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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 […]

Novo Nordisk Resumes Clinical Trials Investigating Concizumab in Hemophilia A and B With or Without Inhibitors

August 20, 2020

The following is an excerpt from a press release from Novo Nordisk. Read the press release in its entirety here. Novo Nordisk announced the clinical trials in the concizumab phase 3 programme (explorer 6, 7 and 8) are being resumed. The clinical trials are investigating subcutaneous concizumab prophylaxis treatment in hemophilia A and B patients […]

BioMarin Update on Impact of COVID-19 on Clinical Trials

April 27, 2020

The following information is provided by BioMarin to provide general information about its ongoing clinical trials in the context of the COVID-19 pandemic.聽 “As more cases of COVID-19 are confirmed, we recognize the increased challenges and concerns faced by participants in the BioMarin Gene Therapy clinical studies. The safety and well-being of patients, healthcare providers, […]

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 […]

BioMarin Announces Gene Therapy Program Updates

January 23, 2020

The following is an announcement from BioMarin. BioMarin announced an update to the community regarding our ongoing gene therapy聽clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for聽hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating聽its safety and efficacy. Clinical Trial Overview BioMarin鈥檚 investigational gene therapy valoctocogene roxaparvovec […]

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, […]


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