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US 11,560,554 B2Granted
Lysosomal storage disease enzymes
AssigneeAlexion Pharmaceuticals, Inc.
InventorsAnthony Quinn, Alex J. Harvey
Life of this patent
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Abstract
The present invention provides compositions of recombinant human lysosomal acid lipase having particular glycosylation patterns for internalization into target cells, a vector containing the nucleic acid encoding human lysosomal acid lipase, a host cell transformed with the vector, pharmaceutical compositions comprising the recombinant human lysosomal acid lipase and method of treating conditions associated with lysosomal acid lipase deficiency.
Claims13
A nucleic acid vector comprising a nucleic acid sequence encoding human recombinant lysosomal acid lipase comprising the amino acid sequence of SEQ ID NO:
- 2, 3, 4, or 19, wherein the nucleic acid sequence is operably linked to a promoter and wherein the nucleic acid sequence comprises an adenine (A) at position 93 relative to SEQ ID NO:
- 5.
Dependencies are inferred from the claim text.