Hemophilia gene therapy Beqvez (fidanacogene elaparvovec), marketed as Durveqtix in the European Union, results in stable production of factor IX (FIX), the clotting protein that is missing or faulty in hemophilia B, leading to about three times fewer bleeding episodes compared with prophylaxis, or preventive treatment, with regular infusions…
Almost two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce their quality of life, even when they’re on factor replacement therapy to reduce the frequency of bleeding episodes, a real-world study shows. “Overall, the data indicate that joint problems still…
Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for adults and children with hemophilia A, according to an analysis of three Phase 3 clinical trials. “These analyses highlight the relevance of pain, physical health, and overall wellbeing as…
A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis, or preventive therapy, for as long as seven years — the longest follow-up to date for any hemophilia A gene therapy clinical trial. While most of the 12 patients…
Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…
Artificial intelligence (AI) can comb through ultrasound scans to detect joint bleeding and inflammation occurring as a result of hemophilia, a study has found, suggesting it may help doctors make quicker and more accurate diagnoses. “AI models have the potential to aid diagnosis and enable earlier therapeutic interventions, helping…
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…
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