announces License Agreement with CSL Behring to Commercialize Hemophilia B Gene Therapy

June 25, 2020

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced that uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene […]

uniQure Presents New Preclinical Data in Hemophilia A

May 15, 2020

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 […]

uniQure Announces Highlights of Recent Progress

May 1, 2020

The following is an excerpt from a press release from uniQure. Read the release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, reported recent progress across its business. 鈥淒espite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of […]

uniQure Announces Achievement of Target Patient Dosing in HOPE-B Pivotal Trial

March 26, 2020

The following is a press release from uniQure. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the […]

uniQure Announces Follow-Up Data from Study in Patients with Hemophilia B

July 10, 2019

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated clinical data on the three patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene […]

uniQure Presents Updated Clinical Data on Gene Therapy for Hemophilia B

May 10, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure聽announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. […]

uniQure Announces New Preclinical Data in Hemophilia A and Fabry Disease

May 2, 2019

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company, presented new preclinical data on its gene therapy candidates for the treatment of hemophilia A and for the treatment of Fabry disease during back-to-back oral presentations at the 22nd聽American Society […]

uniQure Announces Updated Clinical Data from Phase IIb Study in Patients with Hemophilia B

February 8, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure,聽a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a […]

uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B

February 6, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the […]

uniQure Announces Long-Term Clinical Data from Ongoing Phases and Confirms Dose for Hemophilia B Study

December 5, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results聽from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company鈥檚 next-generation gene therapy聽candidate […]

Update on uniQure’s Hemophilia B Gene Therapy Program

November 28, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic […]

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

November 28, 2018

The following is a press release from uniQure. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, 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鈥檚 Research & Development Day held this […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

November 19, 2018

The following is an excerpt from a press release from uniQure. uniQure announced聽that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). 鈥淎s a one-time administered therapy, AMT-061 has the […]

uniQure Presents New Data Demonstrating Clinical Benefits at American Society of Gene and Cell Therapy Annual Meeting in Chicago

May 22, 2018

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented data showing successful liver transduction with the AAV5 vector in both non-human primates and humans with pre-existing anti-AAV5 neutralizing antibodies (NABs). […]

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

December 15, 2017

  Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. uniQure N.V.聽a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced on Dec. 11, 2017, updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene […]

uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study

October 19, 2017

Note: The followed is excerpted from a press release from uniQure.聽Read uniQure’s full press release HERE.聽 uniQure announced today聽plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their聽product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from […]

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

July 11, 2017

Note: The following is an edited version of a press release from uniQure.聽Read the press release from uniQure聽in it鈥檚 entirety here. uniQure N.V.聽has聽announced updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy聽in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose […]

uniQure Announces Successful Gene Therapy Delivery in Animal Model

May 12, 2017

uniQure recently presented data at the American Society of Gene and Cell Therapy (ASGCT) 20th聽Annual Meeting in Washington, D.C.聽on successful repeated hepatic gene delivery with their聽AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results described successful sequential transduction with the two reporter transgenes and highlighted the progress made in readministration protocols […]

uniQure Receives EU PRIME Designation For Hemophilia B Product

May 2, 2017

聽uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B. From the press release: uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy聽in patients with severe […]

UniQure Receives FDA Breakthrough Designation of AMT-060 Investigational Gene Therapy

January 30, 2017

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety聽here. UniQure announced that AMT-060, 聽its proprietary, investigational gene therapy聽in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).聽 This designation is based on results from the […]

UniQure Announces New Data in Phase I/II Clinical Trial

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting聽in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure鈥檚 proprietary, investigational gene therapy聽for patients with severe […]

uniQure Presents Updated Clinical Data in Patients with Severe Hemophilia B

July 28, 2016

Note: The below is an edited version of an article originally published on Globe Newswire. The original release can be read in its entirety聽here. uniQure N.V. yesterday announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B.聽 The updated data show that […]

uniQure Announces Data from Phase I/II AMT-060 Clinical Trial

June 28, 2016

Note: The below is an edited version of a press release by UniQure.聽The original release can be read in it’s entirety聽here. On June 11, 2016,聽uniQure N.V. announced that additional data from its Phase I/II clinical trial of AMT-060 in hemophilia B patients were presented at the 21st聽Congress of the European Hematology Association (EHA) in Copenhagen, […]

uniQure Announces Preliminary Results from Hemophilia B Gene Therapy Trial

January 7, 2016

Note: the following is the 聽edited form of a 聽press releaseoriginally published by uniQure. The full release can be read here.聽 On Thursday, January 7, 2016, uniQure N.V. announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure鈥檚 novel AAV5/FIX […]


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