Catarina Silva, MSc,  —

A single administration of AMT-061, an investigational gene therapy for people with severe and moderately severe hemophilia B, increased therapeutic levels of factor IX (FIX) in all patients enrolled in uniQure’s ongoing Phase 2b trial, the company announced. Ultimately, this trial’s purpose is to confirm the dose of AMT-061…

Sangamo Therapeutics has dosed the first patient in a Phase 1/2 clinical trial testing its investigational genome-editing therapy, SB-FIX, for patients with severe hemophilia B. The Georgetown University and MedStar Georgetown University Hospital are conducting the study, which is still recruiting participants in nine sites across the United…

Idelvion (albutrepenonacog alfa), a hemophilia B treatment approved for up to 14-day dosing, is now available in a large, 3500 IU vial size (3500 IU),…

uniQure has treated the first patient in a Phase 2b dose-confirmation study of its investigational gene therapy, AMT-061, for people with severe and moderately severe hemophilia B. The trial is still recruiting participants. AMT-061 uses a viral vector, AAV5, to deliver the gene for a mutated clotting factor…

The Hemophilia Federation of America (HFA) is seeking a variety of historical artifacts and records to add to its archival project in honor of its upcoming 25th year of helping the bleeding disorders community. The nonprofit is collecting vintage medical equipment, supplies, rally posters, newsletters, documents, personal journals, diaries, and…

BioMarin Pharmaceutical Inc. is partnering with digital content agency Believe Limited to produce a three-day musical workshop for young members of the bleeding disorders community. The “Breaking Through!” musical theater intensive, the first program of its kind, will be held Nov. 9-12 in New York City, and aims at teaching…

UniQure has enrolled the first patient in its Phase 3 HOPE-B single-dose trial investigating the effectiveness and safety of AMT-061 in treating people with severe or moderately severe hemophilia B. AMT-061 uses a viral vector to deliver the gene for a mutated clotting factor IX (FIX), which is also known as…

In honor of Bleeding Disorder Awareness Month, celebrated in March, CVS Health is donating more than $260,000 to national and local hemophilia and bleeding disorder programs across the United States. Although there’s no exact number of how many people are now living…

The Oklahoma Medical Research Foundation (OMRF) and Shanghai RAAS Blood Products are working together on the development of new hemophilia and traumatic bleeding therapies. This is the first time OMRF has collaborated with a Chinese blood products manufacturer. In 2017, Shanghai RAAS was 25th on Forbes’ “Growth Champions”…

Researchers at Emory University School of Medicine have developed a small-scale model system to study bleeding and clotting of wounds. The miniature model also accurately reproduces the alterations observed in hemophilia A patients. Their work, “A microengineered vascularized bleeding model that integrates the principal components of hemostasis”, was published in…