Patent ID: 9206401
Filing Date: 2015-12-08
Classification: A61K,A61P,C07K,C12N,C12Y

Abstract:
1. A method for treating a human subject diagnosed with a mucopolysaccharidoses (MPS) pathology, comprising administering to the subject a suitable amount of a pharmaceutical composition comprising a viral vector, via a systemic route of administration, wherein the MPS pathology is MPS type IIIA, wherein the viral vector comprises a recombinant plasmid suitable for gene therapy of MPS type IIIA, wherein the recombinant plasmid comprises a nucleotide sequence encoding for a chimeric sulfatase, said chimeric sulfatase consisting essentially of, in the N-terminal to C-terminal sequence order of: a) a signal peptide derived from either the human α-antitrypsin (hAAT) amino acid sequence or the human Iduronate-2-sulfatase (IDS) amino acid sequence; b) a human sulfatase derived amino acid sequence deprived of its signal peptide; c) the ApoB LDLR-binding domain; and wherein the human sulfatase is human sulfamidase.