Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
AssigneeBiomarin Pharmaceutical Inc.
InventorsStuart Bunting, Peter Cameron Colosi, Erno Pungor
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Abstract
The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
Claims64
A pharmaceutical formulation comprising a recombinant AAV FVIII virus, a buffering agent, an isotonicity agent, a bulking agent and a surfactant.
A method of treating a subject suffering from hemophilia A comprising administering to said subject a therapeutically effective amount of a recombinant AAV FVIII virus.
The method of any one of claims 10-16, wherein said subject expresses at least about 5 IU/dl of functional Factor VIII protein 3 weeks or more after said administration.
A method of reducing bleeding time of a bleeding episode in a subject suffering from hemophilia A comprising administering to said subject, prior to said bleeding episode, a therapeutically effective amount of a recombinant AAV FVIII virus.
A method of increasing Factor VIII protein expression in a subject in need thereof comprising administering to said subject a recombinant AAV FVIII virus.
A method of treating a subject suffering from hemophilia A comprising the steps of (i) determining the absence of anti-AAV capsid antibodies in the serum of said subject, and (ii) administering to said subject a therapeutically effective amount of a recombinant AAV FVIII virus.
A method of treating a subject suffering from hemophilia A comprising the steps of (i) administering to said subject a therapeutically effective amount of a recombinant AAV FVIII virus, and (ii) after administration of said therapeutically effective amount of said recombinant AAV FVIII virus, determining the absence or presence of anti-AAV capsid antibodies in the serum of said subject.
Dependencies are inferred from the claim text.