TiumBio is preparing to launch a Phase 1b clinical trial to test TU7710, its experimental treatment to manage bleeding in people with hemophilia who have inhibitors, that is, neutralizing antibodies that can lower standard therapies’ effectiveness. “Our team has been developing TU7710 as an innovative hemophilia treatment option,”…
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…
In observance of Bleeding Disorders Awareness Month, the integrated infusion therapy provider BioTek reMEDys is calling attention to the importance of safe treatment use and education among people with conditions such as hemophilia and von Willebrand disease. Each March, the bleeding disorders community and supporters seek to heighten…
Nearly all medical directors at hemophilia treatment centers in the U.S. said they would consider starting preventive Hemlibra (emicizumab-KXWH) in untreated infants with hemophilia A, ages 12 months and younger, a survey study reports. Most respondents also said they’d transition from FVIII replacement therapies, used on-demand to treat…
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…
Hemlibra (emicizumab-KXWH) safely controlled bleeds among hemophilia A patients without inhibitors over nearly five years of preventive treatment, according to long-term data pooled from two Phase 3 clinical trials. “Five years of follow-up data showed a favorable benefit-risk profile of [Hemlibra] prophylaxis,” the researchers wrote in “…
Switching to Jivi (damoctocog alfa pegol), an extended half-life replacement therapy, from Kovaltry (octocog alfa), may help adults and children with severe hemophilia A experience fewer or no bleeds, according to a Canadian study. “Data from this study provide routine clinical evidence supporting the use of [Jivi]…
When used long term, Novoeight (turoctocog alfa) safely and effectively prevented and treated bleeding episodes in people with hemophilia A in Japan, according to a real-world study. In fact, Novoeight successfully treated 91.4% of the reported bleeds, and kept bleeding under control in 85.7% of surgical procedures. Moreover,…
March is Bleeding Disorders Awareness Month (BDAM) and patients, caregivers, advocates, and other supporters are coming together to call attention to conditions like hemophilia and von Willebrand disease that impact more than 3 million people in the U.S. Bleeding disorders are marked by uncontrolled bleeding that occurs from a…
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…
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