JP7197463B2 - Ｓｍｎ２の調節のための化合物及び方法 - Google Patents

Ｓｍｎ２の調節のための化合物及び方法 Download PDF

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Publication number
JP7197463B2
JP7197463B2 JP2019500807A JP2019500807A JP7197463B2 JP 7197463 B2 JP7197463 B2 JP 7197463B2 JP 2019500807 A JP2019500807 A JP 2019500807A JP 2019500807 A JP2019500807 A JP 2019500807A JP 7197463 B2 JP7197463 B2 JP 7197463B2
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modified
oligomeric compound
certain embodiments
nucleosides
modified oligonucleotide
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Japanese (ja)
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JP2019525918A (ja
JP2019525918A5 (enExample
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リゴ，フランク
プラカシュ，タジャ・ピー
セス，プニット・ピー
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Steroid Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2019500807A 2016-07-15 2017-07-17 Ｓｍｎ２の調節のための化合物及び方法 Active JP7197463B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022199992A JP2023027285A (ja) 2016-07-15 2022-12-15 Ｓｍｎ２の調節のための化合物及び方法
JP2025003382A JP2025063137A (ja) 2016-07-15 2025-01-09 Ｓｍｎ２の調節のための化合物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662363195P 2016-07-15 2016-07-15
US62/363,195 2016-07-15
PCT/US2017/042463 WO2018014041A2 (en) 2016-07-15 2017-07-17 Compounds and methods for modulation of smn2

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JP2019525918A JP2019525918A (ja) 2019-09-12
JP2019525918A5 JP2019525918A5 (enExample) 2020-08-27
JP7197463B2 true JP7197463B2 (ja) 2022-12-27

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JP2019500807A Active JP7197463B2 (ja) 2016-07-15 2017-07-17 Ｓｍｎ２の調節のための化合物及び方法
JP2022199992A Pending JP2023027285A (ja) 2016-07-15 2022-12-15 Ｓｍｎ２の調節のための化合物及び方法
JP2025003382A Pending JP2025063137A (ja) 2016-07-15 2025-01-09 Ｓｍｎ２の調節のための化合物及び方法

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JP2025003382A Pending JP2025063137A (ja) 2016-07-15 2025-01-09 Ｓｍｎ２の調節のための化合物及び方法

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US (3) US20190323006A1 (enExample)
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KR (1) KR102556825B1 (enExample)
CN (1) CN109844115B (enExample)
AU (2) AU2017297622A1 (enExample)
BR (1) BR112019000356A2 (enExample)
CA (1) CA3030864A1 (enExample)
CL (2) CL2019000115A1 (enExample)
CO (1) CO2019001236A2 (enExample)
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FI (1) FI3484524T3 (enExample)
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HU (1) HUE060831T2 (enExample)
IL (2) IL264216B2 (enExample)
LT (1) LT3484524T (enExample)
MX (1) MX2019000619A (enExample)
NZ (1) NZ750713A (enExample)
PE (1) PE20190513A1 (enExample)
PL (1) PL3484524T3 (enExample)
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SM (1) SMT202200489T1 (enExample)
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EP2983676B1 (en) * 2013-04-12 2019-03-20 The Curators of the University of Missouri Smn2 element 1 antisense compositions and methods and uses thereof
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
WO2017035366A1 (en) 2015-08-26 2017-03-02 Incyte Corporation Pyrrolopyrimidine derivatives as tam inhibitors
IL261957B (en) 2016-03-28 2022-07-01 Incyte Corp Pyrrolotriazine compounds as TAM inhibitors
JOP20200081A1 (ar) 2017-09-27 2020-04-30 Incyte Corp أملاح من مشتقات بيرولوتريازين مفيدة بوصفها مثبطات لـ tam
DK3813800T3 (da) 2018-06-29 2025-06-02 Incyte Corp Formuleringer af en axl/mer-hæmmer
EP4335503A3 (en) * 2019-07-19 2024-06-05 Biogen MA Inc. Methods of treating or preventing spinal muscular atrophy
EP4013387A4 (en) * 2019-08-15 2023-09-27 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
JP7737667B2 (ja) * 2019-09-18 2025-09-11 国立大学法人東京科学大学 核酸複合体
TW202517787A (zh) 2020-02-28 2025-05-01 美商Ionis製藥公司 用於調節smn2之化合物及方法
US20230043648A1 (en) * 2020-02-28 2023-02-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn1a expression
WO2021178779A1 (en) 2020-03-06 2021-09-10 Incyte Corporation Combination therapy comprising axl/mer and pd-1/pd-l1 inhibitors
EP4326878A4 (en) * 2021-04-22 2025-07-02 Molecular Axiom Llc COMPOSITIONS AND METHODS FOR MODULATING SOS GENE EXPRESSIONS
FR3128873A1 (fr) 2021-11-10 2023-05-12 Biophytis Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale

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WO2015161170A2 (en) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject

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