JP2024546667A - 核酸、当該核酸を含む組成物及び複合体並びに調製方法と使用 - Google Patents
核酸、当該核酸を含む組成物及び複合体並びに調製方法と使用 Download PDFInfo
- Publication number
- JP2024546667A JP2024546667A JP2024533904A JP2024533904A JP2024546667A JP 2024546667 A JP2024546667 A JP 2024546667A JP 2024533904 A JP2024533904 A JP 2024533904A JP 2024533904 A JP2024533904 A JP 2024533904A JP 2024546667 A JP2024546667 A JP 2024546667A
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- nucleotide
- sirna
- seq
- nucleotides
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- 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
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- 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
-
- 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
-
- 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 nucleic acids [NA]
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Hospice & Palliative Care (AREA)
- Gastroenterology & Hepatology (AREA)
- Psychiatry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202111575800 | 2021-12-21 | ||
| CN202111575800.2 | 2021-12-21 | ||
| PCT/CN2022/139942 WO2023116607A1 (zh) | 2021-12-21 | 2022-12-19 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024546667A true JP2024546667A (ja) | 2024-12-26 |
| JPWO2023116607A5 JPWO2023116607A5 (https=) | 2025-12-24 |
| JP2024546667A5 JP2024546667A5 (https=) | 2025-12-24 |
Family
ID=86901326
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024533904A Pending JP2024546667A (ja) | 2021-12-21 | 2022-12-19 | 核酸、当該核酸を含む組成物及び複合体並びに調製方法と使用 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20250057870A1 (https=) |
| EP (1) | EP4455284A1 (https=) |
| JP (1) | JP2024546667A (https=) |
| KR (1) | KR20240117104A (https=) |
| CN (1) | CN118202049A (https=) |
| AU (1) | AU2022422864A1 (https=) |
| CA (1) | CA3242059A1 (https=) |
| WO (1) | WO2023116607A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2020280438B2 (en) | 2019-05-22 | 2025-03-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| JP7610268B2 (ja) | 2019-05-22 | 2025-01-08 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、薬物組成物及び複合体ならびに調製方法と使用 |
| WO2020233680A1 (zh) | 2019-05-22 | 2020-11-26 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与缀合物及制备方法和用途 |
| EP3978609A4 (en) | 2019-05-24 | 2024-02-07 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| CN121780524A (zh) * | 2024-08-02 | 2026-04-03 | 北京尧景基因技术有限公司 | 抑制MAPT基因表达的siRNA及其缀合物和应用 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009143371A2 (en) * | 2008-05-21 | 2009-11-26 | Intradigm Corporation | COMPOSITIONS COMPRISING mTOR SIRNA AND METHODS OF USE THEREOF |
| WO2010064851A2 (ko) * | 2008-12-02 | 2010-06-10 | 울산대학교 산학협력단 | 종간 교차활성을 지닌 mTOR을 표적으로 하는 siRNA, 이를 포함하는 재조합벡터 및 이를 함유하는 약학조성물 |
| US20120136073A1 (en) | 2010-11-15 | 2012-05-31 | Life Technologies Corporation | Amine-Containing Transfection Reagents and methods for making and using same |
| WO2017151732A1 (en) * | 2016-03-02 | 2017-09-08 | Beth Israel Deaconess Medical Center Technology Venture Office, Br2 | Therapeutic targets for lin-28-expressing cancers |
| AU2018216509B2 (en) * | 2017-01-31 | 2024-02-29 | Curigin Co.,Ltd. | Nucleic acid simultaneously inhibiting expression of mTOR gene and STAT3 gene |
| TN2019000308A1 (en) | 2017-07-06 | 2021-05-07 | Arrowhead Pharmaceuticals Inc | RNAi AGENTS FOR INHIBITING EXPRESSION OF ALPHA-ENaC AND METHODS OF USE |
| WO2019105437A1 (zh) | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
| EP3732185B1 (en) | 2017-12-29 | 2025-02-26 | Suzhou Ribo Life Science Co., Ltd. | Conjugates and preparation and use thereof |
| WO2019217459A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| WO2020018461A1 (en) * | 2018-07-16 | 2020-01-23 | The University Of Virginia Patent Foundation | Compositions and methods of diagnosis and treatment for neurological diseases |
| CN112423795A (zh) * | 2018-12-28 | 2021-02-26 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
-
2022
- 2022-12-19 AU AU2022422864A patent/AU2022422864A1/en active Pending
- 2022-12-19 EP EP22909920.5A patent/EP4455284A1/en active Pending
- 2022-12-19 CN CN202280068763.2A patent/CN118202049A/zh active Pending
- 2022-12-19 KR KR1020247021150A patent/KR20240117104A/ko active Pending
- 2022-12-19 JP JP2024533904A patent/JP2024546667A/ja active Pending
- 2022-12-19 US US18/722,280 patent/US20250057870A1/en active Pending
- 2022-12-19 WO PCT/CN2022/139942 patent/WO2023116607A1/zh not_active Ceased
- 2022-12-19 CA CA3242059A patent/CA3242059A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN118202049A (zh) | 2024-06-14 |
| AU2022422864A1 (en) | 2024-07-04 |
| CA3242059A1 (en) | 2023-06-29 |
| WO2023116607A1 (zh) | 2023-06-29 |
| WO2023116607A9 (zh) | 2023-10-05 |
| US20250057870A1 (en) | 2025-02-20 |
| EP4455284A1 (en) | 2024-10-30 |
| KR20240117104A (ko) | 2024-07-31 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6715325B2 (ja) | siRNA、siRNAを含む医薬組成物及び結合体、並びにそれらの応用 | |
| JP2024546667A (ja) | 核酸、当該核酸を含む組成物及び複合体並びに調製方法と使用 | |
| JP7488254B2 (ja) | 17β-HSD13型(HSD17B13)の発現を阻害するためのRNAi剤、その組成物、および使用方法 | |
| CN103747805B (zh) | 用于治疗、延缓和/或预防人类遗传性疾病如强直性肌营养不良症1型(dm1)的化合物 | |
| AU2008314860B2 (en) | Lipid-modified double-stranded RNA having potent RNA interference effect | |
| CN108239644B (zh) | 一种小干扰核酸和药物组合物及其用途 | |
| JP2024528634A (ja) | 二本鎖オリゴヌクレオチド、二本鎖オリゴヌクレオチドを含む組成物及び複合体並びに調製方法と使用 | |
| CA3020487A1 (en) | Treatment of idiopathic pulmonary fibrosis using rna complexes that target connective tissue growth factor | |
| CN108220293A (zh) | 一种小干扰核酸和药物组合物及其用途 | |
| CN115819484A (zh) | 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 | |
| CN120699970A (zh) | 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法 | |
| CA3232743A1 (en) | Polynucleic acid molecules targeting pcsk9 and uses thereof | |
| JP2024525800A (ja) | 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用 | |
| CN120981570A (zh) | 靶向agt的多核酸分子及其用途 | |
| CN108210510B (zh) | 一种小干扰核酸药物组合物及其用途 | |
| WO2026046236A1 (zh) | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 | |
| CN118480552B (zh) | 一种靶向CEBPA基因表达的saRNA及其用途 | |
| CN114632089B (zh) | 含酰基糖胺基团化合物在制备治疗新型冠状病毒肺炎药物中的应用及疾病的治疗方法 | |
| WO2026057066A1 (zh) | 寡核苷酸、寡核苷酸缀合物及组合物和用途 | |
| TW202606729A (zh) | 靶向inhbe之聚核酸分子及其用途 | |
| JP2026507286A (ja) | 医薬組成物及びその使用 | |
| WO2023168202A2 (en) | Certain dux4 inhibitors and methods of use thereof | |
| KR20260008676A (ko) | HBV 유전자 발현을 표적하고 조절하기 위한 이중 가닥 RNAi 제제 및 그 응용 | |
| AU2024323785A1 (en) | Sirna, sirna conjugate, pharmaceutical composition and use thereof | |
| TW202440921A (zh) | 經修飾之寡核苷酸 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20251215 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20251215 |