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…

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…

I surprised myself when I was in Montreal for a hemophilia conference in 2022. I decided to stay an extra day after the event to explore the city. I have to say, I found the most amazing gluten-free French pastry shop! The croissants and pain au chocolat were to…

It’s finally happened: validation. The battle to get here was rough, but perseverance paid off. Seeing “Shellye, a patient with superior mesenteric artery syndrome (SMAS), presents for follow-up visit prior to surgical repair” written in my medical records made me want to cry. I felt the same way when…

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…

My last home had a great fence. It was a wall of concrete blocks surrounding my backyard and stood 7 feet high. With two dogs who bark at the wind blowing, the wall deterred them from jumping and kept some of their barking at bay. However, there was an iron…