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Publication of AR108687A1publicationCriticalpatent/AR108687A1/en
Medicines That Contain Protein Lipid Enzymes And Other Medicines
(AREA)
Medicines Containing Material From Animals Or Micro-Organisms
(AREA)
Abstract
Se proporcionan composiciones y regímenes útiles en el tratamiento de la hemofilia A. Las composiciones incluyen virus adenoasociado recombinante (rAAV) con un potenciador de transtiretina y promotor que conduce la expresión de un Factor VIII humano. Reivindicación 1: Un virus adenoasociado recombinante (rAAV) útil como un agente terapéutico dirigido al hígado para la hemofilia A, donde dicho rAAV comprende una cápside de AAV y un genoma de vector allí empaquetado, donde dicho genoma de vector comprende: (a) una secuencia de repetición terminal invertida (ITR) 5’ AAV; (b) un potenciador de transtiretina (enTTR); (c) un promotor de transtiretina (TTR); (d) una secuencia codificadora que codifica un Factor VIII humano que tiene función de coagulación; (e) una ITR de 3’ AAV.Compositions and regimens useful in the treatment of hemophilia A are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter that drives the expression of a human Factor VIII. Claim 1: A recombinant adeno-associated virus (rAAV) useful as a liver-directed therapeutic agent for hemophilia A, wherein said rAAV comprises an AAV capsid and a packaged vector genome, wherein said vector genome comprises: (a) a inverted terminal repeat sequence (ITR) 5 'AAV; (b) a transthyretin enhancer (enTTR); (c) a transthyretin promoter (TTR); (d) a coding sequence encoding a human Factor VIII that has a coagulation function; (e) a 3 ’AAV ITR.
ARP170100975A2016-04-152017-04-17
GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A
AR108687A1
(en)
Enhanced vectors of adeno-associated virus factor viii less than 5.0 kb in length, which produce a functionally active factor viii polypeptide for the treatment of hemophilia a.