Xenetic Biociences has received a $3 million milestone payment from its partner Shire for advancing the Phase 1/2 clinical trial of PSA-recombinant SHP656, a factor VIII protein, evaluating the drug’s effectiveness as a long-acting therapy for hemophilia A. Shire’s main objective behind the agreement is to develop a novel FVIII…
Two presentations at the 2016 American Society of Hematology Annual Meeting (ASH) highlight ongoing treatment advances for hemophilia B. The data suggest that life with hemophilia could soon become easier to manage. Long-acting replacement therapy The ASH meeting, held in San Diego on Dec. 3-6, was a platform for CSL Behring to…
A collaborative research study offers new insights into how a protein called furin influences blood clotting and found it functions differently in hemophilia A and B, findings that are promising for improving gene therapy for hemophilia A. The study, “Circumventing furin enhances factor VIII biological activity and ameliorates bleeding…
Hemophilia A patients who receive replacement therapies of factor VIII often develop inhibitors against factor concentrates which hinders the effectiveness of the therapy, increases disability, and reduces quality of life. Although immune tolerance induction (ITI) can reduce inhibitors against factor VIII concentrates, the study “Long-term course of…
Transplants of embryonic stem (ES) cells that secrete the human factor VIII (FVIII) may be a promising therapeutic approach for patients with hemophilia A, according to an animal study conducted by researchers at the Nara Medical University School of Medicine in Japan. The study, “Therapeutic approaches for treating hemophilia…
uniQure N.V. has updated the clinical data from its ongoing clinical trial of a gene therapy for the treatment of patients with hemophilia B. The results indicate sustained improvement by all patients at a low-dose, with durable levels of Factor IX (FIX) gene activity for several weeks after treatment. The…
Researchers at the Center for Research in Transplantation and Immunology in France have reviewed a paper concerning certain strategies utilized to correct hemophilia B and the importance of gene delivery vehicles in successful therapies for treatment of the condition. The review paper, “Successful correction of hemophilia by CRISPR/Cas9 genome…
Jivi (damoctocog alfa pegol) is an approved lab-made replacement therapy designed to prevent or treat bleeding episodes in previously treated children and adults with hemophilia A. It is administered via into-the-vein (intravenous) infusions.
Wilate (human von Willebrand factor/coagulation factor VIII complex) is a plasma-derived replacement therapy that's approved to prevent and control bleeds in adults and adolescents with hemophilia A. It is given via an into-the-vein, or intravenous, injection.
Altuviiio (efanesoctocog alfa) is an approved long-lasting factor VIII (FVIII) replacement therapy used to reduce the frequency of bleeding episodes or control and manage active bleeds, including those occurring during surgery, in children and adults with hemophilia A.
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