A Phase 1/2 clinical trial on SB-FIX, an investigative gene-editing therapy for hemophilia B, is now authorized to enroll adult and adolescent patients in the U.K., Sangamo Therapeutics announced. Approval from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) will make this the first genome-editing study conducted in Europe. Once safety…
The European Commission has approved Hemlibra (emicizumab) for routine preventive treatment of bleeding episodes in people who have hemophilia A with factor VIII inhibitors. The antibody therapy is designed to restore the factor VIII, which is deficient in hemophilia A. Hemlibra is being co-developed by Japan’s Chugai Pharmaceuticals, Switzerland’s…
Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare disease…
AGC Biologics has agreed to produce the hemophilia B clotting therapy that Catalyst Biosciences is testing in clinical trials, the companies announced. The next step in testing CB 2679d will be a Phase 2b trial in the third quarter of 2018, according to Catalyst, which is based in South San Francisco.
In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…
A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…
Eloctate use in immune tolerance induction (ITI) therapy shows promise in high-risk patients with severe hemophilia A and inhibitors treated for the first time, Bioverativ announced. The results support a potential benefit for some patients who tried and failed ITI with other factors. The study, “Recombinant factor VIII…
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”…
Shire Korea recently received approval from South Korea’s Ministry of Food and Drug Safety for Adynovate (BAX 855), a long-lasting factor VIII gene recombinant therapy for hemophilia A. Adynovate is an injectable form of Shire’s Advate, which is Korea’s most commonly used treatment for the blood disorder. Shire Korea…
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…
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