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Fidanacogene elaparvovec is a novel, investigational vector that contains a bio-engineered adeno-associated virus capsid (protein shell) and a high-activity human coagulation factor IX gene.
The company added the US FDA has approved 3500 IU (international unit) vial size for IDELVION [Coagulation Factor IX (Recombinant), Albumin Fusion Protein (rFIX-FP)] for treating hemophilia B.
Visit Hemophilia Treatment Market by Drug Type (Antifibrinolytic Agents, Desmopressin, Plasma Derived Coagulation Factor Concentrates - Factor VIII, Factor IX, Combination, Recombinant Coagulation Factor Concentrates - Factor VIII, Factor IX, Combination), Indication Type (Acquired Hemophilia, Hemophilia A, Hemophilia B, Hemophilia C), and Distribution Channel (E-commerce, Hospital Pharmacies, Retail Pharmacies) and Forecast 2017-2021 at https://www.ihealthcareanalyst.com/report/hemophilia-treatment-market/
In one of our earlier studies we had reported 45 documented cases with 36 (81%) factor VIII deficiency, 8 (17%) factor IX deficiency and one factor X deficiency case.
In April Maurits, an engineering designer, joined a study in which he was dosed with viruses packed with a correct version of the gene that codes for factor IX. The Philadelphia company that ran the gene-therapy study, Spark Therapeutics, reported that in all four patients, factor IX activity has reached about 30 percent of average.
Elimination of antibody is considerably difficult and treatment success is poor in patients with hemophilia B who are allergic against Factor IX concentrates and who develop antibody.
Alprolix (rFIXFc) is an extended half-life recombinant factor IX Fc fusion protein product candidate for people with haemophilia B.
Background: Haemophilia B is X-linked recessive inherited disorder of factor IX deficiency.
The British patients were injected with an intact version of the human gene for the clotting agent known as Factor IX. The replacement gene was loaded onto a virus that introduced it into the human cells, thereby replacing the defective gene.
Patients must receive preventive injections of a clotting compound called factor IX to prevent bleeding from cuts, scratches or bruises.
The current study used adeno-associated virus (AAV) 8 as the vector to deliver the Factor IX gene along with additional genetic material into the patient's liver.