A single infusion of AMT-061 (etranacogene dezaparvovec), uniQure’s experimental gene therapy for the treatment of hemophilia B, led to increased and sustained activity of clotting factor IX (FIX), according to one-year data from a Phase 2b clinical trial. The therapy effectively prevented…
BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec safely and significantly reduced bleeding events — including in target joints — and the use of prophylactic clotting factor VIII in adults with severe hemophilia A, data from a Phase 1/2 study show. These results, along with an interim analysis of…
Sangamo Therapeutics has handed over the development of SB-525, a gene therapy for hemophilia A, to Pfizer, which will now advance the therapy into Phase 3 clinical trials. Pfizer has already started recruiting participants for the lead-in Phase 3 trial (NCT03587116). which will support…
Hemophilia News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to hemophilia throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read stories of 2019. No.
Takeda has presented new real-world data to support the cost-effectiveness and persistent benefits to patients of its hemophilia A and B therapies: Adynovate [antihemophilic factor (recombinant), PEGylated] and Feiba (anti-inhibitor coagulant complex). The findings were discussed at the recent 61st American Society of Hematology…
Takeda has presented early data on the prevalence of — and a possible solution for — one of gene therapy’s main hurdles: the development of an immune reaction against the viral-based delivery vectors used in such therapies. The findings, presented at the 61st American Society of Hematology (ASH) Annual Meeting…
To help promote healthy lifestyle changes, the pharmaceutical company Takeda has enrolled some 900 patients with bleeding disorders like hemophilia in the Virgin Pulse Global Challenge. The annual well-being Global Challenge aims to equip teams of employees with the knowledge, tools and support necessary to enhance performance and…
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
Bayer has entered into a three-year research partnership with the Children’s Hospital of Philadelphia to develop small molecule non-replacement therapies as a first-line and less burdensome oral treatment for hemophilia A and B. Hemophilia is caused by a lack of blood clotting factors or their faulty production,…
New preclinical data on one of Catalyst Biosciences‘ lead candidate therapies — marzeptacog alfa activated (MarzAA), an engineered clotting Factor VIIa (FVIIa) given as an under-the-skin injection — support its potential to treat acute bleeds in people with hemophilia and its ability to be used safely in combination with…
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