PE20190354A1 - Moduladores del factor del complemento b - Google Patents
Moduladores del factor del complemento bInfo
- Publication number
- PE20190354A1 PE20190354A1 PE2018003111A PE2018003111A PE20190354A1 PE 20190354 A1 PE20190354 A1 PE 20190354A1 PE 2018003111 A PE2018003111 A PE 2018003111A PE 2018003111 A PE2018003111 A PE 2018003111A PE 20190354 A1 PE20190354 A1 PE 20190354A1
- Authority
- PE
- Peru
- Prior art keywords
- wing segment
- segment
- linked
- complement
- nucleobase sequence
- Prior art date
Links
- 230000000295 complement effect Effects 0.000 title 1
- 102000003712 Complement factor B Human genes 0.000 abstract 2
- 108090000056 Complement factor B Proteins 0.000 abstract 2
- 108091034117 Oligonucleotide Proteins 0.000 abstract 2
- 239000002777 nucleoside Substances 0.000 abstract 2
- 125000003835 nucleoside group Chemical group 0.000 abstract 2
- 125000002619 bicyclic group Chemical group 0.000 abstract 1
- 230000024203 complement activation Effects 0.000 abstract 1
- 150000001875 compounds Chemical class 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000008482 dysregulation Effects 0.000 abstract 1
- 239000003112 inhibitor Substances 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 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
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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
- C12N2310/32—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
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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
- C12N2310/33—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)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (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)
- 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)
Abstract
Se refiere a un compuesto que comprende un oligonucleotido modificado que consiste de 16 a 30 nucleotidos enlazados y que tiene una secuencia de nucleobases que comprende la secuencia de nucleobases de cualquiera de las SEQ ID NO: 6 A 197; 199 a 227, 229 a 236, 238 a 439, entre otros; en donde el oligonucleotido modificado comprende: (a) un segmento hueco que consiste en desoxinucleosidos enlazados; (b) un segmento de ala 5´ que consiste en nucleosidos enlazados; y (c) un segmento de ala 3´ que consiste en nucleosidos enlazados; en donde el segmento de hueco se encuentra ubicado inmediatamente adyacente a y entre el segmento de ala 5´ y el segmento de ala 3´, y donde cada segmento ala comprende un azucar biciclico modificado, la cual se selecciona del grupo que consiste en: 4´-(CH2)-O-2´ (LNA); 4´- (CH2)2-O-2´ (ENA); y 4´- CH(CH3)-O-2´ (cEt), siendo la preferida 2´-O-metoxietilo. Dicha composicion es util para prevenir, mejorar o tratar una enfermedad asociada con la desregulacion de la via alternativa de complemento mediante la administracion de un inhibidor especifico del factor del complemento B (CFB) a un sujeto.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361877624P | 2013-09-13 | 2013-09-13 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20190354A1 true PE20190354A1 (es) | 2019-03-07 |
Family
ID=52666522
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2018003111A PE20190354A1 (es) | 2013-09-13 | 2014-09-12 | Moduladores del factor del complemento b |
| PE2016000376A PE20160500A1 (es) | 2013-09-13 | 2014-09-12 | Moduladores del factor del complemento b |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2016000376A PE20160500A1 (es) | 2013-09-13 | 2014-09-12 | Moduladores del factor del complemento b |
Country Status (33)
| Country | Link |
|---|---|
| US (5) | US20160222389A1 (es) |
| EP (2) | EP3603677A1 (es) |
| JP (3) | JP6666250B2 (es) |
| KR (1) | KR102381790B1 (es) |
| CN (2) | CN105744959B (es) |
| AU (2) | AU2014318580B2 (es) |
| BR (1) | BR112016004671B1 (es) |
| CA (1) | CA2921842A1 (es) |
| CL (2) | CL2016000606A1 (es) |
| CR (1) | CR20160170A (es) |
| DK (1) | DK3043827T3 (es) |
| DO (1) | DOP2016000063A (es) |
| EA (1) | EA035433B1 (es) |
| ES (1) | ES2745758T3 (es) |
| HK (1) | HK1225638A1 (es) |
| HR (1) | HRP20191600T1 (es) |
| HU (1) | HUE045109T2 (es) |
| IL (1) | IL244095A0 (es) |
| LT (1) | LT3043827T (es) |
| MA (1) | MA38959A1 (es) |
| MX (2) | MX371518B (es) |
| MY (1) | MY181251A (es) |
| PE (2) | PE20190354A1 (es) |
| PH (1) | PH12016500443A1 (es) |
| PL (1) | PL3043827T3 (es) |
| PT (1) | PT3043827T (es) |
| RS (1) | RS59252B1 (es) |
| RU (1) | RU2662967C2 (es) |
| SG (2) | SG10201806787VA (es) |
| SI (1) | SI3043827T1 (es) |
| UA (1) | UA119046C2 (es) |
| WO (1) | WO2015038939A2 (es) |
| ZA (1) | ZA201601236B (es) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE540118T1 (de) | 2006-10-18 | 2012-01-15 | Isis Pharmaceuticals Inc | Antisense-verbindungen |
| WO2012109395A1 (en) | 2011-02-08 | 2012-08-16 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| MX2015015239A (es) | 2013-05-01 | 2016-10-03 | Ionis Pharmaceuticals Inc | Composiciones y metodos. |
| EP3603677A1 (en) * | 2013-09-13 | 2020-02-05 | Ionis Pharmaceuticals, Inc. | Modulators of complement factor b |
| EA201691215A1 (ru) * | 2013-12-12 | 2016-11-30 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ iRNA К КОМПОНЕНТУ КОМПЛЕМЕНТА И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| RS59182B1 (sr) * | 2014-05-01 | 2019-10-31 | Ionis Pharmaceuticals Inc | Kompozicije i postupci za modulaciju ekspresije faktora b komplementa |
| 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 |
| JP7057290B2 (ja) | 2016-06-27 | 2022-04-19 | アキリオン ファーマシューティカルズ，インコーポレーテッド | 医学的障害を治療するためのキナゾリン及びインドール化合物 |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | 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 |
| WO2019027015A1 (ja) * | 2017-08-02 | 2019-02-07 | 協和発酵キリン株式会社 | 核酸複合体 |
| 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 |
| KR20230017789A (ko) * | 2020-04-30 | 2023-02-06 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 인자 B (CFB) iRNA 조성물 및 이의 사용 방법 |
| KR20230049115A (ko) | 2020-08-07 | 2023-04-12 | 상하이 메이유에 바이오테크 디벨롭먼트 컴퍼니 리미티드 | 보체 인자 b 억제제 및 이의 약학적 조성물, 제조 방법 및 용도 |
| 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 |
| CA3225937A1 (en) * | 2021-07-17 | 2023-02-16 | Dmitry Samarsky | Products and compositions |
| EP4396346A1 (en) | 2021-09-02 | 2024-07-10 | Silence Therapeutics GmbH | Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell |
| AU2022378567A1 (en) * | 2021-10-29 | 2024-04-11 | 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 |
| WO2023143293A1 (zh) | 2022-01-26 | 2023-08-03 | 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 |
| WO2024097861A1 (en) * | 2022-11-02 | 2024-05-10 | Ionis Pharmaceuticals, Inc. | Methods for modulating complement factor b expression |
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