Lysosomal Storage Disease Enzyme
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.
1 - 43 . (canceled)
44 . A composition comprising a mixture of recombinant human lysosomal acid lipase (LAL), the mixture comprising at least two human LAL selected from the group consisting of SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4 and SEQ ID NO:19.
45 - 68 . (canceled)
69 . A pharmaceutical formulation comprising a composition according to claim 44 in combination with a pharmaceutically acceptable carrier, diluent or excipient.
70 . The pharmaceutical formulation of claim 69 , wherein the formulation comprises at least one agent selected from the group consisting of trisodium citrate dehydrate, citric acid and human serum albumin.
71 . The pharmaceutical formulation of claim 69 , wherein the formulation is provided in an aqueous solution maintained at pH between about 5.6 and about 6.2.
72 . The pharmaceutical formulation of claim 71 , wherein the formulation is maintained at pH between 5.7 and 6.1.