JP2018502836A5 - - Google Patents

Info

Publication number

JP2018502836A5

JP2018502836A5 JP2017529820A JP2017529820A JP2018502836A5 JP 2018502836 A5 JP2018502836 A5 JP 2018502836A5 JP 2017529820 A JP2017529820 A JP 2017529820A JP 2017529820 A JP2017529820 A JP 2017529820A JP 2018502836 A5 JP2018502836 A5 JP 2018502836A5

Authority

JP

Japan

Prior art keywords

composition

use according

alkaline phosphatase

subject

human subject

Prior art date

2015-12-04

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

• Espacenet
• Global Dossier
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• 102000002260 Alkaline Phosphatase Human genes 0.000 claims description 49
• 108020004774 Alkaline Phosphatase Proteins 0.000 claims description 49
• NGVDGCNFYWLIFO-UHFFFAOYSA-N pyridoxal 5'-phosphate Chemical compound CC1=NC=C(COP(O)(O)=O)C(C=O)=C1O NGVDGCNFYWLIFO-UHFFFAOYSA-N 0.000 claims description 13
• 235000007682 pyridoxal 5'-phosphate Nutrition 0.000 claims description 13
• 239000011589 pyridoxal 5'-phosphate Substances 0.000 claims description 13
• BTCSSZJGUNDROE-UHFFFAOYSA-N gamma-aminobutyric acid Chemical compound NCCCC(O)=O BTCSSZJGUNDROE-UHFFFAOYSA-N 0.000 claims description 12
• 230000003442 weekly effect Effects 0.000 claims description 12
• LXNHXLLTXMVWPM-UHFFFAOYSA-N pyridoxine Chemical compound CC1=NC=C(CO)C(CO)=C1O LXNHXLLTXMVWPM-UHFFFAOYSA-N 0.000 claims description 10
• 210000004556 brain Anatomy 0.000 claims description 8
• 239000003814 drug Substances 0.000 claims description 8
• 229940124597 therapeutic agent Drugs 0.000 claims description 8
• 206010010904 Convulsion Diseases 0.000 claims description 7
• 239000012634 fragment Substances 0.000 claims description 7
• OGNSCSPNOLGXSM-UHFFFAOYSA-N (+/-)-DABA Natural products NCCC(N)C(O)=O OGNSCSPNOLGXSM-UHFFFAOYSA-N 0.000 claims description 6
• 206010049933 Hypophosphatasia Diseases 0.000 claims description 6
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 6
• 229960003692 gamma aminobutyric acid Drugs 0.000 claims description 6
• 102100024321 Alkaline phosphatase, placental type Human genes 0.000 claims description 5
• 102100025683 Alkaline phosphatase, tissue-nonspecific isozyme Human genes 0.000 claims description 5
• 101000574445 Homo sapiens Alkaline phosphatase, tissue-nonspecific isozyme Proteins 0.000 claims description 5
• SUHOOTKUPISOBE-UHFFFAOYSA-N O-phosphoethanolamine Chemical compound NCCOP(O)(O)=O SUHOOTKUPISOBE-UHFFFAOYSA-N 0.000 claims description 5
• KAKKHKRHCKCAGH-UHFFFAOYSA-L disodium;(4-nitrophenyl) phosphate;hexahydrate Chemical compound O.O.O.O.O.O.[Na+].[Na+].[O-][N+](=O)C1=CC=C(OP([O-])([O-])=O)C=C1 KAKKHKRHCKCAGH-UHFFFAOYSA-L 0.000 claims description 5
• 230000000694 effects Effects 0.000 claims description 5
• 108010031345 placental alkaline phosphatase Proteins 0.000 claims description 5
• 229940011671 vitamin b6 Drugs 0.000 claims description 5
• 108060003951 Immunoglobulin Proteins 0.000 claims description 4
• 229960003965 antiepileptics Drugs 0.000 claims description 4
• 102000018358 immunoglobulin Human genes 0.000 claims description 4
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
• 230000002159 abnormal effect Effects 0.000 claims description 3
• 238000011260 co-administration Methods 0.000 claims description 3
• 238000001990 intravenous administration Methods 0.000 claims description 3
• RADKZDMFGJYCBB-UHFFFAOYSA-N pyridoxal hydrochloride Natural products CC1=NC=C(CO)C(C=O)=C1O RADKZDMFGJYCBB-UHFFFAOYSA-N 0.000 claims description 3
• 235000019158 vitamin B6 Nutrition 0.000 claims description 3
• 239000011726 vitamin B6 Substances 0.000 claims description 3
• 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 2
• YPWSLBHSMIKTPR-UHFFFAOYSA-N Cystathionine Natural products OC(=O)C(N)CCSSCC(N)C(O)=O YPWSLBHSMIKTPR-UHFFFAOYSA-N 0.000 claims description 2
• ILRYLPWNYFXEMH-UHFFFAOYSA-N D-cystathionine Natural products OC(=O)C(N)CCSCC(N)C(O)=O ILRYLPWNYFXEMH-UHFFFAOYSA-N 0.000 claims description 2
• ILRYLPWNYFXEMH-WHFBIAKZSA-N L-cystathionine Chemical compound [O-]C(=O)[C@@H]([NH3+])CCSC[C@H]([NH3+])C([O-])=O ILRYLPWNYFXEMH-WHFBIAKZSA-N 0.000 claims description 2
• MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims description 2
• 230000018678 bone mineralization Effects 0.000 claims description 2
• XPPKVPWEQAFLFU-UHFFFAOYSA-J diphosphate(4-) Chemical compound [O-]P([O-])(=O)OP([O-])([O-])=O XPPKVPWEQAFLFU-UHFFFAOYSA-J 0.000 claims description 2
• 235000011180 diphosphates Nutrition 0.000 claims description 2
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
• 208000035475 disorder Diseases 0.000 claims description 2
• 230000004927 fusion Effects 0.000 claims description 2
• 108020001507 fusion proteins Proteins 0.000 claims description 2
• 102000037865 fusion proteins Human genes 0.000 claims description 2
• 210000004602 germ cell Anatomy 0.000 claims description 2
• 230000003834 intracellular effect Effects 0.000 claims description 2
• 238000007918 intramuscular administration Methods 0.000 claims description 2
• 238000007913 intrathecal administration Methods 0.000 claims description 2
• 235000008160 pyridoxine Nutrition 0.000 claims description 2
• 239000011677 pyridoxine Substances 0.000 claims description 2
• 210000002966 serum Anatomy 0.000 claims description 2
• 210000000813 small intestine Anatomy 0.000 claims description 2
• 238000007920 subcutaneous administration Methods 0.000 claims description 2
• 230000002485 urinary effect Effects 0.000 claims description 2
• 235000020951 vitamin B6 vitamer Nutrition 0.000 claims description 2
• 239000011625 vitamin B6 vitamer Substances 0.000 claims description 2
• 239000000203 mixture Substances 0.000 claims 28
• 229920000805 Polyaspartic acid Polymers 0.000 claims 1
• 108010064470 polyaspartate Proteins 0.000 claims 1
• 238000000034 method Methods 0.000 description 47
• 230000001594 aberrant effect Effects 0.000 description 1
• 230000003247 decreasing effect Effects 0.000 description 1
• 238000012986 modification Methods 0.000 description 1
• 230000004048 modification Effects 0.000 description 1