P97-IDS fusion proteins
AssigneeBioasis Technologies, Inc.
InventorsTimothy Z. Vitalis, Reinhard Gabathuler
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Abstract
Provided are fusion proteins between p97 (melanotransferrin) and iduronate-2-sulfatase (IDS), and related compositions and methods of use thereof, for instance, to facilitate delivery of IDS across the blood-brain barrier (BBB) and/or improve its tissue penetration in CNS and/or peripheral tissues, and thereby treat and/or diagnose Hunter Syndrome (Mucopolysaccharidosis type II; MPS II) and related lysosomal storage disorders, including those having a central nervous system (CNS) component.
Claims20
A p97 (melanotransferrin) fusion protein, comprising an iduronate-2-sulfatase (IDS) polypeptide fused to the N-terminus of a p97 polypeptide fragment and an optional heterologous peptide linker (L) in between, wherein the p97 polypeptide fragment consists of the amino acid sequence having at least 80% sequence identity to DSSHAFTLDELR (SEQ ID NO:
- 14) and having transport activity.
A p97 (melanotransferrin) fusion protein comprising an iduronate-2-sulfatase (IDS) polypeptide fused to the C-terminus of a p97 polypeptide fragment and an optional heterologous peptide linker (L) in between, wherein the p97 polypeptide fragment consists of the amino acid sequence having at least 80% sequence identity to DSSHAFTLDELR (SEQ ID NO:
- 14) and having transport activity.
Dependencies are inferred from the claim text.