Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…

  The difference in disease severity between Hemophilia A and B (HA, HB) was recently assessed in the study, “Clinical, Instrumental, Serological And Histological Findings Suggest That Hemophilia B May Be Less Severe Than Hemophilia A,” and published in Haematologica. HA and HB are caused by deficient clotting proteins,…

CSL Behring’s rVIII-SingleChain, a novel recombinant Factor VIII (rFVIII) treatment candidate, recently demonstrated effectiveness in surgery and in the control of bleeding in patients with severe hemophilia A. The Phase 1 to Phase 3 clinical trial results, recently published in the journal Blood, also demonstrated low annualized bleeding rates in patients…

Low-dose recombinant factor VIII lowered bleeding rates in Chinese children with hemophilia, and the earlier the preventive treatment began, the more effective it was in minimizing bleeding episodes, according to a new study. The research, “The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A:…

Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…

SCT800, a replacement therapy containing a man-made form of clotting factor VIII (FVIII), appears to be safe and effective at treating and preventing bleeds in adolescents and adults with severe hemophilia A, a study has found. The study, “Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted…

My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb. 28 — Rare Disease Day — by launching the world’s largest research repository of its kind to researchers and scholars. The program is opening to U.S. scientists and will expand to worldwide scientists in 2018.

The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…

Starting or switching to prophylactic, or preventive Hemlibra (emicizumab) may lead to a significant reduction in treatment-associated expenses for hemophilia A patients in the U.S., a real-world study reported. These findings highlight the cost-benefits of using Hemlibra — and may help future cost analysis studies in assessing new…

Researchers have found that adults with severe hemophilia A coupled with pre-existing joint disease benefit most from prophylaxis to improve function, quality of life, and activities, and to better manage pain. The study, “Effect of late prophylaxis in hemophilia on joint status: a randomized trial,” was published in…