HUE046272T2 - Készítmények és eljárások a komplement B-faktor expressziójának modulálására - Google Patents

Készítmények és eljárások a komplement B-faktor expressziójának modulálására

Info

Publication number
HUE046272T2
HUE046272T2 HUE15786214A HUE15786214A HUE046272T2 HU E046272 T2 HUE046272 T2 HU E046272T2 HU E15786214 A HUE15786214 A HU E15786214A HU E15786214 A HUE15786214 A HU E15786214A HU E046272 T2 HUE046272 T2 HU E046272T2
Authority
HU
Hungary
Prior art keywords
modulating
expression
compositions
methods
complement factor
Prior art date
Application number
HUE15786214A
Other languages
English (en)
Hungarian (hu)
Inventor
Thazha Prakash
Punit Seth
Eric Swayze
Tamar Grossman
Michael Mccaleb
Andrew Watt
Susan Freier
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of HUE046272T2 publication Critical patent/HUE046272T2/hu

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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K47/555—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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
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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
    • C12N15/1137—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 against enzymes
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21047—Alternative-complement-pathway C3/C5 convertase (3.4.21.47), i.e. properdin factor B
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • 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
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • 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

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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Urology & Nephrology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
HUE15786214A 2014-05-01 2015-05-01 Készítmények és eljárások a komplement B-faktor expressziójának modulálására HUE046272T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461987471P 2014-05-01 2014-05-01
US201462076273P 2014-11-06 2014-11-06

Publications (1)

Publication Number Publication Date
HUE046272T2 true HUE046272T2 (hu) 2020-02-28

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HUE15786214A HUE046272T2 (hu) 2014-05-01 2015-05-01 Készítmények és eljárások a komplement B-faktor expressziójának modulálására

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US (5) US10280423B2 (enExample)
EP (3) EP4219718A3 (enExample)
JP (2) JP6637442B2 (enExample)
KR (1) KR102369736B1 (enExample)
CN (2) CN106232804B (enExample)
AU (1) AU2015252858C1 (enExample)
BR (1) BR112016022593B1 (enExample)
CA (1) CA2943894A1 (enExample)
CL (2) CL2016002764A1 (enExample)
CR (2) CR20160554A (enExample)
DK (1) DK3137596T3 (enExample)
DO (1) DOP2016000291A (enExample)
EA (1) EA036496B1 (enExample)
ES (2) ES2745825T3 (enExample)
HR (1) HRP20191664T1 (enExample)
HU (1) HUE046272T2 (enExample)
IL (2) IL247883B (enExample)
LT (1) LT3137596T (enExample)
MX (1) MX380866B (enExample)
PE (2) PE20170010A1 (enExample)
PH (1) PH12016502062B1 (enExample)
PL (2) PL3137596T3 (enExample)
PT (1) PT3137596T (enExample)
RS (1) RS59182B1 (enExample)
RU (1) RU2701645C2 (enExample)
SG (2) SG11201608502TA (enExample)
SI (1) SI3137596T1 (enExample)
UA (1) UA121656C2 (enExample)
WO (1) WO2015168635A2 (enExample)

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DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
US20160222389A1 (en) * 2013-09-13 2016-08-04 Ionis Pharmaceuticals, Inc. Modulators of complement factor b
KR20200102553A (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
DK3137596T3 (da) 2014-05-01 2019-09-02 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af komplement faktor b-ekspression
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EP3990465A1 (en) * 2019-06-25 2022-05-04 Amgen Inc. Purification methods for carbohydrate-linked oligonucleotides
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MX2022006514A (es) 2019-12-26 2022-07-11 Eisai R&D Man Co Ltd Composicion farmaceutica que contiene un acido ribonucleico de doble hebra que inhibe la expresion de c5 de complemento.
WO2021222549A1 (en) * 2020-04-30 2021-11-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2023018523A2 (en) * 2021-07-17 2023-02-16 Sirnaomics, Inc. Products and compositions
WO2023031359A1 (en) 2021-09-02 2023-03-09 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
EP4423273A1 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
IL320604A (en) * 2022-11-02 2025-07-01 Ionis Pharmaceuticals Inc Methods for modulating complement factor B expression
CN120569477A (zh) * 2022-12-19 2025-08-29 美国圣因生物股份有限公司 靶向cfb的小干扰rna及其用途
KR20250159727A (ko) * 2023-03-21 2025-11-11 애로우헤드 파마슈티컬스 인코포레이티드 보체 인자 B (CFB)의 발현을 억제하기 위한 RNAi 작용제, 그의 제약 조성물 및 사용 방법
CN121311588A (zh) * 2023-06-02 2026-01-09 维亚臻生物技术（苏州）有限公司 一种用于免疫类疾病的双链核苷酸化合物及其应用
CN118620964A (zh) * 2023-06-28 2024-09-10 百奥赛图(北京)医药科技股份有限公司 一种cfb基因修饰的非人动物
WO2025026228A1 (zh) * 2023-07-28 2025-02-06 苏州炫景生物科技有限公司 Cfb抑制剂组合物及其应用
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