PE20160500A1 - Moduladores del factor del complemento b - Google Patents
Moduladores del factor del complemento bInfo
- Publication number
- PE20160500A1 PE20160500A1 PE2016000376A PE2016000376A PE20160500A1 PE 20160500 A1 PE20160500 A1 PE 20160500A1 PE 2016000376 A PE2016000376 A PE 2016000376A PE 2016000376 A PE2016000376 A PE 2016000376A PE 20160500 A1 PE20160500 A1 PE 20160500A1
- Authority
- PE
- Peru
- Prior art keywords
- modified
- seq
- antisense compounds
- nucleobase
- compounds
- Prior art date
Links
- 230000000295 complement effect Effects 0.000 title abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 5
- 102000003712 Complement factor B Human genes 0.000 abstract 3
- 108090000056 Complement factor B Proteins 0.000 abstract 3
- 108091034117 Oligonucleotide Proteins 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 abstract 2
- 206010064930 age-related macular degeneration Diseases 0.000 abstract 2
- 208000002780 macular degeneration Diseases 0.000 abstract 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 abstract 1
- 208000035913 Atypical hemolytic uremic syndrome Diseases 0.000 abstract 1
- 102100035325 Complement factor H-related protein 5 Human genes 0.000 abstract 1
- 206010018364 Glomerulonephritis Diseases 0.000 abstract 1
- 101000878134 Homo sapiens Complement factor H-related protein 5 Proteins 0.000 abstract 1
- 208000005777 Lupus Nephritis Diseases 0.000 abstract 1
- 125000002619 bicyclic group Chemical group 0.000 abstract 1
- 230000024203 complement activation Effects 0.000 abstract 1
- 230000003831 deregulation Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000017169 kidney disease Diseases 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 239000002777 nucleoside Substances 0.000 abstract 1
- 125000003835 nucleoside group Chemical group 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 201000000596 systemic lupus erythematosus Diseases 0.000 abstract 1
- 238000002560 therapeutic procedure Methods 0.000 abstract 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 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
-
- 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)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (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)
- Saccharide Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (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 |
|---|---|
| PE20160500A1 true PE20160500A1 (es) | 2016-05-21 |
Family
ID=52666522
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2016000376A PE20160500A1 (es) | 2013-09-13 | 2014-09-12 | Moduladores del factor del complemento b |
| PE2018003111A PE20190354A1 (es) | 2013-09-13 | 2014-09-12 | Moduladores del factor del complemento b |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2018003111A PE20190354A1 (es) | 2013-09-13 | 2014-09-12 | Moduladores del factor del complemento b |
Country Status (32)
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5665317B2 (ja) | 2006-10-18 | 2015-02-04 | 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 | アンチセンス化合物 |
| RU2562861C2 (ru) | 2009-09-11 | 2015-09-10 | Айсис Фармасьютикалс, Инк. | Модуляция экспрессии гентингтина |
| EP2673361B1 (en) | 2011-02-08 | 2016-04-13 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| KR102482890B1 (ko) | 2013-05-01 | 2022-12-30 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법 |
| EP3043827B1 (en) | 2013-09-13 | 2019-07-03 | Ionis Pharmaceuticals, Inc. | Modulators of complement factor b |
| KR102344559B1 (ko) | 2013-12-12 | 2021-12-31 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법 |
| PE20170010A1 (es) * | 2014-05-01 | 2017-03-04 | Ionis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion del factor b del complemento |
| 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 |
| AU2017290593A1 (en) | 2016-06-27 | 2019-01-03 | Achillion Pharmaceuticals, Inc. | Quinazoline and indole compounds to treat medical disorders |
| 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 |
| WO2019027015A1 (ja) * | 2017-08-02 | 2019-02-07 | 協和発酵キリン株式会社 | 核酸複合体 |
| 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 |
| KR20230017789A (ko) * | 2020-04-30 | 2023-02-06 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 인자 B (CFB) iRNA 조성물 및 이의 사용 방법 |
| US20230331710A1 (en) | 2020-08-07 | 2023-10-19 | Shanghai Meiyue Biotech Development Co. Ltd | Heterocyclic compound, preparation method and use thereof |
| IL300432A (en) | 2020-08-07 | 2023-04-01 | Shanghai Meiyue Biotech Dev Co Ltd | An inhibitor of complement factor B, and the composition of the drugs, the method of preparation therefor and its use |
| CA3225937A1 (en) | 2021-07-17 | 2023-02-16 | Dmitry Samarsky | Products and compositions |
| 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. |
| EP4423273A1 (en) * | 2021-10-29 | 2024-09-04 | 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 |
| 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 |
| CN119923389A (zh) | 2022-09-10 | 2025-05-02 | 江苏豪森药业集团有限公司 | 2-取代哌啶衍生物、其制备方法和医药用途 |
| IL320604A (en) * | 2022-11-02 | 2025-07-01 | 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 |
| 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 |
| 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 |
| 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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