PT3043827T - Moduladores do fator b do complemento - Google Patents
Moduladores do fator b do complementoInfo
- Publication number
- PT3043827T PT3043827T PT14844168T PT14844168T PT3043827T PT 3043827 T PT3043827 T PT 3043827T PT 14844168 T PT14844168 T PT 14844168T PT 14844168 T PT14844168 T PT 14844168T PT 3043827 T PT3043827 T PT 3043827T
- Authority
- PT
- Portugal
- 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
-
- 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)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (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 (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361877624P | 2013-09-13 | 2013-09-13 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT3043827T true PT3043827T (pt) | 2019-09-26 |
Family
ID=52666522
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT14844168T PT3043827T (pt) | 2013-09-13 | 2014-09-12 | Moduladores do fator b do complemento |
Country Status (32)
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI2161038T1 (sl) | 2006-01-26 | 2014-05-30 | Isis Pharmaceuticals, Inc. | Sestavki in njihove uporabe, usmerjeni proti huntingtinu |
| CA2667055C (en) | 2006-10-18 | 2017-05-09 | Isis Pharmaceuticals, Inc. | Antisense compounds |
| RU2562861C2 (ru) | 2009-09-11 | 2015-09-10 | Айсис Фармасьютикалс, Инк. | Модуляция экспрессии гентингтина |
| EP3067421B1 (en) | 2011-02-08 | 2018-10-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| CA2921162A1 (en) | 2013-05-01 | 2014-11-06 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| JP6666250B2 (ja) * | 2013-09-13 | 2020-03-13 | 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | 補体ｂ因子の調節薬 |
| CA2931090A1 (en) * | 2013-12-12 | 2015-06-18 | Alnylam Pharmaceuticals, Inc. | Complement component irna compositions and methods of use thereof |
| US10280423B2 (en) * | 2014-05-01 | 2019-05-07 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating complement factor B expression |
| 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 |
| CN109414441A (zh) | 2016-06-27 | 2019-03-01 | 艾其林医药公司 | 治疗医学障碍的喹唑啉和吲哚化合物 |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| 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 |
| US11866701B2 (en) | 2017-11-01 | 2024-01-09 | 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 |
| BR112022007540A2 (pt) | 2019-10-22 | 2022-07-12 | Alnylam Pharmaceuticals Inc | Composições de irna de componente complementar c3 e métodos de uso das mesmas |
| EP4142738A1 (en) * | 2020-04-30 | 2023-03-08 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| US20230331710A1 (en) | 2020-08-07 | 2023-10-19 | Shanghai Meiyue Biotech Development Co. Ltd | Heterocyclic compound, preparation method and use thereof |
| US20230286947A1 (en) | 2020-08-07 | 2023-09-14 | Shanghai Meiyue Biotech Development Co. Ltd | Complement factor b inhibitor, and pharmaceutical composition, preparation method and use thereof |
| JP2024525868A (ja) | 2021-07-17 | 2024-07-12 | サーナオミクス インコーポレイテッド | 産物及び組成物 |
| CA3230589A1 (en) | 2021-09-02 | 2023-03-09 | Sibylle DAMES | 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 |
| EP4437104A4 (en) | 2021-11-24 | 2026-01-21 | Adarx Pharmaceuticals Inc | COMPLEMENT FACTOR B MODULATION COMPOSITIONS AND THEIR METHODS OF USE |
| 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 |
| JP2025530222A (ja) | 2022-09-10 | 2025-09-11 | 江▲蘇▼豪森▲薬▼▲業▼集▲団▼有限公司 | ２－置換ピペリジン誘導体、その製造方法及び医薬的用途 |
| AU2023372971A1 (en) * | 2022-11-02 | 2025-05-15 | Ionis Pharmaceuticals, Inc. | Methods for modulating complement factor b expression |
| WO2024222852A1 (zh) | 2023-04-27 | 2024-10-31 | 上海翰森生物医药科技有限公司 | 一种哌啶基吲哚化合物的盐及其制备方法 |
| 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 |
| EP4737577A1 (en) * | 2023-06-28 | 2026-05-06 | Biocytogen Pharmaceuticals (Beijing) Co., Ltd. | Cfb gene modified non-human animal |
| EP4735600A1 (en) * | 2023-06-28 | 2026-05-06 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of complement factor b (cfb) |
| 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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| JP6666250B2 (ja) | 2013-09-13 | 2020-03-13 | 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | 補体ｂ因子の調節薬 |
| US10280423B2 (en) * | 2014-05-01 | 2019-05-07 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating complement factor B expression |
-
2014
- 2014-09-12 JP JP2016542824A patent/JP6666250B2/ja active Active
- 2014-09-12 MX MX2016003263A patent/MX371518B/es active IP Right Grant
- 2014-09-12 EA EA201690582A patent/EA035433B1/ru unknown
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