SI3043827T1 - Modulatorji komplementnega faktorja B - Google Patents
Modulatorji komplementnega faktorja BInfo
- Publication number
- SI3043827T1 SI3043827T1 SI201431272T SI201431272T SI3043827T1 SI 3043827 T1 SI3043827 T1 SI 3043827T1 SI 201431272 T SI201431272 T SI 201431272T SI 201431272 T SI201431272 T SI 201431272T SI 3043827 T1 SI3043827 T1 SI 3043827T1
- Authority
- SI
- Slovenia
- Prior art keywords
- modulators
- complement factor
- complement
- factor
- Prior art date
Links
- 102000003712 Complement factor B Human genes 0.000 title 1
- 108090000056 Complement factor B Proteins 0.000 title 1
Classifications
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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
- 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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- 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
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- 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
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- 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
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Urology & Nephrology (AREA)
- Ophthalmology & Optometry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Peptides Or Proteins (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
- Epidemiology (AREA)
- Amplifiers (AREA)
- Optical Communication System (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361877624P | 2013-09-13 | 2013-09-13 | |
| PCT/US2014/055458 WO2015038939A2 (en) | 2013-09-13 | 2014-09-12 | Modulators of complement factor b |
| EP14844168.6A EP3043827B1 (en) | 2013-09-13 | 2014-09-12 | Modulators of complement factor b |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI3043827T1 true SI3043827T1 (sl) | 2019-08-30 |
Family
ID=52666522
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201431272T SI3043827T1 (sl) | 2013-09-13 | 2014-09-12 | Modulatorji komplementnega faktorja B |
Country Status (32)
Families Citing this family (31)
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| ES2620472T5 (es) | 2006-10-18 | 2020-07-09 | Ionis Pharmaceuticals Inc | Compuestos antisentido |
| RU2751847C9 (ru) | 2009-09-11 | 2021-08-20 | Ионис Фармасьютикалз, Инк. | Модуляция экспрессии гентингтина |
| EP3467109A1 (en) | 2011-02-08 | 2019-04-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| IL296543B2 (en) | 2013-05-01 | 2025-02-01 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating HBV and TTR expression |
| CN105744959B (zh) * | 2013-09-13 | 2020-12-01 | Ionis制药公司 | 补体因子b的调节剂 |
| IL314045A (en) * | 2013-12-12 | 2024-09-01 | Alnylam Pharmaceuticals Inc | Complement component irna compositions and methods of use thereof |
| UA121656C2 (uk) * | 2014-05-01 | 2020-07-10 | Айоніс Фармасьютикалз, Інк. | Олігомерна сполука та спосіб лікування захворювання, пов'язаного з порушенням регуляції альтернативного шляху комплементу |
| WO2017135397A1 (ja) * | 2016-02-05 | 2017-08-10 | 協和発酵キリン株式会社 | 補体ｂ因子の発現を抑制するアンチセンスオリゴヌクレオチド |
| MX2018016046A (es) | 2016-06-27 | 2019-05-02 | Achillion Pharmaceuticals Inc | Quinazolina y compuestos indol para tratar trastornos medicos. |
| CN110177544A (zh) | 2016-11-29 | 2019-08-27 | 普尔泰克健康有限公司 | 用于递送治疗剂的外泌体 |
| WO2018117253A1 (ja) * | 2016-12-23 | 2018-06-28 | 協和発酵キリン株式会社 | 補体ｂ因子の発現を抑制する核酸 |
| TW201909925A (zh) * | 2017-08-02 | 2019-03-16 | 日商協和醱酵麒麟有限公司 | 核酸複合體 |
| WO2019089922A1 (en) | 2017-11-01 | 2019-05-09 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof |
| WO2020109343A1 (en) | 2018-11-29 | 2020-06-04 | F. Hoffmann-La Roche Ag | Combination therapy for treatment of macular degeneration |
| MX2022004726A (es) | 2019-10-22 | 2022-05-13 | Alnylam Pharmaceuticals Inc | Composiciones de acido ribonucleico de interferencia (arni) de componente c3 de complemento y metodos de uso de las mismas. |
| IL297680A (en) * | 2020-04-30 | 2022-12-01 | Alnylam Pharmaceuticals Inc | Complement factor b (cfb) irna compositions and methods of use thereof |
| EP4194451A4 (en) | 2020-08-07 | 2024-07-24 | Shanghai Meiyue Biotech Development Co., Ltd. | SUPPLEMENT FACTOR B INHIBITOR AND PHARMACEUTICAL COMPOSITION THEREOF, CORRESPONDING PREPARATION METHOD AND ASSOCIATED USE |
| CN114057692B (zh) | 2020-08-07 | 2023-07-21 | 上海美悦生物科技发展有限公司 | 一种杂环类化合物、其制备方法及用途 |
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| MX2024002767A (es) | 2021-09-02 | 2024-08-20 | Silence Therapeutics Gmbh | Ácidos nucleicos para inhibir la expresión del factor b del complemento (cfb) en una célula. |
| CA3234636A1 (en) * | 2021-10-29 | 2023-05-04 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| WO2023129496A2 (en) * | 2021-12-27 | 2023-07-06 | Apellis Pharmaceuticals, Inc. | Rnas for complement inhibition |
| CA3242815A1 (en) | 2022-01-26 | 2023-08-03 | Shanghai Meiyue Biotech Development Co., Ltd. | Salt type and crystal form of complement factor b inhibitor, and preparation method therefor and application thereof |
| WO2024051849A1 (en) | 2022-09-10 | 2024-03-14 | Jiangsu Hansoh Pharmaceutical Group Co., Ltd. | 2-substituted piperidine derivatives, preparation methods and medicinal uses thereof |
| AU2023372971A1 (en) * | 2022-11-02 | 2025-05-15 | Ionis Pharmaceuticals, Inc. | Methods for modulating complement factor b expression |
| AU2024261191A1 (en) | 2023-04-27 | 2025-11-06 | Jiangsu Hansoh Pharmaceutical Group Co., Ltd. | Salt of piperidylindole compound and preparation method therefor |
| TW202513799A (zh) * | 2023-06-02 | 2025-04-01 | 大陸商維亞臻生物技術（蘇州）有限公司 | 用於免疫類疾病的雙鏈核苷酸化合物及其用途 |
| WO2025002299A1 (en) * | 2023-06-28 | 2025-01-02 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of complement factor b (cfb) |
| WO2025002401A1 (zh) * | 2023-06-28 | 2025-01-02 | 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 |
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| WO2012142458A1 (en) * | 2011-04-13 | 2012-10-18 | Isis Pharmaceuticals, Inc. | Antisense modulation of ptp1b expression |
| CN105744959B (zh) * | 2013-09-13 | 2020-12-01 | Ionis制药公司 | 补体因子b的调节剂 |
| UA121656C2 (uk) * | 2014-05-01 | 2020-07-10 | Айоніс Фармасьютикалз, Інк. | Олігомерна сполука та спосіб лікування захворювання, пов'язаного з порушенням регуляції альтернативного шляху комплементу |
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2014
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