JP2023514570A - 補体阻害のためのｒｎａ - Google Patents

補体阻害のためのｒｎａ Download PDF

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Publication number
JP2023514570A
JP2023514570A JP2022548899A JP2022548899A JP2023514570A JP 2023514570 A JP2023514570 A JP 2023514570A JP 2022548899 A JP2022548899 A JP 2022548899A JP 2022548899 A JP2022548899 A JP 2022548899A JP 2023514570 A JP2023514570 A JP 2023514570A
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Prior art keywords
sirna
subject
complement
nucleotides
antisense strand
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JP2022548899A
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Japanese (ja)
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JP2023514570A5 (https=
Inventor
マルクス ホスバッハ，
カトリン フルチュ，
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Apellis Pharmaceuticals Inc
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Apellis Pharmaceuticals Inc
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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2022548899A 2020-02-14 2021-02-13 補体阻害のためのｒｎａ Pending JP2023514570A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202062977012P 2020-02-14 2020-02-14
US62/977,012 2020-02-14
US202062980100P 2020-02-21 2020-02-21
US62/980,100 2020-02-21
US202063062321P 2020-08-06 2020-08-06
US63/062,321 2020-08-06
PCT/US2021/018071 WO2021163654A1 (en) 2020-02-14 2021-02-13 Rnas for complement inhibition

Publications (2)

Publication Number Publication Date
JP2023514570A true JP2023514570A (ja) 2023-04-06
JP2023514570A5 JP2023514570A5 (https=) 2024-02-05

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Family Applications (1)

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JP2022548899A Pending JP2023514570A (ja) 2020-02-14 2021-02-13 補体阻害のためのｒｎａ

Country Status (10)

Country Link
US (1) US20230095695A1 (https=)
EP (1) EP4103716A4 (https=)
JP (1) JP2023514570A (https=)
KR (1) KR20220155301A (https=)
CN (1) CN115335524A (https=)
AU (1) AU2021221035A1 (https=)
CA (1) CA3170158A1 (https=)
IL (1) IL295517A (https=)
MX (1) MX2022009870A (https=)
WO (1) WO2021163654A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
WO2022226127A1 (en) * 2021-04-20 2022-10-27 Alexion Pharmaceuticals, Inc. Compositions and methods for inhibiting complement component 3 expression
EP4398926A4 (en) * 2021-09-09 2025-07-02 Apellis Pharmaceuticals Inc TREATMENT OF GEOGRAPHIC ATROPHY
WO2024092105A2 (en) 2022-10-27 2024-05-02 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement component c3 (c3), pharmaceutical compositions thereof, and methods of use
CN121488036A (zh) * 2023-07-21 2026-02-06 上海舶望制药有限公司 用于抑制补体成分3(c3)表达的组合物和方法
WO2025026124A1 (zh) * 2023-08-03 2025-02-06 苏州炫景生物科技有限公司 C4b基因抑制剂组合物及其应用
WO2025179182A2 (en) * 2024-02-22 2025-08-28 Apellis Pharmaceuticals, Inc. C3 targeted inhibitory rna

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009518008A (ja) * 2005-11-30 2009-05-07 イントラディグム コーポレイション 遺伝子発現をノックダウンするため、そして固形臓器および細胞の移植を改善するためにｓｉＲＮＡを使用する組成物および方法
WO2019089922A1 (en) * 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007124452A2 (en) * 2006-04-20 2007-11-01 The Regents Of The University Of California Methods for expressing multiple sirna and shrna from a single vector
IL288931B2 (en) * 2013-03-14 2025-05-01 Alnylam Pharmaceuticals Inc Compositions comprising a complementary portion of C5 IRNA and methods of using them
RU2019113301A (ru) * 2016-10-17 2020-11-17 Апеллис Фармасьютикалс, Инк. Комбинированная терапия для ингибирования с3

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009518008A (ja) * 2005-11-30 2009-05-07 イントラディグム コーポレイション 遺伝子発現をノックダウンするため、そして固形臓器および細胞の移植を改善するためにｓｉＲＮＡを使用する組成物および方法
WO2019089922A1 (en) * 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof

Also Published As

Publication number Publication date
EP4103716A1 (en) 2022-12-21
EP4103716A4 (en) 2025-07-09
CN115335524A (zh) 2022-11-11
CA3170158A1 (en) 2021-08-19
IL295517A (en) 2022-10-01
WO2021163654A1 (en) 2021-08-19
KR20220155301A (ko) 2022-11-22
AU2021221035A1 (en) 2022-09-08
WO2021163654A8 (en) 2021-10-21
MX2022009870A (es) 2022-11-07
US20230095695A1 (en) 2023-03-30

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