Patricia Inácio, PhD, science writer —

Bone defects were seen since birth — and following injury — in mouse models of hemophilia A and B, but not in Von Willebrand (VWD) disease mice, a study shows. Researchers said further study into the potential mechanisms of primary bone deficits in hemophilia may help in…

A preventive treatment for hemophilia B called dalcinonacog alfa (DalcA) led to an increase of more than 12% in the levels of factor IX in two patients with severe disease treated in a Phase 2 trial, reaching the study’s main goal. Researchers also found no immune reaction against the therapy. Enrollment…

Data from the first two patients treated with FLT180a, Freeline’s single-infusion gene therapy for patients with hemophilia B, showed a rise in levels of clotting factor IX to 40%, with levels remaining stable for over a year. Pratima Chowdary, principal investigator for the FLT180a program, presented the data…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…

Genetic variations in certain immune-related genes, namely the HLA and IL-10 genes, are linked with an increased risk for developing inhibitors against factor VIII replacement therapies in patients with severe hemophilia A. The study that determined that finding, “Risk stratification integrating genetic data for factor VIII inhibitor development…

The presence of inhibitors and excessive body weight increase the sense of disease burden felt by parents of children with moderate to severe hemophilia, a study reports. The study “Burden on parents of children with hemophilia: the impact of socio- demographic and child’s medical condition” was published in…

Casebia’s two-stage gene therapy approach was successful in delivering the human gene coding for clotting factor VIII (FVIII) in a mouse model of hemophilia A, leading to stable and increasing levels of the factor, researchers said. “Our scientific team, led by Alan Brooks, Ph.D., have achieved a…

Recommendations for vaccination practices for patients with hemophilia should be reassessed in an effort to minimize fear and emphasize the importance of vaccination to prevent infections, suggests a survey among physicians in Germany. Data from the PEDNET registry, a database of children with hemophilia, suggest that vaccination in close…

Bayer’s Jivi, an FVIII replacement therapy for hemophilia A, has a longer half-life and a slower clearance from blood circulation than Eloctate, and may protect against bleeding for longer periods, results of a Phase 1 trial in people with severe disease suggests. The study “Direct comparison of…

BioMarin Pharmaceutical’s investigative gene therapy, called valoctocogene roxaparvovec, is capable of sustained control of bleeding rate requiring factor VIII infusions by at least 92% in adults with severe hemophilia A, three-year data from a Phase 1/2 trial show. Moreover, a model predicts that the…