Margarida Maia, PhD, science writer —

HCB-101, hC Bioscience’s lead candidate to overwrite certain mutations that lead to hemophilia A, can reach the liver cells of mice, where it should then be able to drive the production of a working version of the blood-clotting protein that is faulty or missing in the disease. Such…

Added to steroids, rituximab seems to be as safe and effective as cyclophosphamide therapy at controlling bleeds and wiping out antibodies against clotting factor VIII (FVIII) in adults with newly diagnosed acquired hemophilia A (AHA), according to a Phase 3 clinical trial. However, data from the CREHA…

Immune tolerance induction (ITI), a type of treatment that’s designed to eliminate neutralizing antibodies, or inhibitors, against clotting factors for people with hemophilia A, may work just as well across different racial and ethnic groups, a study finds. The findings don’t “support the hypothesis that ITI response varies according…

ReciBioPharm and GeneVentiv Therapeutics are teaming up to advance the development of GENV-HEM, a gene therapy for all hemophilia patients, including those who have developed inhibitors against their missing clotting factors. Developing a gene therapy requires efficient, flexible, and fast manufacturing, and each step…

Switching to Jivi (damoctocog alfa pegol), an extended half-life replacement therapy, from Kovaltry (octocog alfa), may help adults and children with severe hemophilia A experience fewer or no bleeds, according to a Canadian study. “Data from this study provide routine clinical evidence supporting the use of [Jivi]…

Sarcopenia, or loss of muscle mass and strength, may occur in as many as half of adults with severe hemophilia A who experience recurrent joint bleeds, according to a small study in Turkey. In addition, measuring muscle mass in the thighs with ultrasound, which is a noninvasive imaging test…

A real-world study in Spain found that, among hemophilia patients, those with hemophilia A may have have worse joint health — and more joint damage, particularly in the ankles — than those with hemophilia B. Importantly, though, such damage appeared to be less frequent for individuals on regular…

Individually tailored prophylaxis, or preventive treatment, with Jivi (damoctocog alfa pegol) nearly halved the number of annual bleeding episodes in people with severe hemophilia A who had previously been on another replacement therapy, according to a Phase 4 post-marketing study. Researchers also observed that none of the patients…

The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as…

SerpinPC, an experimental treatment being developed by Centessa Therapeutics, continues to be safe when used for up to nearly three years and to almost zero out the number of bleeds in adults with severe hemophilia A or B. Those findings come from a one-year extension of the…