Catalyst Biosciences, developers of protease therapeutics for serious hemostasis and anti-complement conditions, and CMC ICOS Biologics, Inc., which manufactures therapeutic proteins, have partnered for the manufacture of a next generation hemophelia drug. Catalyst is developing CB 813d, a longer-acting Factor VIIa drug for hemophilia A and B inhibitor patients. “We selected CMC Biologics…

The National Hemophilia Foundation (NHF) and Impact Education, LLC (IMPACT) have jointly announced the fourth annual National Hemophilia Foundation Annual Meeting pre-conference symposium has been specifically designed for pharmacists — including specialty, home health, health plan, and managed Medicaid pharmacists — as well as for medical directors and other…

CSL Behring, a global biotherapeutics company focused on the development and delivery of innovative therapies that are used to treat coagulation disorders, announced this week that the European Commission has approved Idelvion (albutrepenonacog alfa), its long-acting albumin fusion protein for the treatment of bleeding in patients with hemophilia B…

A team led by Lurie Children’s Hospital of Chicago researchers reported a case study of a newborn with combined spontaneous splenic rupture and severe hemophilia A. This baby, whose case is believed to be the first documented, was successfully treated without surgical intervention. Results from the study, “Successful medical management of a…

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…

The biotech companies Swedish Orphan Biovitrum AB (Sobi) and Biogen have announced that the European Commission (EC) has approved their extended half-life therapy Alprolix (rFIXFc) for hemophilia B in all 28 European Union (EU) countries. In addition, the EC maintained the drug’s orphan designation. The World Federation of Hemophilia (WFH) estimated…

Researchers at the Centre Hospitalier Metropole Savoie in France identified a rare case of associated myelofibrosis (MF) and acquired hemophilia A (AHA), which they believe to be only the second case reported to date. The study, “Myelofibrosis and acquired hemophilia A: a case report,” was published in the…

Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of…

Starting Hemlibra (emicizumab) early is safe and helps to prevent bleeding in babies with severe hemophilia A who are untreated or minimally treated, though doctors should still monitor for self-reactive antibodies that can make other treatments less effective, a study suggested. The study, “Emicizumab in Previously Untreated…

Data from non-human primates suggest gene-editing therapy MGX-001 may be able to restore clinically meaningful levels of factor VIII (FVIII) after a single dose, supporting its potential as a one-time treatment for hemophilia A. Developer Metagenomi expects to meet with health authorities later this year to develop a strategy for…