JP6672270B2 - トランスサイレチン(ttr)媒介アミロイドーシスの治療方法 - Google Patents

トランスサイレチン(ttr)媒介アミロイドーシスの治療方法 Download PDF

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JP6672270B2
JP6672270B2 JP2017511912A JP2017511912A JP6672270B2 JP 6672270 B2 JP6672270 B2 JP 6672270B2 JP 2017511912 A JP2017511912 A JP 2017511912A JP 2017511912 A JP2017511912 A JP 2017511912A JP 6672270 B2 JP6672270 B2 JP 6672270B2
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ttr
pharmaceutical composition
dose
composition according
administered
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JP2017530953A (ja
JP2017530953A5 (cg-RX-API-DMAC7.html
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ブライアン・ベッテンコート
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Alnylam Pharmaceuticals Inc
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/566Immunoassay; Biospecific binding assay; Materials therefor using specific carrier or receptor proteins as ligand binding reagents where possible specific carrier or receptor proteins are classified with their target compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/42Oxazoles
    • A61K31/423Oxazoles condensed with carbocyclic rings
    • AHUMAN NECESSITIES
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    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
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    • A61P25/00Drugs for disorders of the nervous system
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    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D263/00Heterocyclic compounds containing 1,3-oxazole or hydrogenated 1,3-oxazole rings
    • C07D263/52Heterocyclic compounds containing 1,3-oxazole or hydrogenated 1,3-oxazole rings condensed with carbocyclic rings or ring systems
    • C07D263/54Benzoxazoles; Hydrogenated benzoxazoles
    • C07D263/56Benzoxazoles; Hydrogenated benzoxazoles with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached in position 2
    • C07D263/57Aryl or substituted aryl radicals
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
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    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/566Immunoassay; Biospecific binding assay; Materials therefor using specific carrier or receptor proteins as ligand binding reagents where possible specific carrier or receptor proteins are classified with their target compounds
    • G01N33/567Immunoassay; Biospecific binding assay; Materials therefor using specific carrier or receptor proteins as ligand binding reagents where possible specific carrier or receptor proteins are classified with their target compounds utilising isolate of tissue or organ as binding agent
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896Neurological disorders, e.g. Alzheimer's disease
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K1/00General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]

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  • Bioinformatics & Cheminformatics (AREA)
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  • Food Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
JP2017511912A 2014-08-29 2015-08-27 トランスサイレチン(ttr)媒介アミロイドーシスの治療方法 Active JP6672270B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462044100P 2014-08-29 2014-08-29
US62/044,100 2014-08-29
US201562150596P 2015-04-21 2015-04-21
US62/150,596 2015-04-21
PCT/US2015/047185 WO2016033326A2 (en) 2014-08-29 2015-08-27 Methods of treating transthyretin (ttr) mediated amyloidosis

Related Child Applications (1)

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JP2020037018A Division JP2020105205A (ja) 2014-08-29 2020-03-04 トランスサイレチン(ttr)媒介アミロイドーシスの治療方法

Publications (3)

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JP2017530953A JP2017530953A (ja) 2017-10-19
JP2017530953A5 JP2017530953A5 (cg-RX-API-DMAC7.html) 2018-10-04
JP6672270B2 true JP6672270B2 (ja) 2020-03-25

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JP2017511912A Active JP6672270B2 (ja) 2014-08-29 2015-08-27 トランスサイレチン(ttr)媒介アミロイドーシスの治療方法
JP2020037018A Pending JP2020105205A (ja) 2014-08-29 2020-03-04 トランスサイレチン(ttr)媒介アミロイドーシスの治療方法
JP2022193040A Pending JP2023029942A (ja) 2014-08-29 2022-12-01 トランスサイレチン(ttr)媒介アミロイドーシスの治療方法

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JP2020037018A Pending JP2020105205A (ja) 2014-08-29 2020-03-04 トランスサイレチン(ttr)媒介アミロイドーシスの治療方法
JP2022193040A Pending JP2023029942A (ja) 2014-08-29 2022-12-01 トランスサイレチン(ttr)媒介アミロイドーシスの治療方法

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US (4) US10060921B2 (cg-RX-API-DMAC7.html)
EP (2) EP3185957B1 (cg-RX-API-DMAC7.html)
JP (3) JP6672270B2 (cg-RX-API-DMAC7.html)
KR (3) KR102631505B1 (cg-RX-API-DMAC7.html)
CN (2) CN107106874B (cg-RX-API-DMAC7.html)
DK (1) DK3185957T3 (cg-RX-API-DMAC7.html)
ES (1) ES2928500T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20221055T1 (cg-RX-API-DMAC7.html)
HU (1) HUE059857T2 (cg-RX-API-DMAC7.html)
LT (1) LT3185957T (cg-RX-API-DMAC7.html)
PL (1) PL3185957T3 (cg-RX-API-DMAC7.html)
PT (1) PT3185957T (cg-RX-API-DMAC7.html)
SI (1) SI3185957T1 (cg-RX-API-DMAC7.html)
TW (3) TWI702045B (cg-RX-API-DMAC7.html)
WO (1) WO2016033326A2 (cg-RX-API-DMAC7.html)

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EP3684931A1 (en) * 2017-09-19 2020-07-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
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