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
-
- 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)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
- Epidemiology (AREA)
- Optical Communication System (AREA)
- Amplifiers (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 |
|---|---|
| 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 (32)
Families Citing this family (34)
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| EP2410053B2 (en) | 2006-10-18 | 2020-07-15 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
| EP3626823A1 (en) | 2009-09-11 | 2020-03-25 | Ionis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
| US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| PL2992098T3 (pl) | 2013-05-01 | 2019-09-30 | Ionis Pharmaceuticals, Inc. | Kompozycje i sposoby modulowania ekspresji hbv i ttr |
| PL3043827T3 (pl) * | 2013-09-13 | 2020-03-31 | Ionis Pharmaceuticals, Inc. | Modulatory czynnika b dopełniacza |
| WO2015089368A2 (en) * | 2013-12-12 | 2015-06-18 | Alnylam Pharmaceuticals, Inc. | Complement component irna compositions and methods of use thereof |
| CR20160554A (es) * | 2014-05-01 | 2017-03-07 | Ionis Pharmaceuticals Inc | Composiciones y métodos para modular la expresión 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 |
| MX2018016046A (es) | 2016-06-27 | 2019-05-02 | Achillion Pharmaceuticals Inc | Quinazolina y compuestos indol para tratar trastornos medicos. |
| US20180193270A1 (en) | 2016-11-29 | 2018-07-12 | PureTech Health LLC | 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 |
| CA3078971A1 (en) | 2017-11-01 | 2019-05-09 | 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 |
| CA3158320A1 (en) | 2019-10-22 | 2021-04-29 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof |
| EP4114947A1 (en) | 2020-03-05 | 2023-01-11 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases |
| 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. |
| 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 |
| CN114057692B (zh) | 2020-08-07 | 2023-07-21 | 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 |
| EP4373940A4 (en) | 2021-07-17 | 2025-10-01 | Sirnaomics Inc | PRODUCTS AND COMPOSITIONS |
| US20250136977A1 (en) | 2021-09-02 | 2025-05-01 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell |
| AU2022376760A1 (en) | 2021-10-27 | 2024-05-02 | Hansoh Bio Llc | Piperidinyl indole derivatives, preparation methods and medicinal uses thereof |
| AR127477A1 (es) * | 2021-10-29 | 2024-01-31 | Alnylam Pharmaceuticals Inc | COMPOSICIONES DE ARNi CONTRA EL FACTOR B DEL COMPLEMENTO (CFB) Y MÉTODOS DE USO DE LAS MISMAS |
| EP4437104A4 (en) | 2021-11-24 | 2026-01-21 | Adarx Pharmaceuticals Inc | COMPLEMENT FACTOR B MODULATION COMPOSITIONS AND THEIR METHODS OF USE |
| WO2023129496A2 (en) * | 2021-12-27 | 2023-07-06 | 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 |
| AU2024261191A1 (en) | 2023-04-27 | 2025-11-06 | Jiangsu Hansoh Pharmaceutical Group Co., Ltd. | Salt of piperidylindole compound and preparation method therefor |
| 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 |
| KR20260030856A (ko) * | 2023-06-28 | 2026-03-06 | 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. | 보체 인자 b(cfb) 발현을 억제하기 위한 조성물 및 방법 |
| 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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| WO2010036696A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Cyclohexenyl nucleic acid analogs |
| WO2011139702A2 (en) | 2010-04-28 | 2011-11-10 | Isis Pharmaceuticals, Inc. | Modified nucleosides and oligomeric compounds prepared therefrom |
| ES2756326T3 (es) * | 2010-07-19 | 2020-04-27 | Ionis Pharmaceuticals Inc | Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK) |
| CN105886506A (zh) * | 2011-04-13 | 2016-08-24 | Isis制药公司 | Ptp1b 表达的反义调节 |
| PL3043827T3 (pl) | 2013-09-13 | 2020-03-31 | Ionis Pharmaceuticals, Inc. | Modulatory czynnika b dopełniacza |
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