BR112022003513A2 - Ácidos nucleicos para inibição da expressão de c3 em uma célula - Google Patents
Ácidos nucleicos para inibição da expressão de c3 em uma célulaInfo
- Publication number
- BR112022003513A2 BR112022003513A2 BR112022003513A BR112022003513A BR112022003513A2 BR 112022003513 A2 BR112022003513 A2 BR 112022003513A2 BR 112022003513 A BR112022003513 A BR 112022003513A BR 112022003513 A BR112022003513 A BR 112022003513A BR 112022003513 A2 BR112022003513 A2 BR 112022003513A2
- Authority
- BR
- Brazil
- Prior art keywords
- expression
- nucleic acids
- inhibiting
- cell
- iga
- Prior art date
Links
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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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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
- C12N2310/14—Type of nucleic acid interfering N.A.
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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
- C12N2310/312—Phosphonates
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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
- C12N2310/312—Phosphonates
- C12N2310/3125—Methylphosphonates
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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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
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Urology & Nephrology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Abstract
ácidos nucleicos para inibição da expressão de c3 em uma célula. a presente invenção refere-se aos produtos de ácido nucleico que interferem com a expressão do gene c3 do componente do complemento ou inibiem sua expressão. os ácidos nucleicos são preferivelmente, para uso como tratamento, prevenção ou redução do risco de sofrer de doenças, distúrbios ou síndromes associadas com o componente c3 do complemento, particularmente glomerulopatia c3 (c3g), hemoglobinúria paroxística noturna (pnh), síndrome hemolítico-urêmica atípica (ahus), nefrite lúpica, nefropatia por iga (iga n), doença por aglutinina a frio (cad), miastenia grave (mg), e nefropatia membranosa primária.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP19193840 | 2019-08-27 | ||
EP19219497 | 2019-12-23 | ||
EP20176947 | 2020-05-27 | ||
PCT/EP2020/073904 WO2021037941A1 (en) | 2019-08-27 | 2020-08-26 | Nucleic acids for inhibiting expression of c3 in a cell |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112022003513A2 true BR112022003513A2 (pt) | 2022-05-17 |
Family
ID=72148160
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022003513A BR112022003513A2 (pt) | 2019-08-27 | 2020-08-26 | Ácidos nucleicos para inibição da expressão de c3 em uma célula |
Country Status (12)
Country | Link |
---|---|
US (2) | US20220195436A1 (pt) |
EP (1) | EP4022060A1 (pt) |
JP (1) | JP2022547429A (pt) |
KR (1) | KR20220047865A (pt) |
CN (1) | CN114981430A (pt) |
AU (1) | AU2020339703A1 (pt) |
BR (1) | BR112022003513A2 (pt) |
CA (1) | CA3149534A1 (pt) |
CO (1) | CO2022003455A2 (pt) |
IL (1) | IL290750A (pt) |
MX (1) | MX2022002397A (pt) |
WO (1) | WO2021037941A1 (pt) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2024013334A1 (en) * | 2022-07-15 | 2024-01-18 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of agt in a cell |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
NL9201440A (nl) | 1992-08-11 | 1994-03-01 | Univ Leiden | Triantennaire clusterglycosiden, hun bereiding en toepassing. |
US6074642A (en) | 1994-05-02 | 2000-06-13 | Alexion Pharmaceuticals, Inc. | Use of antibodies specific to human complement component C5 for the treatment of glomerulonephritis |
US20030096775A1 (en) | 2001-10-23 | 2003-05-22 | Isis Pharmaceuticals Inc. | Antisense modulation of complement component C3 expression |
WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
JPWO2005116204A1 (ja) * | 2004-05-11 | 2008-06-19 | 株式会社アルファジェン | Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
KR20100085951A (ko) * | 2007-10-03 | 2010-07-29 | 쿠아크 파마수티칼스 인코퍼레이티드 | 신규 sirna 구조 |
MX366660B (es) * | 2013-03-14 | 2019-07-18 | Alnylam Pharmaceuticals Inc | Composiciones de arni contra el componente c5 del complemento y métodos para su uso. |
JP6710638B2 (ja) * | 2013-12-12 | 2020-06-17 | 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Alnylam Pharmaceuticals, Inc. | 補体成分iRNA組成物及びその使用方法 |
EP3960860A3 (en) * | 2014-02-11 | 2022-06-08 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (khk) irna compositions and methods of use thereof |
WO2016201301A1 (en) * | 2015-06-12 | 2016-12-15 | Alnylam Pharmaceuticals, Inc. | Complement component c5 irna compositions and methods of use thereof |
EP3228326A1 (en) * | 2016-04-05 | 2017-10-11 | Silence Therapeutics GmbH | Nucleic acid linked to a trivalent glycoconjugate |
EP3704252A1 (en) * | 2017-11-01 | 2020-09-09 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof |
EP3884052A1 (en) | 2018-11-23 | 2021-09-29 | Silence Therapeutics GmbH | Nucleic acids for inhibiting expression of c3 in a cell |
-
2020
- 2020-08-26 EP EP20758259.4A patent/EP4022060A1/en not_active Withdrawn
- 2020-08-26 MX MX2022002397A patent/MX2022002397A/es unknown
- 2020-08-26 BR BR112022003513A patent/BR112022003513A2/pt unknown
- 2020-08-26 CN CN202080074954.0A patent/CN114981430A/zh active Pending
- 2020-08-26 US US17/612,120 patent/US20220195436A1/en active Pending
- 2020-08-26 CA CA3149534A patent/CA3149534A1/en active Pending
- 2020-08-26 JP JP2022513321A patent/JP2022547429A/ja active Pending
- 2020-08-26 KR KR1020227009600A patent/KR20220047865A/ko unknown
- 2020-08-26 AU AU2020339703A patent/AU2020339703A1/en active Pending
- 2020-08-26 WO PCT/EP2020/073904 patent/WO2021037941A1/en unknown
-
2022
- 2022-02-20 IL IL290750A patent/IL290750A/en unknown
- 2022-03-24 CO CONC2022/0003455A patent/CO2022003455A2/es unknown
- 2022-05-26 US US17/825,376 patent/US11753642B2/en active Active
Also Published As
Publication number | Publication date |
---|---|
JP2022547429A (ja) | 2022-11-14 |
CA3149534A1 (en) | 2021-03-04 |
IL290750A (en) | 2022-04-01 |
AU2020339703A1 (en) | 2022-03-17 |
US20220195436A1 (en) | 2022-06-23 |
KR20220047865A (ko) | 2022-04-19 |
US20230024926A1 (en) | 2023-01-26 |
EP4022060A1 (en) | 2022-07-06 |
CO2022003455A2 (es) | 2022-05-10 |
CN114981430A (zh) | 2022-08-30 |
WO2021037941A1 (en) | 2021-03-04 |
US11753642B2 (en) | 2023-09-12 |
MX2022002397A (es) | 2022-08-25 |
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