News

Gene Therapy Paper on Repair in Hemophilia B Reviewed

Researchers at the Center for Research in Transplantation and Immunology in France have reviewed a paper concerning certain strategies utilized to correct hemophilia B and the importance of gene delivery vehicles in successful therapies for treatment of the condition. The review paper, “Successful correction of hemophilia by CRISPR/Cas9 genome…

European Commission Approves Hemophilia B Therapy Alprolix Extended Protection

The biotech companies Swedish Orphan Biovitrum AB (Sobi) and Biogen have announced that the European Commission (EC) has approved their extended half-life therapy Alprolix (rFIXFc) for hemophilia B in all 28 European Union (EU) countries. In addition, the EC maintained the drug’s orphan designation. The World Federation of Hemophilia (WFH) estimated…

Very Rare Case of Acquired Hemophilia A and Associated Myelofibrosis Reported in Patient

Researchers at the Centre Hospitalier Metropole Savoie in France identified a rare case of associated myelofibrosis (MF) and acquired hemophilia A (AHA), which they believe to be only the second case reported to date. The study, “Myelofibrosis and acquired hemophilia A: a case report,” was published in the…

Dimension Announces Positive Preclinical Results for an Adeno-Associated Virus Delivery of Factor VIII in Hemophilia A Treatment

Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of…