Gentle electrical stimulation may help liver cells uptake gene therapies, such as those used to treat hemophilia, according to new research by a team of U.S. scientists. In lab studies, the team found that liver cells exposed to a brief electrical field more easily took up a viral carrier,…
The U.S. Food and Drug Administration (FDA) has approved Labcorp’s companion diagnostic test for detecting preexisting antibodies against the viral carrier that’s used in Beqvez (fidanacogene elaparvovec-dzkt), Pfizer’s recently approved gene therapy for hemophilia B. A negative result on an FDA-cleared test is needed to be eligible…
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…
Ixinity (trenonacog alfa) has been approved by the U.S. Food and Drug Administration (FDA) for use in children younger than 12 with hemophilia B. This label expansion by the FDA means that the factor IX (FIX) replacement therapy, sold by Medexus Pharmaceuticals, can now be used in…
Note: This story was updated March 28, 2024, to clarify the gene therapy delivers the mRNA of the Cas9 enzyme. Intellia Therapeutics is ending its collaborative agreement with Regeneron, which had the two companies working together in developing hemophilia B gene-editing therapies, according to a securities and exchange…
Almost three years of treatment with SerpinPC was associated with a nearly complete resolution of bleeds and a reduction in the number of affected joints among men with severe hemophilia A or B who participated in a Phase 1/2a clinical trial. The…
Adding an antibody targeting the anticoagulant protein S to factor IX (FIX) replacement therapy accelerated blood clotting in samples from children with hemophilia B, including those with severe disease, a study showed. Greater production of the blood clotting protein thrombin also was seen in these samples, while blocking…
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…
Impairments in joint health are associated with certain functional deficits in the lower limbs among people with mild hemophilia, a study reports. A combination of aging, pain, and joint damage had a significant influence on patients’ performance in tests of muscle power, while aging alone explained most variation in…
Preventive treatment with Eloctate (efmoroctocog alfa) for up to five years led to sustained reductions in all bleed types for children, adolescents, and adults with severe hemophilia A, according to data from two Phase 3 trials and their open-label extension study. New analyses of these pivotal trials also…
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