Targeted enzyme compounds and uses thereof
AssigneeAngiochem Inc.
InventorsDominique Boivin, Jean-Paul Castaigne, Michel Demeule
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Abstract
The present invention is related to a compound that includes (a) α-L-iduronidase (IDUA), fragment, or analog thereof and (b) a targeting moiety, for example, where compound is a fusion protein including IDUA and Angiopep-2. In certain embodiments, these compounds, owning to the presence of the targeting moiety can crossing the blood-brain barrier or accumulate in the lysosome more effectively than the enzyme alone. The invention also features methods for treating mucopolysaccharidosis type I (MPS-I) using such compounds.
Claims33
A compound comprising (a) a peptide or peptidic targeting moiety less than 150 amino acids and (b) an IDUA enzyme, an active fragment thereof, or an analog thereof, wherein said targeting moiety and said enzyme, fragment, or analog are joined by a linker.
Dependencies are inferred from the claim text.