Last updated Dec. 10, 2024, by Susie Strachan Hemophilia has affected people from all walks of life, including royalty, athletes, actors, and advocates. It’s a rare genetic bleeding disorder that mostly affects males, while females are more likely to be carriers. Learn about some of the notable names…
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Hemophilia A is a rare genetic bleeding disorder that primarily affects males, with females more often being carriers. Treatments like Hemlibra have shown to reduce bleeding episodes in severe hemophilia A patients. Research efforts include a large genetic repository for hemophilia A and B, and gene therapies like SB-525 are being developed.
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When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to data from a Phase 1 clinical trial and its long-term extension study. Importantly, reductions in the number of bleeds…
My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb. 28 — Rare Disease Day — by launching the world’s largest research repository of its kind to researchers and scholars. The program is opening to U.S. scientists and will expand to worldwide scientists in 2018.
SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
A Sangamo Therapeutics‘ gene therapy program to potentially treat hemophilia A, called SB-525, has been cleared by the U.S. Food and Drug Administration (FDA) for clinical testing in adult patients. The company announced that the FDA approved its Investigational New Drug (IND) application for SB-525, a necessary first step…
The safety and efficacy of emicizumab in patients with severe hemophilia A (HA) were recently evaluated in a study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” published in The New England Journal Of Medicine. Hemophilia A is caused by a lack of blood clotting factor…
Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human primates used a research construct, not the gene therapy. Generation Bio has announced new data showing that its investigational non-viral gene therapy platform can increase levels of the clotting…
Hemophilia News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to hemophilia throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read stories of 2019. No.
A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety and effectiveness of AMT-061 in adult men with hemophilia B. Patient recruitment is continuing for an estimated total of 56…
Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…