Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
AssigneeThe Regents of the University of Colorado
InventorsJan P KRAUS, Thomas MAJTAN, Erez BUBLIL
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Abstract
The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine β-synthase (CBS) to treat homocystinuria and other related diseases and disorders.
Claims36
A pharmaceutical composition comprising an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide comprising the amino acid sequence of SEQ ID NO:
- 2, wherein said CBS protein is a chemically or enzymatically cleaved or genetically engineered truncate thereof, that is covalently linked to at least one water-soluble polymer or mixture of such molecules.
The use of an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide comprising the amino acid sequence of SEQ ID NO:
- 2, wherein said CBS protein is a chemically or enzymatically cleaved or genetically engineered truncate thereof, that is covalently linked to at least one water-soluble polymer or mixture of such molecules, for the manufacture of a medicament for treating or ameliorating a disease, disorder, or condition associated with elevated homocysteine.
Use of an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide comprising the amino acid sequence of SEQ ID NO:
- 3, wherein said CBS protein is a naturally occurring, chemically cleaved or genetically engineered truncate thereof, that is covalently linked to at least one water-soluble polymer or mixture of such molecules, such as PEG, for the manufacture of a medicament for preventing a disease, disorder, or condition associated with elevated homocysteine.
A composition of matter comprising an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide comprising the amino acid sequence of SEQ ID NO:
- 2, wherein said CBS protein is a chemically or enzymatically cleaved or genetically engineered truncate thereof, that is covalently linked to at least one water-soluble polymer or mixture of such molecules.
Dependencies are inferred from the claim text.