Sangamo Therapeutics recently presented what it characterized as key improvements to its technology platform for engineering highly specific zinc finger nucleases (ZFNs), which are used for targeted editing of the human genome. This technique has broad applicability, namely for the treatment of hemophilia B and other disorders. The…
News
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…
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 U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…
Cardiovascular Risk Factors Probably Not the Link Between Hypertension and Hemophilia, Study Says
The high incidence of hypertension among hemophilia patients may not be associated with common cardiovascular risk factors, such as obesity, cholesterol and smoking, according to a new study. The researchers call for more studies to uncover possible links between hypertension and hemophilia. The study, “The Hypertension Of Hemophilia Is…
Health Canada, the agency that oversees health decisions and regulations in Canada, has approved Afstyla (lonoctocog alfa) for the treatment and prevention of bleeding in adults and children with hemophilia A. In May, the U.S. Food and Drug Administration (FDA) approved Afstyla for the same indications. Developed by CSL Behring, Afstyla is…
Low-dose recombinant factor VIII lowered bleeding rates in Chinese children with hemophilia, and the earlier the preventive treatment began, the more effective it was in minimizing bleeding episodes, according to a new study. The research, “The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A:…
More than 1,300 people who have been affected by acquired immune deficiency syndrome (AIDS) gathered at the National AIDS Memorial in San Francisco on Dec. 1 to commemorate World AIDS Day. During two days of events, family and friends paid tributes to those lost to the disease, while inspiring remembrance…
Two presentations at the 2016 American Society of Hematology Annual Meeting (ASH) highlight ongoing treatment advances for hemophilia B. The data suggest that life with hemophilia could soon become easier to manage. Long-acting replacement therapy The ASH meeting, held in San Diego on Dec. 3-6, was a platform for CSL Behring to…
Researchers have successfully applied a potential gene therapy for hemophilia B based on the genome editing properties of the CRISPR/Cas9 system. The results of this study using mice were presented recently at the 58th Annual American Society of Hematology Meeting and Exposition in San Diego, Calif. Hemophilia B is a disease…
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