JP2018516566A5 - - Google Patents

Info

Publication number

JP2018516566A5

JP2018516566A5 JP2017560327A JP2017560327A JP2018516566A5 JP 2018516566 A5 JP2018516566 A5 JP 2018516566A5 JP 2017560327 A JP2017560327 A JP 2017560327A JP 2017560327 A JP2017560327 A JP 2017560327A JP 2018516566 A5 JP2018516566 A5 JP 2018516566A5

Authority

JP

Japan

Prior art keywords

seq

polypeptide

enpp1

pharmaceutical composition

residues

Prior art date

2016-05-19

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.)

Granted

Links

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• 229920001184 polypeptide Polymers 0.000 claims description 37
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 37
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 37
• 150000001875 compounds Chemical class 0.000 claims description 27
• 239000012634 fragment Substances 0.000 claims description 24
• 208000004434 Calcinosis Diseases 0.000 claims description 10
• 230000002308 calcification Effects 0.000 claims description 10
• 239000002243 precursor Substances 0.000 claims description 9
• 102000004169 proteins and genes Human genes 0.000 claims description 8
• 108090000623 proteins and genes Proteins 0.000 claims description 8
• 108010076504 Protein Sorting Signals Proteins 0.000 claims description 7
• XPPKVPWEQAFLFU-UHFFFAOYSA-J diphosphate(4-) Chemical compound [O-]P([O-])(=O)OP([O-])([O-])=O XPPKVPWEQAFLFU-UHFFFAOYSA-J 0.000 claims description 7
• 210000000988 bone and bone Anatomy 0.000 claims description 6
• 230000008685 targeting Effects 0.000 claims description 6
• 150000001413 amino acids Chemical class 0.000 claims description 5
• 230000000747 cardiac effect Effects 0.000 claims description 5
• 208000020832 chronic kidney disease Diseases 0.000 claims description 5
• 235000011180 diphosphates Nutrition 0.000 claims description 5
• FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 4
• 102000008100 Human Serum Albumin Human genes 0.000 claims description 4
• 108091006905 Human Serum Albumin Proteins 0.000 claims description 4
• 229920000805 Polyaspartic acid Polymers 0.000 claims description 3
• 201000000523 end stage renal failure Diseases 0.000 claims description 3
• 108010064470 polyaspartate Proteins 0.000 claims description 3
• 206010051714 Calciphylaxis Diseases 0.000 claims description 2
• 208000025500 Hutchinson-Gilford progeria syndrome Diseases 0.000 claims description 2
• 208000007932 Progeria Diseases 0.000 claims description 2
• 208000028208 end stage renal disease Diseases 0.000 claims description 2
• 101000812677 Homo sapiens Nucleotide pyrophosphatase Proteins 0.000 claims 20
• 102100039306 Nucleotide pyrophosphatase Human genes 0.000 claims 20
• 101000995829 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) Nucleotide pyrophosphatase Proteins 0.000 claims 20
• 239000008194 pharmaceutical composition Substances 0.000 claims 19
• KUBWJGWIWGGEPZ-UHFFFAOYSA-N 1-[amino(ethoxy)phosphoryl]oxy-4-nitrobenzene Chemical compound CCOP(N)(=O)OC1=CC=C([N+]([O-])=O)C=C1 KUBWJGWIWGGEPZ-UHFFFAOYSA-N 0.000 claims 6
• 101100225890 Aplysia californica ENPP gene Proteins 0.000 claims 6
• 102100021977 Ectonucleotide pyrophosphatase/phosphodiesterase family member 2 Human genes 0.000 claims 6
• 101000897035 Homo sapiens Ectonucleotide pyrophosphatase/phosphodiesterase family member 2 Proteins 0.000 claims 6
• 101000995838 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) Nucleotide pyrophosphatase Proteins 0.000 claims 6
• 239000000203 mixture Substances 0.000 claims 6
• 102100036093 Ectonucleotide pyrophosphatase/phosphodiesterase family member 7 Human genes 0.000 claims 3
• 101000876377 Homo sapiens Ectonucleotide pyrophosphatase/phosphodiesterase family member 7 Proteins 0.000 claims 3
• 230000037396 body weight Effects 0.000 claims 2
• 210000004027 cell Anatomy 0.000 claims 2
• 230000002797 proteolythic effect Effects 0.000 claims 2
• 200000000007 Arterial disease Diseases 0.000 claims 1
• 108010009413 Pyrophosphatases Proteins 0.000 claims 1
• 102000009609 Pyrophosphatases Human genes 0.000 claims 1
• 102000011016 Type 5 Cyclic Nucleotide Phosphodiesterases Human genes 0.000 claims 1
• 108010037581 Type 5 Cyclic Nucleotide Phosphodiesterases Proteins 0.000 claims 1
• 125000003275 alpha amino acid group Chemical group 0.000 claims 1
• 210000001367 artery Anatomy 0.000 claims 1
• 239000003937 drug carrier Substances 0.000 claims 1
• 210000004962 mammalian cell Anatomy 0.000 claims 1
• 239000000546 pharmaceutical excipient Substances 0.000 claims 1
• 230000002035 prolonged effect Effects 0.000 claims 1
• 230000028327 secretion Effects 0.000 claims 1
• 238000000034 method Methods 0.000 description 19
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 15
• 201000010099 disease Diseases 0.000 description 8
• 208000005475 Vascular calcification Diseases 0.000 description 6
• 208000035475 disorder Diseases 0.000 description 6
• 208000037824 growth disorder Diseases 0.000 description 6
• 230000035772 mutation Effects 0.000 description 5
• 208000034970 Heterotopic Ossification Diseases 0.000 description 4
• 101150011046 NPP1 gene Proteins 0.000 description 4
• 101100080092 Phytophthora capsici NLP1 gene Proteins 0.000 description 4
• 230000001575 pathological effect Effects 0.000 description 4
• 101150071357 NPP2 gene Proteins 0.000 description 3
• 101100080097 Phytophthora capsici NLP2 gene Proteins 0.000 description 3
• 241000282326 Felis catus Species 0.000 description 2
• 101710163270 Nuclease Proteins 0.000 description 2
• 230000007062 hydrolysis Effects 0.000 description 2
• 238000006460 hydrolysis reaction Methods 0.000 description 2
• 201000008482 osteoarthritis Diseases 0.000 description 2
• 230000002265 prevention Effects 0.000 description 2
• 150000003839 salts Chemical class 0.000 description 2
• 238000007920 subcutaneous administration Methods 0.000 description 2
• RSEBUVRVKCANEP-UHFFFAOYSA-N 2-pyrroline Chemical compound C1CC=CN1 RSEBUVRVKCANEP-UHFFFAOYSA-N 0.000 description 1
• 208000037260 Atherosclerotic Plaque Diseases 0.000 description 1
• 101001098814 Dictyostelium discoideum 3',5'-cyclic-nucleotide phosphodiesterase regA Proteins 0.000 description 1
• 101001117086 Dictyostelium discoideum cAMP/cGMP-dependent 3',5'-cAMP/cGMP phosphodiesterase A Proteins 0.000 description 1
• 102000004190 Enzymes Human genes 0.000 description 1
• 108090000790 Enzymes Proteins 0.000 description 1
• 108090001126 Furin Proteins 0.000 description 1
• 102000004961 Furin Human genes 0.000 description 1
• 206010061218 Inflammation Diseases 0.000 description 1
• 208000009469 Ossification of Posterior Longitudinal Ligament Diseases 0.000 description 1
• 239000002253 acid Substances 0.000 description 1
• 238000003776 cleavage reaction Methods 0.000 description 1
• 208000011111 hypophosphatemic rickets Diseases 0.000 description 1
• 230000004054 inflammatory process Effects 0.000 description 1
• 201000002044 ossification of the posterior longitudinal ligament of spine Diseases 0.000 description 1
• 208000001040 ossification of the posterior longitudinal ligament of the spine Diseases 0.000 description 1
• 230000007030 peptide scission Effects 0.000 description 1
• ZVJHJDDKYZXRJI-UHFFFAOYSA-N pyrroline Natural products C1CC=NC1 ZVJHJDDKYZXRJI-UHFFFAOYSA-N 0.000 description 1
• 230000007017 scission Effects 0.000 description 1
• 238000006467 substitution reaction Methods 0.000 description 1
• 230000009885 systemic effect Effects 0.000 description 1