JP2018538288A5 - - Google Patents

Info

Publication number

JP2018538288A5

JP2018538288A5 JP2018529221A JP2018529221A JP2018538288A5 JP 2018538288 A5 JP2018538288 A5 JP 2018538288A5 JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018538288 A5 JP2018538288 A5 JP 2018538288A5

Authority

JP

Japan

Prior art keywords

mrna

pharmaceutical composition

item

composition according

nucleobases

Prior art date

2016-12-13

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.)

Ceased

Links

• Espacenet
• Global Dossier
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• 108020004999 messenger RNA Proteins 0.000 claims description 100
• 108090000623 proteins and genes Proteins 0.000 claims description 77
• 238000000034 method Methods 0.000 claims description 70
• 102000004169 proteins and genes Human genes 0.000 claims description 67
• 230000000717 retained effect Effects 0.000 claims description 62
• 230000000692 anti-sense effect Effects 0.000 claims description 58
• 101000994437 Homo sapiens Protein jagged-1 Proteins 0.000 claims description 54
• 102100032702 Protein jagged-1 Human genes 0.000 claims description 52
• 239000008194 pharmaceutical composition Substances 0.000 claims description 36
• 108700028369 Alleles Proteins 0.000 claims description 33
• 108020005067 RNA Splice Sites Proteins 0.000 claims description 28
• 230000002950 deficient Effects 0.000 claims description 15
• 230000002829 reductive effect Effects 0.000 claims description 10
• 230000007812 deficiency Effects 0.000 claims description 8
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• 239000002243 precursor Substances 0.000 claims description 8
• 108020004707 nucleic acids Proteins 0.000 claims description 7
• 102000039446 nucleic acids Human genes 0.000 claims description 7
• 230000000295 complement effect Effects 0.000 claims description 6
• 238000004519 manufacturing process Methods 0.000 claims description 6
• 201000011374 Alagille syndrome Diseases 0.000 claims description 5
• 238000010255 intramuscular injection Methods 0.000 claims description 5
• 239000007927 intramuscular injection Substances 0.000 claims description 5
• 239000007928 intraperitoneal injection Substances 0.000 claims description 5
• 238000010253 intravenous injection Methods 0.000 claims description 5
• 238000010254 subcutaneous injection Methods 0.000 claims description 5
• 239000007929 subcutaneous injection Substances 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
• 108091093037 Peptide nucleic acid Proteins 0.000 claims description 4
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• 239000007924 injection Substances 0.000 claims description 4
• 238000012986 modification Methods 0.000 claims description 4
• 230000004048 modification Effects 0.000 claims description 4
• 150000007523 nucleic acids Chemical class 0.000 claims description 4
• 238000007913 intrathecal administration Methods 0.000 claims description 2
• 238000007914 intraventricular administration Methods 0.000 claims description 2
• ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims 1
• 239000003814 drug Substances 0.000 claims 1
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
• 239000000203 mixture Substances 0.000 description 36
• 108091032973 (ribonucleotides)n+m Proteins 0.000 description 35
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 15
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• 108091027974 Mature messenger RNA Proteins 0.000 description 4
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