AMT-061 (etranacogene dezaparvovec), uniQure‘s investigational gene therapy for hemophilia B, is safe and effective — even in patients with pre-existing antibodies against the treatment’s viral carrier, according to new trial data. Data from the Phase 3 HOPE-B trial suggest that nearly all hemophilia B patients, regardless of pre-existing viral…

Hemophilia care providers in the U.K. have insufficient knowledge on how to assess the risk of prolonged bleeding associated with dental procedures, a study has found. The study, “U.K. hemophilia treaters’ knowledge of risk assessment for prolonged bleeding associated with dental procedures,” was published in the journal…

Xenetic Biociences has received a $3 million milestone payment from its partner Shire for advancing the Phase 1/2 clinical trial of PSA-recombinant SHP656, a factor VIII protein, evaluating the drug’s effectiveness as a long-acting therapy for hemophilia A. Shire’s main objective behind the agreement is to develop a novel FVIII…

Should regulatory authorities agree with favorable results from Genentech’s ongoing Phase 3 studies of Hemlibra (emicizumab-kxwh),  hemophilia A patients may have a treatment they can use regardless of inhibitor status, with a choice of three dosing schedules that might better match their lifestyle and preferences. “The ultimate goal for…

Bayer announced that six hemophilia clinicians and researchers in the United States were among 16 grant recipients under the company’s Hemophilia Awards Program for 2016. The announcement was part of the World Federation of Hemophilia 2016 World Congress now taking place in Orlando, Florida. Grant amounts for the six were not given,…

AMT-060, a first-generation gene therapy candidate by uniQure, continues to safely reduce the number of bleeding episodes and the use of clotting factor IX (FIX) in men with moderate to severe hemophilia B, long-term data from a Phase 1/2 clinical trial show. The therapy also prevented…

CAR T-cell therapy, a type of treatment that weaponizes the body’s immune system and was originally developed to treat cancer, for the first time successfully treated a patient with acquired hemophilia A (AHA), a study reported. The study, “Anti-CD19 CAR-T cell therapy for acquired hemophilia A,”…

Welcome to 2022, a new year and a clean slate. Year after year, my New Year’s resolutions are the same: eat a more healthy diet, work out, and lose weight. This year will be different. Recently, a nurse phoned from the specialty pharmacy that provides medication to treat my von…

The past two weeks have been a whirlwind of activity for me, highlighted by two significant events. First, I attended an event in Washington, D.C., called “Voices of Hemophilia,” which was sponsored by the biotech company CSL Behring. This small but powerful gathering brought together eight leading voices from…