PL3043827T3 - Modulatory czynnika b dopełniacza - Google Patents
Modulatory czynnika b dopełniaczaInfo
- Publication number
- PL3043827T3 PL3043827T3 PL14844168T PL14844168T PL3043827T3 PL 3043827 T3 PL3043827 T3 PL 3043827T3 PL 14844168 T PL14844168 T PL 14844168T PL 14844168 T PL14844168 T PL 14844168T PL 3043827 T3 PL3043827 T3 PL 3043827T3
- Authority
- PL
- Poland
- 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
-
- 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)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (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)
- Optical Communication System (AREA)
- Amplifiers (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 |
|---|---|
| PL3043827T3 true PL3043827T3 (pl) | 2020-03-31 |
Family
ID=52666522
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL14844168T PL3043827T3 (pl) | 2013-09-13 | 2014-09-12 | Modulatory czynnika b dopełniacza |
Country Status (32)
| Country | Link |
|---|---|
| US (5) | US20160222389A1 (pl) |
| EP (2) | EP3043827B1 (pl) |
| JP (3) | JP6666250B2 (pl) |
| KR (1) | KR102381790B1 (pl) |
| CN (2) | CN112359042A (pl) |
| AU (2) | AU2014318580B2 (pl) |
| BR (1) | BR112016004671B1 (pl) |
| CA (1) | CA2921842A1 (pl) |
| CL (2) | CL2016000606A1 (pl) |
| CR (1) | CR20160170A (pl) |
| DK (1) | DK3043827T3 (pl) |
| DO (1) | DOP2016000063A (pl) |
| EA (1) | EA035433B1 (pl) |
| ES (1) | ES2745758T3 (pl) |
| HR (1) | HRP20191600T1 (pl) |
| HU (1) | HUE045109T2 (pl) |
| IL (1) | IL244095A0 (pl) |
| LT (1) | LT3043827T (pl) |
| MA (1) | MA38959A1 (pl) |
| MX (2) | MX371518B (pl) |
| MY (1) | MY181251A (pl) |
| PE (2) | PE20190354A1 (pl) |
| PH (1) | PH12016500443A1 (pl) |
| PL (1) | PL3043827T3 (pl) |
| PT (1) | PT3043827T (pl) |
| RS (1) | RS59252B1 (pl) |
| RU (1) | RU2662967C2 (pl) |
| SG (2) | SG10201806787VA (pl) |
| SI (1) | SI3043827T1 (pl) |
| UA (1) | UA119046C2 (pl) |
| WO (1) | WO2015038939A2 (pl) |
| ZA (1) | ZA201601236B (pl) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2667055C (en) | 2006-10-18 | 2017-05-09 | Isis Pharmaceuticals, Inc. | Antisense compounds |
| KR102279458B1 (ko) | 2009-09-11 | 2021-07-21 | 아이오니스 파마수티컬즈, 인코포레이티드 | 헌팅틴 발현의 조절 |
| EP3467109A1 (en) | 2011-02-08 | 2019-04-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| US9127276B2 (en) | 2013-05-01 | 2015-09-08 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| EP3043827B1 (en) | 2013-09-13 | 2019-07-03 | Ionis Pharmaceuticals, Inc. | Modulators of complement factor b |
| MX382900B (es) * | 2013-12-12 | 2025-03-13 | Alnylam Pharmaceuticals Inc | COMPOSICIONES DE ÁCIDO RIBONUCLEICO DE INTERFERENCIA (ARNi) CONTRA EL COMPONENTE DEL COMPLEMENTO Y MÉTODOS PARA SU USO. |
| PH12016502062B1 (en) * | 2014-05-01 | 2023-01-27 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
| 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 |
| KR20190036520A (ko) | 2016-06-27 | 2019-04-04 | 아칠리온 파르마세우티칼스 인코포레이티드 | 의학적 장애를 치료하기 위한 퀴나졸린 및 인돌 화합물 |
| EP3548005A4 (en) | 2016-11-29 | 2020-06-17 | 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 |
| AU2018360697A1 (en) | 2017-11-01 | 2020-05-14 | 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 |
| JP7676377B2 (ja) | 2019-10-22 | 2025-05-14 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 補体成分Ｃ３ ｉＲＮＡ組成物およびその使用方法 |
| MX2022013606A (es) * | 2020-04-30 | 2023-01-16 | Alnylam Pharmaceuticals Inc | Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas. |
| US20230331710A1 (en) | 2020-08-07 | 2023-10-19 | Shanghai Meiyue Biotech Development Co. Ltd | Heterocyclic compound, preparation method and use thereof |
| CN118108700A (zh) | 2020-08-07 | 2024-05-31 | 诺华股份有限公司 | 补体因子b抑制剂及其药物组合物、制备方法和用途 |
| AU2022325715A1 (en) * | 2021-07-17 | 2024-02-01 | Sirnaomics, Inc. | Products and compositions |
| IL311146A (en) | 2021-09-02 | 2024-04-01 | 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 |
| US20250075209A1 (en) * | 2021-12-27 | 2025-03-06 | Apellis Pharmaceuticals, Inc. | Rnas for complement inhibition |
| CN116496249A (zh) | 2022-01-26 | 2023-07-28 | 上海美悦生物科技发展有限公司 | 补体因子b抑制剂的盐型、晶型及其制备方法和应用 |
| WO2024051849A1 (en) | 2022-09-10 | 2024-03-14 | Jiangsu Hansoh Pharmaceutical Group Co., Ltd. | 2-substituted piperidine derivatives, preparation methods and medicinal uses thereof |
| CA3272023A1 (en) * | 2022-11-02 | 2024-05-10 | Ionis Pharmaceuticals, Inc. | METHODS FOR MODULATING THE EXPRESSION OF COMPLEMENT FACTOR B |
| 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 |
| 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 |
| WO2025002299A1 (en) * | 2023-06-28 | 2025-01-02 | 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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