KR20230173116A - 보체 성분 3 발현을 저해하기 위한 조성물 및 방법 - Google Patents
보체 성분 3 발현을 저해하기 위한 조성물 및 방법 Download PDFInfo
- Publication number
- KR20230173116A KR20230173116A KR1020237036421A KR20237036421A KR20230173116A KR 20230173116 A KR20230173116 A KR 20230173116A KR 1020237036421 A KR1020237036421 A KR 1020237036421A KR 20237036421 A KR20237036421 A KR 20237036421A KR 20230173116 A KR20230173116 A KR 20230173116A
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- South Korea
- Prior art keywords
- oligonucleotide
- pharmaceutically acceptable
- nucleotides
- acceptable salt
- rnai
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- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychiatry (AREA)
- Urology & Nephrology (AREA)
- Rheumatology (AREA)
- Transplantation (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163177254P | 2021-04-20 | 2021-04-20 | |
| US63/177,254 | 2021-04-20 | ||
| PCT/US2022/025648 WO2022226127A1 (en) | 2021-04-20 | 2022-04-20 | Compositions and methods for inhibiting complement component 3 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20230173116A true KR20230173116A (ko) | 2023-12-26 |
Family
ID=83723122
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020237036421A Pending KR20230173116A (ko) | 2021-04-20 | 2022-04-20 | 보체 성분 3 발현을 저해하기 위한 조성물 및 방법 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20250002908A1 (https=) |
| EP (1) | EP4326876A4 (https=) |
| JP (2) | JP2024515344A (https=) |
| KR (1) | KR20230173116A (https=) |
| CN (1) | CN117295819A (https=) |
| AU (1) | AU2022261922A1 (https=) |
| BR (1) | BR112023021703A2 (https=) |
| CA (1) | CA3177629A1 (https=) |
| CO (1) | CO2023014681A2 (https=) |
| IL (1) | IL307721A (https=) |
| MX (1) | MX2023012385A (https=) |
| WO (1) | WO2022226127A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TWI868755B (zh) | 2022-06-24 | 2025-01-01 | 丹麥商諾佛 儂迪克股份有限公司 | 抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法 |
| WO2024169907A1 (zh) * | 2023-02-17 | 2024-08-22 | 苏州时安生物技术有限公司 | 一种调节补体C3表达的siRNA、其缀合物和药物组合物及用途 |
| CN121488036A (zh) * | 2023-07-21 | 2026-02-06 | 上海舶望制药有限公司 | 用于抑制补体成分3(c3)表达的组合物和方法 |
| WO2025168134A1 (zh) * | 2024-02-08 | 2025-08-14 | 北京安龙生物医药有限公司 | 靶向补体成分c3基因的寡核苷酸及其用途 |
| CN121154665B (zh) * | 2025-11-21 | 2026-02-27 | 天津医科大学第二医院 | 尿苷在防治糖尿病肾病药物开发过程中及药物中的应用 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2305812A3 (en) * | 2002-11-14 | 2012-06-06 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| WO2007064846A2 (en) * | 2005-11-30 | 2007-06-07 | Intradigm Corporation | COMPOSITIONS AND METHODS OF USING siRNA TO KNOCKDOWN GENE EXPRESSION AND TO IMPROVE SOLID ORGAN AND CELL TRANSPLANTATION |
| WO2010042755A2 (en) * | 2008-10-08 | 2010-04-15 | Chimeros Inc. | Chimeric therapeutics, compositions, and methods for using same |
| SG10201804960RA (en) * | 2013-12-12 | 2018-07-30 | Alnylam Pharmaceuticals Inc | Complement component irna compositions and methods of use thereof |
| RU2019113301A (ru) * | 2016-10-17 | 2020-11-17 | Апеллис Фармасьютикалс, Инк. | Комбинированная терапия для ингибирования с3 |
| AU2018360697A1 (en) * | 2017-11-01 | 2020-05-14 | Alnylam Pharmaceuticals, Inc. | Complement component C3 iRNA compositions and methods of use thereof |
| CA3119234A1 (en) * | 2018-11-23 | 2020-05-28 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of c3 in a cell |
| EP4022060A1 (en) * | 2019-08-27 | 2022-07-06 | Silence Therapeutics GmbH | Nucleic acids for inhibiting expression of c3 in a cell |
| TW202134435A (zh) * | 2019-10-22 | 2021-09-16 | 美商阿尼拉製藥公司 | 補體成分c3 irna組成物及其使用方法 |
| CN115335524A (zh) * | 2020-02-14 | 2022-11-11 | 阿佩利斯制药有限公司 | 用于补体抑制的rna |
| MX2023008469A (es) * | 2021-01-22 | 2023-11-22 | Alnylam Pharmaceuticals Inc | Oligonucleotidos de doble hebra modificados. |
| WO2022251484A1 (en) * | 2021-05-26 | 2022-12-01 | Apellis Pharmaceuticals, Inc. | Treatment of complement-mediated disorders |
-
2022
- 2022-04-20 WO PCT/US2022/025648 patent/WO2022226127A1/en not_active Ceased
- 2022-04-20 CN CN202280034163.4A patent/CN117295819A/zh active Pending
- 2022-04-20 MX MX2023012385A patent/MX2023012385A/es unknown
- 2022-04-20 KR KR1020237036421A patent/KR20230173116A/ko active Pending
- 2022-04-20 AU AU2022261922A patent/AU2022261922A1/en active Pending
- 2022-04-20 CA CA3177629A patent/CA3177629A1/en active Pending
- 2022-04-20 IL IL307721A patent/IL307721A/en unknown
- 2022-04-20 US US18/287,260 patent/US20250002908A1/en active Pending
- 2022-04-20 JP JP2023564255A patent/JP2024515344A/ja active Pending
- 2022-04-20 BR BR112023021703A patent/BR112023021703A2/pt unknown
- 2022-04-20 EP EP22792454.5A patent/EP4326876A4/en active Pending
-
2023
- 2023-10-30 CO CONC2023/0014681A patent/CO2023014681A2/es unknown
-
2025
- 2025-04-15 JP JP2025066627A patent/JP2025100729A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| BR112023021703A2 (pt) | 2024-01-30 |
| WO2022226127A1 (en) | 2022-10-27 |
| CO2023014681A2 (es) | 2024-01-25 |
| EP4326876A1 (en) | 2024-02-28 |
| JP2025100729A (ja) | 2025-07-03 |
| US20250002908A1 (en) | 2025-01-02 |
| CN117295819A (zh) | 2023-12-26 |
| IL307721A (en) | 2023-12-01 |
| CA3177629A1 (en) | 2022-10-27 |
| AU2022261922A1 (en) | 2023-10-26 |
| JP2024515344A (ja) | 2024-04-09 |
| EP4326876A4 (en) | 2026-04-08 |
| MX2023012385A (es) | 2024-02-07 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20231024 Patent event code: PA01051R01D Comment text: International Patent Application |
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| PG1501 | Laying open of application | ||
| A201 | Request for examination | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20250418 Comment text: Request for Examination of Application |