ES2745758T3 - Moduladores del factor B del complemento - Google Patents
Moduladores del factor B del complemento Download PDFInfo
- Publication number
- ES2745758T3 ES2745758T3 ES14844168T ES14844168T ES2745758T3 ES 2745758 T3 ES2745758 T3 ES 2745758T3 ES 14844168 T ES14844168 T ES 14844168T ES 14844168 T ES14844168 T ES 14844168T ES 2745758 T3 ES2745758 T3 ES 2745758T3
- Authority
- ES
- Spain
- Prior art keywords
- certain embodiments
- antisense
- cfb
- nucleosides
- compound
- Prior art date
- Legal status (The legal status 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 status listed.)
- Active
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- 0 CC1(NCC(*)(**2)C1C2(*)OC)OC Chemical compound CC1(NCC(*)(**2)C1C2(*)OC)OC 0.000 description 3
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
-
- 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
-
- 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
-
- 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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Virology (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)
- Peptides Or Proteins (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
- Epidemiology (AREA)
- Amplifiers (AREA)
- Optical Communication System (AREA)
Applications Claiming Priority (2)
| 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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2745758T3 true ES2745758T3 (es) | 2020-03-03 |
Family
ID=52666522
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES14844168T Active ES2745758T3 (es) | 2013-09-13 | 2014-09-12 | Moduladores del factor B del complemento |
Country Status (32)
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5425474B2 (ja) | 2006-01-26 | 2014-02-26 | アイシス ファーマシューティカルズ， インコーポレーテッド | ハンチンチン対する、組成物及びその使用 |
| DK2092065T4 (da) | 2006-10-18 | 2019-10-21 | Ionis Pharmaceuticals Inc | Antisense-forbindelser |
| NZ599032A (en) | 2009-09-11 | 2014-09-26 | Isis Pharmaceuticals Inc | Modulation of huntingtin expression |
| EP3467109A1 (en) | 2011-02-08 | 2019-04-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| PE20152002A1 (es) | 2013-05-01 | 2016-01-21 | Isis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion de ttr y vhb |
| CN112359042A (zh) * | 2013-09-13 | 2021-02-12 | 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 |
| RS59182B1 (sr) * | 2014-05-01 | 2019-10-31 | Ionis Pharmaceuticals Inc | Kompozicije i postupci za modulaciju ekspresije faktora b komplementa |
| TW201730341A (zh) * | 2016-02-05 | 2017-09-01 | Kyowa Hakko Kirin Co Ltd | 抑制補體b因子之表現之反義寡核苷酸 |
| EP3939591A1 (en) | 2016-06-27 | 2022-01-19 | Achillion Pharmaceuticals, Inc. | Quinazoline and indole compounds to treat medical disorders |
| WO2018102397A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| TW201827596A (zh) * | 2016-12-23 | 2018-08-01 | 日商協和醱酵麒麟有限公司 | 抑制補體b因子之表現之核酸 |
| 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 |
| SG11202002940QA (en) | 2017-11-01 | 2020-04-29 | 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 |
| AU2020369515A1 (en) | 2019-10-22 | 2022-04-21 | Alnylam Pharmaceuticals, Inc. | Complement component C3 iRNA compositions and methods of use thereof |
| BR112022021136A2 (pt) * | 2020-04-30 | 2022-11-29 | Alnylam Pharmaceuticals Inc | Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas |
| WO2022028527A1 (zh) | 2020-08-07 | 2022-02-10 | 上海美悦生物科技发展有限公司 | 补体因子b抑制剂及其药物组合物、制备方法和用途 |
| WO2022028507A1 (zh) | 2020-08-07 | 2022-02-10 | 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 |
| US20230135763A1 (en) * | 2021-07-17 | 2023-05-04 | Sirnaomics, Inc. | Products and compositions |
| AU2022336157A1 (en) | 2021-09-02 | 2024-03-14 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell |
| CA3234636A1 (en) * | 2021-10-29 | 2023-05-04 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| US20250075209A1 (en) * | 2021-12-27 | 2025-03-06 | Apellis Pharmaceuticals, Inc. | Rnas for complement inhibition |
| EP4471021A4 (en) | 2022-01-26 | 2025-04-30 | Shanghai Meiyue Biotech Development Co., Ltd. | Salt type and crystal form of complement factor b inhibitor, and preparation method therefor and application thereof |
| CA3266935A1 (en) | 2022-09-10 | 2024-03-14 | Jiangsu Hansoh Pharmaceutical Group Co., Ltd. | Piperidine derivatives substituted at position 2, their preparation methods and medicinal uses |
| JP2025537139A (ja) * | 2022-11-02 | 2025-11-14 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | 補体因子ｂの発現を調節するための方法 |
| EP4703356A1 (en) | 2023-04-27 | 2026-03-04 | Shanghai Hansoh Biomedical Co., Ltd. | Salt of piperidylindole compound and preparation method therefor |
| WO2024245424A1 (zh) * | 2023-06-02 | 2024-12-05 | 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 |
| AU2024307421A1 (en) * | 2023-06-28 | 2026-02-12 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of complement factor b (cfb) |
| 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 |
| WO2025122669A1 (en) * | 2023-12-05 | 2025-06-12 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a humanized complement factor b gene |
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| CN112359042A (zh) * | 2013-09-13 | 2021-02-12 | Ionis制药公司 | 补体因子b的调节剂 |
| RS59182B1 (sr) * | 2014-05-01 | 2019-10-31 | Ionis Pharmaceuticals Inc | Kompozicije i postupci za modulaciju ekspresije faktora b komplementa |
-
2014
- 2014-09-12 CN CN202011263979.3A patent/CN112359042A/zh active Pending
- 2014-09-12 EP EP14844168.6A patent/EP3043827B1/en active Active
- 2014-09-12 PL PL14844168T patent/PL3043827T3/pl unknown
- 2014-09-12 PE PE2018003111A patent/PE20190354A1/es unknown
- 2014-09-12 ES ES14844168T patent/ES2745758T3/es active Active
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