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.
Claims6
A pharmaceutical formulation comprising a recombinant AAV FVIII virus, sodium phosphate at a concentration of from about 0.1 mg/ml to about 3 mg/ml, sodium chloride at a concentration of from about 1 mg/ml to about 20 mg/ml, mannitol at a concentration of from about 5 mg/ml to about 40 mg/ml, and poloxamer 188 at a concentration of from about 0.1 mg/ml to about 4 mg/ml.
Dependencies are inferred from the claim text.