JP2024545222A - ＬＰＡを標的とするｓｉＲＮＡ及び複合体 - Google Patents
ＬＰＡを標的とするｓｉＲＮＡ及び複合体 Download PDFInfo
- Publication number
- JP2024545222A JP2024545222A JP2024535521A JP2024535521A JP2024545222A JP 2024545222 A JP2024545222 A JP 2024545222A JP 2024535521 A JP2024535521 A JP 2024535521A JP 2024535521 A JP2024535521 A JP 2024535521A JP 2024545222 A JP2024545222 A JP 2024545222A
- Authority
- JP
- Japan
- Prior art keywords
- seq
- group
- sirna
- antisense strand
- nucleotide
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- 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
- C12N2310/322—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
- C12N2310/35—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)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Heart & Thoracic Surgery (AREA)
- Vascular Medicine (AREA)
- Cardiology (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)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202111545699 | 2021-12-16 | ||
| CN202111545699.6 | 2021-12-16 | ||
| PCT/CN2022/139500 WO2023109940A1 (zh) | 2021-12-16 | 2022-12-16 | 靶向lpa的sirna及缀合物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024545222A true JP2024545222A (ja) | 2024-12-05 |
| JP2024545222A5 JP2024545222A5 (https=) | 2025-12-26 |
| JPWO2023109940A5 JPWO2023109940A5 (https=) | 2025-12-26 |
Family
ID=86774879
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024535521A Pending JP2024545222A (ja) | 2021-12-16 | 2022-12-16 | ＬＰＡを標的とするｓｉＲＮＡ及び複合体 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20250332190A1 (https=) |
| EP (1) | EP4450624A4 (https=) |
| JP (1) | JP2024545222A (https=) |
| KR (1) | KR20240116539A (https=) |
| CN (1) | CN118076738A (https=) |
| CA (1) | CA3240604A1 (https=) |
| TW (1) | TW202340465A (https=) |
| WO (1) | WO2023109940A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119506275A (zh) * | 2023-08-22 | 2025-02-25 | 苏州炫景生物科技有限公司 | 抑制lpa基因表达的双链寡核苷酸及其应用 |
| CN121079106A (zh) | 2023-05-31 | 2025-12-05 | 上海拓界生物医药科技有限公司 | 一种靶向LPA的dsRNA及其应用 |
| WO2025064821A2 (en) | 2023-09-21 | 2025-03-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for inhibiting lpa |
| WO2025082059A1 (zh) * | 2023-10-16 | 2025-04-24 | 润佳(上海)医药技术有限公司 | 经修饰的dsRNA、双链RNAi剂及其用途 |
| WO2025120571A1 (en) * | 2023-12-05 | 2025-06-12 | Janssen Biotech, Inc. | Compositions and methods to discover targeted oligonucleotide conjugates |
| WO2025252159A1 (zh) * | 2024-06-06 | 2025-12-11 | 大睿生物医药科技(上海)有限公司 | 调控LPA表达的dsRNA分子 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013299717B2 (en) | 2012-08-06 | 2018-06-28 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
| DK2991656T3 (da) * | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| JOP20160211B1 (ar) * | 2015-10-01 | 2021-08-17 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| WO2018013525A1 (en) * | 2016-07-11 | 2018-01-18 | Translate Bio Ma, Inc. | Nucleic acid conjugates and uses thereof |
| WO2019204668A1 (en) * | 2018-04-18 | 2019-10-24 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease |
| CN111378655B (zh) * | 2018-12-28 | 2024-03-19 | 苏州瑞博生物技术股份有限公司 | 抑制CTGF基因表达的siRNA、含有该siRNA的药物组合物及其用途 |
| CN112876534B (zh) * | 2019-11-29 | 2024-02-09 | 苏州瑞博生物技术股份有限公司 | 肝靶向化合物及缀合物 |
| US20230201361A1 (en) * | 2020-01-11 | 2023-06-29 | Oncolmmunin, Inc. | Targeted and localized in vivo delivery of oligonucleotides |
| WO2021254360A1 (zh) | 2020-06-16 | 2021-12-23 | 上海拓界生物医药科技有限公司 | 一种碳水化合物分子簇及其制备方法和医药用途 |
| KR102812602B1 (ko) * | 2020-08-05 | 2025-05-28 | 다이서나 파마수이티컬, 인크. | Lpa 발현을 저해하기 위한 조성물 및 방법 |
| WO2022121959A1 (zh) * | 2020-12-09 | 2022-06-16 | 纳肽得有限公司 | siRNA分子及其在治疗冠状动脉疾病中的应用 |
| JP2024534508A (ja) * | 2021-09-18 | 2024-09-20 | 成都心正合医▲葯▼科技有限公司 | Ｌｐａ阻害剤及びその使用 |
| CN114703184B (zh) * | 2022-03-11 | 2024-06-18 | 厦门甘宝利生物医药有限公司 | Lpa抑制剂及其用途 |
| CN115851723B (zh) * | 2022-10-24 | 2023-10-03 | 厦门甘宝利生物医药有限公司 | 一种抑制lpa基因表达的rna抑制剂及其应用 |
-
2022
- 2022-12-16 WO PCT/CN2022/139500 patent/WO2023109940A1/zh not_active Ceased
- 2022-12-16 JP JP2024535521A patent/JP2024545222A/ja active Pending
- 2022-12-16 US US18/719,828 patent/US20250332190A1/en active Pending
- 2022-12-16 CN CN202280067981.4A patent/CN118076738A/zh active Pending
- 2022-12-16 EP EP22906685.7A patent/EP4450624A4/en active Pending
- 2022-12-16 TW TW111148548A patent/TW202340465A/zh unknown
- 2022-12-16 KR KR1020247022570A patent/KR20240116539A/ko active Pending
- 2022-12-16 CA CA3240604A patent/CA3240604A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4450624A4 (en) | 2025-03-26 |
| CN118076738A (zh) | 2024-05-24 |
| TW202340465A (zh) | 2023-10-16 |
| KR20240116539A (ko) | 2024-07-29 |
| EP4450624A1 (en) | 2024-10-23 |
| US20250332190A1 (en) | 2025-10-30 |
| CA3240604A1 (en) | 2023-06-22 |
| WO2023109940A1 (zh) | 2023-06-22 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20251216 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20251216 |