Ixinity (recombinant factor IX product, IB1001) was found to be safe and effective in previously treated children with hemophilia B younger than 12, according to data from Aptevo Therapeutics‘ ongoing Phase 3 clinical trial. The company presented data from the analysis of two studies at the recent Hemostasis and Thrombosis…
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San Francisco-based Catalyst Biosciences has completed toxicology studies of its new drug CB 2679d/ISU304 — a highly potent next-generation coagulation factor to treat hemophilia B. This  follows the therapy’s recent Investigational New Drug (IND) approval, a necessary step before starting clinical studies, by the Korean Ministry of Food and Drug Safety. Patients…
The lives of four inspiring patients with bleeding disorders were introduced at the “Hope Ignites: Stories to Inspire” patient program at the Hemophilia Federation of America (HFA) annual symposium recently in Providence, Rhode Island. Sponsored by Octapharma USA, the “Hope Ignites” symposium was for patients with hemophilia to offer hope…
All of the hemophilia B patients in an ongoing Phase 1/2 clinical trial of SPK-9001 experienced clotting-factor increases and reductions in bleeding episodes, according to the therapy’s maker, Spark Therapeutics. University of Pennsylvania medical researcher Adam Cuker will present the updated preliminary results today at the Hemostasis and Thrombosis Research Society (HTRS) 2017…
Dimension Therapeutics’ gene therapy candidate drug DTX101 shows promising results by improving the levels of blood clotting factor IX (FIX) in adult patients with moderate/severe to severe hemophilia B, the company said. Hemophilia B is caused by FIX deficiency, due to mutations on the gene that encodes this protein. Treating…
Although gene therapy for hemophilia has reached clinical testing in humans, researchers continue to work toward improved delivery systems of the treatment — not an easy task considering the large size of the human factor VIII gene. In a study titled “Characterization of Adeno-Associated Viral Vector-Mediated Human Factor…
CSL Behring, a biotherapeutics firm based in King of Prussia, Pa., has awarded Local Empowerment for Advocacy Development (LEAD) grants of $10,000 each to four U.S. nonprofit groups that advocate for patients with hemophilia and other bleeding disorders. The funds will help these organizations make their voices heard on Capitol Hill…
South Korea has approved an Investigational New Drug (IND) application for Catalyst Biosciences’ experimental therapy CB 2679d/ISU304, a highly potent next-generation coagulation Factor IX variant for the treatment of hemophilia B. Catalyst focuses on developing medications for hematology conditions (blood disorders), which include coagulation factors that can prevent spontaneous…
Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP) received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) for treatment of adolescents and adults with hemophilia B. The CHMP, under the European Medicines Agency (EMA), recommends the granting of a marketing authorization in Europe for…
An infant was diagnosed with severe hemophilia A after developing splenic injury (injury to the spleen), a rare condition among newborns. For the first time, however, a newborn with these conditions was successfully treated with recombinant factor VIII replacement therapy without the need for surgical intervention, doctors reported. The report, titled…
