Patricia Inácio, PhD,  science writer—

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 notable…

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…

BrightInsight has launched a web-based dosing calculator to help physicians select the correct loading and maintenance dose of the treatment Hemlibra according to the body measurements of their patients with hemophilia A. The dosing calculator, launched with the support of Hemlibra’s manufacturer, Roche, will support the…

Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. These results were shared at the 2019 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, in a…

ASC Therapeutics has gotten exclusive rights to Expression Therapeutics‘ technology to develop liver-targeting hemophilia A gene therapies. “We have a common mission with Expression Therapeutics — to develop novel therapies that will dramatically improve the lives of persons with hemophilia around the world. Hemophilia A is a…

Trio Health has launched a novel database with real-time clinical data of patients with hemophilia which combines information from medical doctors and pharmacies. The main aim of the database is to provide information on the treatment regimen to physicians in order to improve patients’ outcomes. Access to the database…

Investigational gene therapy SB-525 is well-tolerated and leads to a dose-dependent increase in factor VIII (FVIII), according to interim results from eight patients with severe hemophilia A enrolled in the Phase 1/2 Alta clinical trial. Data showed that patients who received the highest dose of SB-525 — 3e13 vg/kg dose — reached…

New evidence suggests that a parameter called FVIII-EC50 has potential as a new way to predict the response of an individual with severe hemophilia to preventive treatment with FVIII, according to a small study. The study “Evaluation of EC50 of factor VIII as predictor of prophylaxis efficacy in patients…