Maintaining high concentrations of Adynovate in the blood of hemophilia A patients decreases the number of bleedings and enables more patients to stop having bleeds, according to the first results of a Phase 3b/4 study by Takeda.
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Higher doses of Adynovate effectively prevent bleeding in hemophilia A patients. Gene therapies for hemophilia A and B are being developed, with Takeda addressing immune reactions to viral vectors. Microbubble-delivered gene therapy shows promise for hemophilia B in mice. Marstacimab improves clotting in hemophilic blood samples, and MarzAA shows potential for treating acute bleeds in hemophilia.
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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…
Gene therapy delivered by microbubbles shows promise in treating hemophilia B, a hereditary bleeding disorder caused by the lack of blood clotting factor IX, new research suggests. The study, “Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice,” appeared in the journal Gene Therapy. Hemophilia B affects approximately…
Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…
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 Hemlibra…
Single or combination therapy with bypassing agents used for hemophilia A patients with inhibitors can be suitable treatment options for children with hemophilia B with factor IX inhibitors as well, a case series suggests. The report, “Alternative treatment options…
Cell therapy with endothelial progenitor cells and stem cells genetically engineered to produce a functional clotting factor VIII (FVIII) may provide a stable and long-term treatment for hemophilia A, a mouse study has found. The study shows that the transplant of cells…
U.S. doctors caring for people with hemophilia are more likely to make treatment decisions based on patient preferences than doctors in the U.K., who tend to be more influenced by their colleagues and government policies, according to a study comparing decision-making in both…
An ultrasound-mediated, non-viral gene therapy safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and lessened bleeding in a mouse model of the disease, a study shows. The delivery of a modified, improved version of the disease-associated F8 gene…