JPWO2022174114A5 - - Google Patents

Info

Publication number

JPWO2022174114A5

JPWO2022174114A5 JP2023547794A JP2023547794A JPWO2022174114A5 JP WO2022174114 A5 JPWO2022174114 A5 JP WO2022174114A5 JP 2023547794 A JP2023547794 A JP 2023547794A JP 2023547794 A JP2023547794 A JP 2023547794A JP WO2022174114 A5 JPWO2022174114 A5 JP WO2022174114A5

Authority

JP

Japan

Prior art keywords

fusion protein

seq

mol

amino acid

acid sequence

Prior art date

2025-02-18

Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)

Pending

Links

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• 108020001507 fusion proteins Proteins 0.000 claims description 156
• 102000037865 fusion proteins Human genes 0.000 claims description 156
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 85
• 238000000034 method Methods 0.000 claims description 57
• 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims description 53
• 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims description 53
• 238000006467 substitution reaction Methods 0.000 claims description 30
• SQVRNKJHWKZAKO-UHFFFAOYSA-N beta-N-Acetyl-D-neuraminic acid Natural products CC(=O)NC1C(O)CC(O)(C(O)=O)OC1C(O)C(O)CO SQVRNKJHWKZAKO-UHFFFAOYSA-N 0.000 claims description 25
• SQVRNKJHWKZAKO-OQPLDHBCSA-N sialic acid Chemical compound CC(=O)N[C@@H]1[C@@H](O)C[C@@](O)(C(O)=O)OC1[C@H](O)[C@H](O)CO SQVRNKJHWKZAKO-OQPLDHBCSA-N 0.000 claims description 25
• NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims description 22
• 239000012634 fragment Substances 0.000 claims description 16
• 239000008194 pharmaceutical composition Substances 0.000 claims description 13
• 239000003814 drug Substances 0.000 claims description 12
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 11
• 201000002273 mucopolysaccharidosis II Diseases 0.000 claims description 8
• 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims description 8
• 229920002683 Glycosaminoglycan Polymers 0.000 claims description 6
• 108010047041 Complementarity Determining Regions Proteins 0.000 claims description 5
• 102000007238 Transferrin Receptors Human genes 0.000 claims description 5
• 108010033576 Transferrin Receptors Proteins 0.000 claims description 5
• 229920000045 Dermatan sulfate Polymers 0.000 claims description 3
• 229920002971 Heparan sulfate Polymers 0.000 claims description 3
• AVJBPWGFOQAPRH-FWMKGIEWSA-L dermatan sulfate Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@H](OS([O-])(=O)=O)[C@@H]1O[C@H]1[C@H](O)[C@@H](O)[C@H](O)[C@H](C([O-])=O)O1 AVJBPWGFOQAPRH-FWMKGIEWSA-L 0.000 claims description 3
• 229940051593 dermatan sulfate Drugs 0.000 claims description 3
• 239000000546 pharmaceutical excipient Substances 0.000 claims description 3
• 102000040430 polynucleotide Human genes 0.000 claims description 2
• 108091033319 polynucleotide Proteins 0.000 claims description 2
• 239000002157 polynucleotide Substances 0.000 claims description 2
• 230000003442 weekly effect Effects 0.000 claims description 2
• 239000000203 mixture Substances 0.000 claims 10
• 238000004519 manufacturing process Methods 0.000 claims 1
• 150000001413 amino acids Chemical class 0.000 description 4
• 102100026144 Transferrin receptor protein 1 Human genes 0.000 description 2
• 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 description 2
• 125000003607 serino group Chemical group [H]N([H])[C@]([H])(C(=O)[*])C(O[H])([H])[H] 0.000 description 2
• 238000012986 modification Methods 0.000 description 1
• 230000004048 modification Effects 0.000 description 1