LYSOSOMAL DISEASE ACCUMULATION ENZYME Russian patent published in 2021 - IPC C12N9/20 

FIELD: biotechnology; medicine.

SUBSTANCE: invention represents an isolated recombinant human lysosomal acid lipase glycoprotein (rLLCLCH), wherein the pGLCLCH essentially consists of an amino acid sequence, selected from the group consisting of SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4 and SEQ ID NO: 19, with N-glycans bound to aspartic residues (Asn), which correspond to Asn36, Asn101, Asn161, Asn273 and Asn321 sequences SEQ ID NO: 1, provided that N-glycans do not contain sialic acid or fucose, and provided that the pGLLCL does not contain O-glycans, and where N-glycans linked to Asn residues which correspond to Asn36, Asn101, Asn161, Asn273 and Asn321 sequences SEQ ID NO: 1, are: a) in position Asn36, GlcNAc4Man3GlcNAc2, or Gal1GlcNAc4Man3GlcNAc2; b) in position Asn101, Phos2Man7GlcNAc2; b) in position Asn161, Phos1Man6GlcNAc2; GlcNAc1Phos1Man6GlcNAc2; Man3GlcNAc2; GlcNAc2Man3GlcNAc2; GlcNAc3Man3GlcNAc2; GlcNAc4Man3GlcNAc2; or Gal1GlcNAc4Man3GlcNAc2; c) in position Asn273, Man7GlcNAc2; Man8GlcNAc2; Man9GlcNAc2; Phos1Man8GlcNAc2; or Phos1Man9GlcNAc2; and d) in position Asn321, GlcNAc2Man3GlcNAc2; GlcNAc3Man3GlcNAc2; GlcNAc4Man3GlcNAc2; Gal1GlcNAc4Man3GlcNAc2; GlcNAc5Man3GlcNAc2; Gal1GlcNAc5Man3GlcNAc2; GlcNAc6Man3GlcNAc2; or Gal1GlcNAc6Man3GlcNAc2; where Man = mannose, GlcNAc = N-acetylglucosamine, Phos = phosphate, and Gal = galactose. Invention also refers to pharmaceutical compositions containing recombinant human lysosomal acid lipase.

EFFECT: invention is used for effective treatment of conditions associated with deficiency of lysosomal acid lipase.

12 cl, 30 dwg, 6 tbl, 20 ex