CO2022003455A2 - Ácidos nucleicos para inhibir la expresión de c3 en una célula - Google Patents
Ácidos nucleicos para inhibir la expresión de c3 en una célulaInfo
- Publication number
- CO2022003455A2 CO2022003455A2 CONC2022/0003455A CO2022003455A CO2022003455A2 CO 2022003455 A2 CO2022003455 A2 CO 2022003455A2 CO 2022003455 A CO2022003455 A CO 2022003455A CO 2022003455 A2 CO2022003455 A2 CO 2022003455A2
- Authority
- CO
- Colombia
- 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
-
- 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
-
- 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.
-
- 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
-
- 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
-
- 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
- C12N2310/34—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
-
- 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
-
- 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
-
- 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
La invención se refiere a productos de ácido nucleico que interfieren con la expresión génica del componente C3 del complemento o que inhiben su expresión. Los ácidos nucleicos, preferentemente, son para ser usados como tratamiento, prevención o reducción del riesgo de padecer de enfermedades, desórdenes o síndromes asociados con el componente C3 del complemento, particularmente la Glomerulopatía por C3 (C3G), Hemoglobinuria Paroxística Nocturna (HPN), Síndrome Hemolítico Urémico atípico (SHUa), Nefritis Lúpica, Nefropatía por IgA (IgA N), Enfermedad por Aglutinina Fría (CAD), Miastenia Gravis (MG), y la Nefropatía Membranosa primaria.
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 |
---|---|
CO2022003455A2 true CO2022003455A2 (es) | 2022-05-10 |
Family
ID=72148160
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2022/0003455A CO2022003455A2 (es) | 2019-08-27 | 2022-03-24 | Ácidos nucleicos para inhibir la expresión de c3 en una célula |
Country Status (12)
Country | Link |
---|---|
US (2) | US20220195436A1 (es) |
EP (1) | EP4022060A1 (es) |
JP (1) | JP2022547429A (es) |
KR (1) | KR20220047865A (es) |
CN (1) | CN114981430A (es) |
AU (1) | AU2020339703A1 (es) |
BR (1) | BR112022003513A2 (es) |
CA (1) | CA3149534A1 (es) |
CO (1) | CO2022003455A2 (es) |
IL (1) | IL290750A (es) |
MX (1) | MX2022002397A (es) |
WO (1) | WO2021037941A1 (es) |
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 |
RU2487716C2 (ru) * | 2007-10-03 | 2013-07-20 | Кварк Фармасьютикалс, Инк. | Новые структуры малых интерферирующих рнк (sirna) |
EA201591707A1 (ru) * | 2013-03-14 | 2016-03-31 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ иРНК КОМПОНЕНТА КОМПЛЕМЕНТА C5 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
WO2015089368A2 (en) * | 2013-12-12 | 2015-06-18 | Alnylam Pharmaceuticals, Inc. | Complement component irna compositions and methods of use thereof |
JP6594902B2 (ja) * | 2014-02-11 | 2019-10-23 | 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 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
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 |
WO2019089922A1 (en) * | 2017-11-01 | 2019-05-09 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof |
US20220017899A1 (en) | 2018-11-23 | 2022-01-20 | 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 JP JP2022513321A patent/JP2022547429A/ja active Pending
- 2020-08-26 KR KR1020227009600A patent/KR20220047865A/ko unknown
- 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 CA CA3149534A patent/CA3149534A1/en active Pending
- 2020-08-26 US US17/612,120 patent/US20220195436A1/en active Pending
- 2020-08-26 WO PCT/EP2020/073904 patent/WO2021037941A1/en unknown
- 2020-08-26 AU AU2020339703A patent/AU2020339703A1/en not_active Abandoned
-
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 |
---|---|
MX2022002397A (es) | 2022-08-25 |
US11753642B2 (en) | 2023-09-12 |
US20220195436A1 (en) | 2022-06-23 |
CA3149534A1 (en) | 2021-03-04 |
KR20220047865A (ko) | 2022-04-19 |
CN114981430A (zh) | 2022-08-30 |
WO2021037941A1 (en) | 2021-03-04 |
EP4022060A1 (en) | 2022-07-06 |
JP2022547429A (ja) | 2022-11-14 |
AU2020339703A1 (en) | 2022-03-17 |
US20230024926A1 (en) | 2023-01-26 |
BR112022003513A2 (pt) | 2022-05-17 |
IL290750A (en) | 2022-04-01 |
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