Marisa Wexler, MS,  senior science writer—

A single dose of Freeline Therapeutics’ experimental gene therapy FLT180a increased the levels of factor IX (FIX) — the missing clotting protein in hemophilia B — for up to around 3.5 years in nine out of 10 men with moderate-to-severe disease. That’s according to data from the Phase…

Most people with hemophilia A or B who switched from an approved prophylactic (preventive) therapy regimen to once-monthly treatment with fitusiran in the ATLAS-PPX clinical trial had no bleeds over seven months on the investigational therapy, new data show. Fitusiran also reduced bleeding rates regardless of the presence…

The first three participants in the B-LIEVE trial all have factor IX (FIX) levels within the normal range more than one month after being treated with FLT180a, Freeline’s experimental gene therapy for hemophilia B, according to the company. “The initial data show that FLT180a provides rapid and consistent…

Once-daily preventive treatment with Novo Nordisk’s experimental therapy concizumab significantly reduced bleeding rates in people with both hemophilia A and B with inhibitors, according to new data from the Phase 3 explorer7 trial. In fact, bleeding rates in patients receiving concizumab in trial decreased by more than 85%. Based…

Most people with moderate or mild hemophilia A who received preventive treatment with Hemlibra (emicizumab) in the HAVEN 6 clinical trial had no bleeds requiring treatment over a median follow-up of over a year, new data show. The findings were presented at the 30th International Society on Thrombosis and…

Japan’s Ministry of Health, Labor and Welfare has extended Hemlibra (emicizumab)’s approval to include routine prophylaxis to prevent or lower the frequency of bleeds in people with acquired hemophilia A. Its decision comes seven months after Hemlibra’s maker, Chugai Pharmaceutical, applied for extended use. Hemlibra was previously approved in…

Dosing has begun in a second group of hemophilia B patients enrolled in a Phase 1/2 dose-confirmation trial of the experimental gene therapy FLT180a, the treatment’s developer, Freeline, announced. The dose determined to be optimal in this study is expected to finalize plans for a pivotal Phase 3…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given by the FDA to help speed the development and review of treatments for serious or life-threatening conditions. Specifically,…

Katherine High, MD, has received the gold medal from Children’s Hospital of Philadelphia (CHOP) in recognition of her work to advance gene therapies for hemophilia and other genetic disorders. In CHOP’s 166-year history, this award — to recognize the most significant achievements in improving the health of children —…

Roche has extended its partnership with the World Federation of Hemophilia (WFH) to expand access to treatments for hemophilia through the end of 2028, the company announced. Under the partnership, Roche will continue to provide its prophylactic (preventive) treatment for hemophilia A to the WFH’s Humanitarian…