Joint problems caused by chronic bleeding often trouble people with hemophilia, especially in the knees, ankles, and elbows. A paper by scientists in Spain and the U.S. reviewed existing literature on knee treatments for people with this disease, focusing on best current practices — including a preventive, if inconveniently lifelong, therapy — and future…

A recent clinical trial indicates that replacement therapy with plasma-derived factor VIII is associated with lower incidence for development of inhibitors in patients with severe hemophilia A. The study,“A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,” was published in The New England Journal of…

The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy fidanacogene elaparvovec for treating certain adults with moderate to severe hemophilia B. The therapy will be sold under the name Beqvez as it is in Canada, where it was approved for a similar indication this…

GeneVentiv Therapeutics has received a $2.5 million grant to conduct further preclinical tests on its one-time infusion gene therapy for hemophilia A and B patients with or without clotting factor inhibitors. The therapy, GENV-HEM, will be tested in a large study with a canine model of hemophilia, following…

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…

Intellia Therapeutics and Regeneron are planning to begin, by mid-year, the first-in-human trial testing a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for people with hemophilia B. The announcement of the planned launch of the Phase 1 clinical trial, in an Intellia financial report, follows the recent approval by…

The development of neutralizing antibodies, or inhibitors, against the clotting factor in replacement therapies administered to people with hemophilia may depend on the type of product used, a recent study has found. In previously untreated people with severe hemophilia A, or those who lack…

Hemgenix (etranacogene dezaparvovec), the first and only one-time gene therapy approved for hemophilia B, has been approved in Switzerland, CSL Behring, the company marketing the therapy globally, announced. “The approval of Hemgenix in Switzerland marks an important milestone for patients, and we look forward to collaborating with…

Health Canada has approved the gene therapy Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely on routine prophylactic therapies to prevent or reduce bleeding episodes. Hemgenix is now the first and only gene therapy available to this patient group. “The approval of Hemgenix in Canada…