DK3019200T3 - Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf - Google Patents
Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf Download PDFInfo
- Publication number
- DK3019200T3 DK3019200T3 DK14744737.9T DK14744737T DK3019200T3 DK 3019200 T3 DK3019200 T3 DK 3019200T3 DK 14744737 T DK14744737 T DK 14744737T DK 3019200 T3 DK3019200 T3 DK 3019200T3
- Authority
- DK
- Denmark
- Prior art keywords
- oligonucleotide
- preparation
- ligand conjugates
- conjugates
- ligand
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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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- 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
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding 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
- 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
- C12N2310/35—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
- C12N2320/30—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
-
- 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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361845279P | 2013-07-11 | 2013-07-11 | |
| PCT/US2014/046425 WO2015006740A2 (en) | 2013-07-11 | 2014-07-11 | Oligonucleotide-ligand conjugates and process for their preparation |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3019200T3 true DK3019200T3 (da) | 2022-06-20 |
Family
ID=51230239
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK14744737.9T DK3019200T3 (da) | 2013-07-11 | 2014-07-11 | Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf |
Country Status (7)
| Country | Link |
|---|---|
| US (3) | US10808246B2 (https=) |
| EP (3) | EP3019200B1 (https=) |
| JP (4) | JP6702862B2 (https=) |
| AU (4) | AU2014287002A1 (https=) |
| CA (2) | CA3177846A1 (https=) |
| DK (1) | DK3019200T3 (https=) |
| WO (1) | WO2015006740A2 (https=) |
Families Citing this family (129)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| SG11201608502TA (en) * | 2014-05-01 | 2016-11-29 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
| US10570169B2 (en) | 2014-05-22 | 2020-02-25 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| WO2015188194A1 (en) * | 2014-06-06 | 2015-12-10 | Isis Pharmaceuticals, Inc. | Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds |
| JP7105065B2 (ja) | 2014-12-15 | 2022-07-22 | ダイセルナ ファーマシューティカルズ, インコーポレイテッド | リガンド修飾二本鎖核酸 |
| EP3303365A4 (en) * | 2015-05-29 | 2019-04-24 | Ascendis Pharma Inc. | PRODRUGS WITH A PYROGLUTAMATE LINKER |
| EP3323893A4 (en) * | 2015-07-16 | 2019-03-27 | Kyowa Hakko Kirin Co., Ltd. | BETA2GPI GENE EXPRESSION-INHIBITING NUCLEIC ACID COMPLEX |
| CN105524124B (zh) * | 2015-12-31 | 2018-10-30 | 天津英吉诺科技有限公司 | 一种n-乙酰氨基半乳糖的绿色合成方法 |
| AU2017213404A1 (en) | 2016-01-29 | 2018-09-20 | Kyowa Kirin Co., Ltd. | Nucleic acid conjugate |
| KR102515329B1 (ko) | 2016-03-07 | 2023-03-29 | 애로우헤드 파마슈티컬스 인코포레이티드 | 치료용 화합물을 위한 표적화 리간드 |
| EP3442983A1 (en) * | 2016-04-14 | 2019-02-20 | H. Hoffnabb-La Roche Ag | TRITYL-MONO-Ga1NAc COMPOUNDS AND THEIR USE |
| SG10201913477UA (en) | 2016-04-29 | 2020-02-27 | Univ Nanyang Tech | G-quadruplex-containing antisense oligonucleotides |
| TWI746590B (zh) * | 2016-06-30 | 2021-11-21 | 日商協和麒麟股份有限公司 | 核酸複合體 |
| WO2018013525A1 (en) | 2016-07-11 | 2018-01-18 | Translate Bio Ma, Inc. | Nucleic acid conjugates and uses thereof |
| IL291323B2 (en) | 2016-07-15 | 2023-09-01 | Am Chemicals Llc | Solid non-nucleoside supports and phosphoramidite building blocks for oligonucleotide synthesis |
| MX2018009853A (es) | 2016-09-02 | 2018-11-09 | Arrowhead Pharmaceuticals Inc | Ligandos de direccion. |
| EP3522898A4 (en) * | 2016-10-06 | 2020-05-27 | Ionis Pharmaceuticals, Inc. | METHOD FOR CONJUGATING OLIGOMERIC COMPOUNDS |
| US11963974B2 (en) | 2017-03-10 | 2024-04-23 | National Center For Child Health And Development | Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia |
| AU2018237139A1 (en) * | 2017-03-22 | 2019-10-17 | The Regents Of The University Of California | Modified oligonucleotides and therapeutic uses thereof |
| JP7155239B2 (ja) | 2017-04-05 | 2022-10-18 | サイレンス・セラピューティクス・ゲーエムベーハー | 産物及び組成物 |
| WO2018199340A1 (ja) | 2017-04-28 | 2018-11-01 | 協和発酵キリン株式会社 | オリゴヌクレオチド誘導体またはその塩 |
| WO2019027015A1 (ja) * | 2017-08-02 | 2019-02-07 | 協和発酵キリン株式会社 | 核酸複合体 |
| WO2019027009A1 (ja) * | 2017-08-02 | 2019-02-07 | 協和発酵キリン株式会社 | 核酸複合体 |
| EP3682007A2 (en) * | 2017-09-14 | 2020-07-22 | Janssen BioPharma, Inc. | Galnac derivatives |
| WO2019105403A1 (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 |
| CA3083968C (en) | 2017-12-01 | 2024-04-23 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof |
| WO2019105435A1 (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 |
| JP7365052B2 (ja) * | 2017-12-01 | 2023-10-19 | 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、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 |
| 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 |
| CN110945130B (zh) * | 2017-12-01 | 2024-04-09 | 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 |
| CN111050807B (zh) * | 2017-12-01 | 2024-05-28 | 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 |
| EP3732185B1 (en) | 2017-12-29 | 2025-02-26 | Suzhou Ribo Life Science Co., Ltd. | Conjugates and preparation and use thereof |
| WO2019140102A1 (en) | 2018-01-10 | 2019-07-18 | Translate Bio Ma, Inc. | Compositions and methods for facilitating delivery of synthetic nucleic acids to cells |
| CN111511915A (zh) * | 2018-03-09 | 2020-08-07 | 第一三共株式会社 | 糖原病Ia型治疗药 |
| WO2019193189A1 (en) * | 2018-04-05 | 2019-10-10 | Silence Therapeutics Gmbh | siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS |
| WO2019217459A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| US11918600B2 (en) | 2018-08-21 | 2024-03-05 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof |
| EP3862024A4 (en) * | 2018-09-30 | 2022-08-17 | Suzhou Ribo Life Science Co., Ltd. | SIRNA CONJUGATE, METHOD FOR ITS PRODUCTION AND ITS USE |
| GB2579253A (en) * | 2018-11-28 | 2020-06-17 | Pedanius Therapeutics Ltd | Antibacterial antisense agents |
| CA3121449A1 (en) | 2018-11-30 | 2020-06-04 | Kyowa Kirin Co., Ltd. | Nucleic acid conjugate |
| US12496347B2 (en) | 2018-12-28 | 2025-12-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof |
| GB201821269D0 (en) | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| CN111377985B (zh) * | 2018-12-29 | 2023-11-10 | 苏州瑞博生物技术股份有限公司 | 化合物和缀合物及其制备方法和用途 |
| CN111377984B (zh) * | 2018-12-29 | 2024-03-05 | 苏州瑞博生物技术股份有限公司 | 化合物和缀合物及其制备方法和用途 |
| CN113330117B (zh) * | 2019-01-18 | 2024-05-28 | 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 |
| AU2020280438B2 (en) * | 2019-05-22 | 2025-03-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| 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 |
| 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 |
| WO2020233651A1 (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 |
| CN111973619B (zh) * | 2019-05-23 | 2024-01-30 | 苏州瑞博生物技术股份有限公司 | 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途 |
| CN111973618B (zh) * | 2019-05-23 | 2024-02-02 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与siRNA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 |
| CA3140233A1 (en) * | 2019-05-24 | 2020-12-03 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition and conjugate, preparation method and use |
| US20220315929A1 (en) | 2019-05-24 | 2022-10-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition and conjugate, preparation method therefor and use thereof |
| CN112007040B (zh) * | 2019-05-31 | 2024-01-30 | 苏州瑞博生物技术股份有限公司 | 用于治疗乙型病毒性肝炎的联合用药物 |
| TW202122093A (zh) | 2019-08-29 | 2021-06-16 | 大陸商蘇州瑞博生物技術股份有限公司 | 化合物、藥物綴合物、試劑盒及其用途 |
| WO2021049504A1 (ja) | 2019-09-10 | 2021-03-18 | 第一三共株式会社 | 肝臓送達用GalNAc-オリゴヌクレオチドコンジュゲートおよび製造方法 |
| CN115003330A (zh) | 2019-09-23 | 2022-09-02 | 科纳克斯资本 | 作为疫苗和诊断工具的新糖缀合物 |
| CA3160329A1 (en) * | 2019-11-06 | 2021-05-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| IL293341B1 (en) * | 2019-11-26 | 2026-04-01 | Dtx Pharma Inc | A compound comprising a nucleic acid and a half-life extension motif |
| 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 |
| US20230312635A1 (en) * | 2020-01-29 | 2023-10-05 | Sumitomo Chemical Company, Limited | Method for producing nucleic acid oligomer |
| CN111269279B (zh) * | 2020-02-24 | 2022-08-05 | 陕西师范大学 | 一种在温和条件下制备卤代糖的方法 |
| MX2022011805A (es) | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| AU2021244559A1 (en) | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| CN113683651A (zh) * | 2020-05-19 | 2021-11-23 | 上海京新生物医药有限公司 | 一种GalNAc中间体的制备方法 |
| JP2023526533A (ja) | 2020-05-22 | 2023-06-21 | ウェイブ ライフ サイエンシズ リミテッド | 二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 |
| WO2021249352A1 (zh) | 2020-06-10 | 2021-12-16 | 正大天晴药业集团股份有限公司 | 双链siRNA类似物的缀合物 |
| IL303886A (en) | 2020-12-23 | 2023-08-01 | Flagship Pioneering Inc | Compositions of modified trems and uses thereof |
| EP4273245A4 (en) | 2020-12-29 | 2024-12-11 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND SIRNA CONJUGATE WITH THE NUCLEIC ACID, MANUFACTURING PROCESS THEREOF AND USE THEREOF |
| WO2022189861A1 (en) | 2021-03-08 | 2022-09-15 | Tollys | Carbohydrate conjugates of tlr3 ligands and uses thereof |
| EP4329884A1 (en) | 2021-04-27 | 2024-03-06 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| CN117881786A (zh) | 2021-04-27 | 2024-04-12 | 世代生物公司 | 表达治疗性抗体的非病毒dna载体及其用途 |
| US20240335551A1 (en) * | 2021-05-20 | 2024-10-10 | Olix Us, Inc. | Functional moieties and their uses and synthetic preparation |
| CN115572241B (zh) * | 2021-06-21 | 2024-08-30 | 润佳(苏州)医药科技有限公司 | 双价化合物、偶联物及其用途 |
| CN118475355A (zh) | 2021-07-08 | 2024-08-09 | 日本新药株式会社 | 肾毒性减轻剂 |
| US20240285770A1 (en) | 2021-07-08 | 2024-08-29 | Nippon Shinyaku Co., Ltd. | Precipitation suppressing agent |
| US20240285547A1 (en) | 2021-07-08 | 2024-08-29 | Nippon Shinyaku Co., Ltd. | Nephrotoxicity reducing agent |
| CN117580953B (zh) | 2021-07-16 | 2025-07-18 | 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 |
| US20240309378A1 (en) | 2021-07-16 | 2024-09-19 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate containing double-stranded oligonucleotide, and preparation methods and uses |
| AU2022324471A1 (en) | 2021-08-05 | 2024-02-15 | Sanegene Bio Usa Inc. | 1'-alkyl modified ribose derivatives and methods of use |
| EP4405368A1 (en) | 2021-09-22 | 2024-07-31 | Sanegene Bio USA Inc. | 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides |
| CN118076621A (zh) | 2021-10-05 | 2024-05-24 | 美国圣因生物股份有限公司 | 多羟基化的环戊烷衍生物及使用方法 |
| WO2023097251A1 (en) * | 2021-11-23 | 2023-06-01 | Yale University | Oligonucleotide-containing transcription factor targeting chimeras |
| IL314850A (en) | 2022-02-22 | 2024-10-01 | Sanegene Bio Usa Inc | Ribonucleotide derivatives substituted at the 5' position in the carboxyl group and methods of use |
| CN120129745A (zh) | 2022-03-10 | 2025-06-10 | 日本新药株式会社 | 抗病毒反义低聚物 |
| WO2023177655A1 (en) | 2022-03-14 | 2023-09-21 | Generation Bio Co. | Heterologous prime boost vaccine compositions and methods of use |
| KR20240161967A (ko) | 2022-03-16 | 2024-11-13 | 다이이찌 산쿄 가부시키가이샤 | 트랜스페린 수용체 2 의 발현을 억제하는 siRNA |
| JPWO2023176863A1 (https=) | 2022-03-16 | 2023-09-21 | ||
| WO2023207615A1 (zh) * | 2022-04-26 | 2023-11-02 | 南京明德新药研发有限公司 | 一类含由天然核苷酸组成突出端的双链RNAi化合物 |
| WO2023212134A2 (en) * | 2022-04-28 | 2023-11-02 | Dicerna Pharmaceuticals, Inc. | Methods for synthesis of peracetylgalactosamine-1-pentanoic acid |
| EP4522742A2 (en) | 2022-05-13 | 2025-03-19 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
| US20250179492A1 (en) | 2022-06-22 | 2025-06-05 | Flagship Pioneering Innovations Vi, Llc | Compositions of modified trems and uses thereof |
| WO2024001172A1 (en) * | 2022-06-27 | 2024-01-04 | Ractigen Therapeutics | Oligonucleotide modulators activating complement factor h expression |
| WO2024006999A2 (en) | 2022-06-30 | 2024-01-04 | Alnylam Pharmaceuticals, Inc. | Cyclic-disulfide modified phosphate based oligonucleotide prodrugs |
| IL317824A (en) | 2022-07-11 | 2025-02-01 | Sanegene Bio Usa Inc | Ribose derivatives modified at the '2' position and methods of use |
| KR20250046303A (ko) | 2022-08-05 | 2025-04-02 | 사네진 바이오 유에스에이 인크. | 안지오텐시노겐 (agt)을 표적화하는 이중 가닥 rna 및 그의 사용 방법 |
| WO2024040222A1 (en) | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Cleavable closed-ended dna (cedna) and methods of use thereof |
| CN120265773A (zh) | 2022-10-14 | 2025-07-04 | 美国圣因生物股份有限公司 | 靶向c3的小干扰rna及其用途 |
| CN120225676A (zh) | 2022-12-02 | 2025-06-27 | 上海舶望制药有限公司 | 双环脱碱基核酸类似物以及由它们制备的寡聚化合物 |
| EP4638743A2 (en) | 2022-12-19 | 2025-10-29 | Sanegene Bio USA Inc. | Small interfering rna targeting cfb and uses thereof |
| CN116178458A (zh) * | 2022-12-26 | 2023-05-30 | 成都药明康德新药开发有限公司 | 一种糖苷化合物的合成 |
| WO2024160204A1 (en) * | 2023-01-30 | 2024-08-08 | Primelink Biotherapeutics (Suzhou) Co., Ltd. | Polymer-oligonucleotide conjugates |
| US20240309383A1 (en) | 2023-02-24 | 2024-09-19 | Suzhou Sanegene Bio Inc. | Small interfering rna targeting hbv and uses thereof |
| WO2024186673A2 (en) | 2023-03-03 | 2024-09-12 | Sanegene Bio Usa Inc. | Small interfering rna targeting apoc3 and uses thereof |
| WO2024188174A1 (zh) | 2023-03-10 | 2024-09-19 | 苏州瑞博生物技术股份有限公司 | 一种药物组合物及其用途 |
| WO2024238385A2 (en) | 2023-05-12 | 2024-11-21 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
| EP4706646A1 (en) | 2023-06-01 | 2026-03-11 | Sunshine Lake Pharma Co., Ltd. | Novel double-stranded sirna, conjugate thereof, and use thereof |
| CN121866332A (zh) | 2023-06-21 | 2026-04-14 | 美国圣因生物股份有限公司 | 靶向前蛋白转化酶枯草杆菌蛋白酶kexin 9(pcsk9)的双链rna及其使用方法 |
| CN119219716A (zh) * | 2023-06-30 | 2024-12-31 | 上海维申医药有限公司 | 新型GalNAc的靶向递送片段及其制备和应用 |
| WO2025016441A1 (zh) * | 2023-07-19 | 2025-01-23 | 上海维申医药有限公司 | 新型GalNAc的靶向递送片段及其制备和应用 |
| US20250064939A1 (en) | 2023-07-24 | 2025-02-27 | Sanegene Bio Usa Inc. | Lipid-based enhancement agent for rna delivery and therapy |
| AU2024321710A1 (en) | 2023-08-04 | 2026-03-05 | Chia Tai Tianqing Pharmaceutical Group Co., Ltd. | Double-stranded ribonucleic acid targeting pd-l1 |
| AU2024321098A1 (en) | 2023-08-09 | 2026-03-19 | Beijing Foyou Pharma Co., Ltd | Sirna for inhibiting dgat2 gene expression, sirna conjugate or prodrug and pharmaceutical composition thereof, and use thereof |
| WO2025042786A1 (en) | 2023-08-18 | 2025-02-27 | Flagship Pioneering Innovations Vi, Llc | Compositions comprising circular polyribonucleotides and uses thereof |
| CN121729496A (zh) | 2023-08-31 | 2026-03-24 | 正大天晴药业集团股份有限公司 | 靶向凝血因子xi的双链核糖核酸 |
| CN117384226B (zh) * | 2023-09-14 | 2025-12-26 | 苏州盛诺维生物科技有限公司 | 一种GalNAc类化合物的合成工艺 |
| CN121909203A (zh) | 2023-10-31 | 2026-04-21 | 科罗生物公司 | 包含氨基磷酸酯核苷酸间键的寡核苷酸 |
| US20250161347A1 (en) | 2023-11-22 | 2025-05-22 | Flagship Pioneering Innovations Vii, Llc | Methods and compositions for treating non-alcoholic fatty liver disease |
| WO2025140299A1 (zh) * | 2023-12-26 | 2025-07-03 | 广东东阳光药业股份有限公司 | 一种新型的化合物及其用途 |
| TW202546227A (zh) | 2024-01-18 | 2025-12-01 | 荷蘭商Proqr治療上市公司Ii | 用於治療賀勒氏症候群(hurler syndrome)的反義寡核苷酸 |
| TW202602932A (zh) | 2024-03-28 | 2026-01-16 | 美商萊西亞醫療公司 | 用於降解甲狀腺刺激荷爾蒙受體自體抗體之胞溶體靶向雙功能分子 |
| WO2025228441A2 (en) | 2024-04-30 | 2025-11-06 | Sanegene Bio Usa Inc. | Small interfering rna targeting inhbe and uses thereof |
| CN118146284B (zh) * | 2024-05-08 | 2024-07-26 | 北京悦康科创医药科技股份有限公司 | 一种GalNAc化合物、其与寡核苷酸缀合物及制备方法 |
| WO2025245188A2 (en) | 2024-05-21 | 2025-11-27 | Flagship Pioneering Innovations Vii, Llc | Methods of treating liver steatosis and non-alcoholic fatty liver disease |
| WO2026017140A2 (en) | 2024-07-18 | 2026-01-22 | Sanegene Bio Usa Inc. | Small interfering rna targeting fxi and uses thereof |
| WO2026041067A2 (en) | 2024-08-20 | 2026-02-26 | Sanegene Bio Usa Inc. | Small interfering rna targeting myostatin (mstn) and uses thereof |
| WO2026046411A1 (en) | 2024-09-02 | 2026-03-05 | Sanegene Bio Usa Inc. | Small interfering rna targeting plasminogen (plg) and uses thereof |
| CN121081660A (zh) * | 2025-02-21 | 2025-12-09 | 上海辉棵生物医药有限公司 | 一种靶向递送系统及其核酸缀合物的制备方法与用途 |
Family Cites Families (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5453566A (en) | 1986-03-28 | 1995-09-26 | Calgene, Inc. | Antisense regulation of gene expression in plant/cells |
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
| CA1340323C (en) | 1988-09-20 | 1999-01-19 | Arnold E. Hampel | Rna catalyst for cleaving specific rna sequences |
| GB8822492D0 (en) | 1988-09-24 | 1988-10-26 | Considine J | Apparatus for removing tumours from hollow organs of body |
| AU637800B2 (en) | 1989-08-31 | 1993-06-10 | City Of Hope | Chimeric dna-rna catalytic sequences |
| US6365730B1 (en) | 1990-06-19 | 2002-04-02 | Gene Shears Pty. Limited | DNA-Armed ribozymes and minizymes |
| US5789573A (en) | 1990-08-14 | 1998-08-04 | Isis Pharmaceuticals, Inc. | Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2 |
| JP3257675B2 (ja) | 1990-10-12 | 2002-02-18 | マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. | 修飾リボザイム |
| DE4216134A1 (de) | 1991-06-20 | 1992-12-24 | Europ Lab Molekularbiolog | Synthetische katalytische oligonukleotidstrukturen |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| EP0642589A4 (en) | 1992-05-11 | 1997-05-21 | Ribozyme Pharm Inc | METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION. |
| WO1994002595A1 (en) | 1992-07-17 | 1994-02-03 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for treatment of animal diseases |
| US5574142A (en) * | 1992-12-15 | 1996-11-12 | Microprobe Corporation | Peptide linkers for improved oligonucleotide delivery |
| IL108367A0 (en) | 1993-01-27 | 1994-04-12 | Hektoen Inst For Medical Resea | Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells |
| US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| US5591317A (en) | 1994-02-16 | 1997-01-07 | Pitts, Jr.; M. Michael | Electrostatic device for water treatment |
| US5631359A (en) | 1994-10-11 | 1997-05-20 | Ribozyme Pharmaceuticals, Inc. | Hairpin ribozymes |
| AU3889595A (en) | 1994-10-05 | 1996-05-02 | Amgen, Inc. | Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein |
| US5783683A (en) | 1995-01-10 | 1998-07-21 | Genta Inc. | Antisense oligonucleotides which reduce expression of the FGFRI gene |
| US5681824A (en) | 1995-06-05 | 1997-10-28 | Eisai Co., Ltd. | Substituted liposaccharides useful in the treatment and prevention of endotoxemia |
| US5747470A (en) | 1995-06-07 | 1998-05-05 | Gen-Probe Incorporated | Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA |
| US5739119A (en) | 1996-11-15 | 1998-04-14 | Galli; Rachel L. | Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA |
| US6300319B1 (en) * | 1998-06-16 | 2001-10-09 | Isis Pharmaceuticals, Inc. | Targeted oligonucleotide conjugates |
| WO2004080406A2 (en) | 2003-03-07 | 2004-09-23 | Alnylam Pharmaceuticals | Therapeutic compositions |
| DK1620544T3 (en) | 2003-04-17 | 2019-01-14 | Alnylam Pharmaceuticals Inc | MODIFIED iRNA AGENTS |
| US20070213292A1 (en) | 2005-08-10 | 2007-09-13 | The Rockefeller University | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| WO2007021896A2 (en) | 2005-08-10 | 2007-02-22 | Alnylam Pharmaceuticals, Inc. | Chemically modified oligonucleotides for use in modulating micro rna and uses thereof |
| EP1989307B1 (en) | 2006-02-08 | 2012-08-08 | Quark Pharmaceuticals, Inc. | NOVEL TANDEM siRNAS |
| WO2009082607A2 (en) * | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| WO2009126933A2 (en) * | 2008-04-11 | 2009-10-15 | Alnylam Pharmaceuticals, Inc. | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
| US9198972B2 (en) * | 2010-01-28 | 2015-12-01 | Alnylam Pharmaceuticals, Inc. | Monomers and oligonucleotides comprising cycloaddition adduct(s) |
| US8501930B2 (en) * | 2010-12-17 | 2013-08-06 | Arrowhead Madison Inc. | Peptide-based in vivo siRNA delivery system |
| EP2594575A1 (en) * | 2011-11-15 | 2013-05-22 | Université de Strasbourg | Mannosylated compounds useful for the prevention and the treatment of infectious diseases |
| HUE048622T2 (hu) * | 2011-11-18 | 2020-08-28 | Alnylam Pharmaceuticals Inc | RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére |
| US8577565B2 (en) | 2011-12-16 | 2013-11-05 | GM Global Technology Operations LLC | Limiting branch pressure to a solenoid valve in a fluid circuit |
| AR090906A1 (es) * | 2012-05-02 | 2014-12-17 | Merck Sharp & Dohme | Conjugados que contienen tetragalnac y procedimientos para la administracion de oligonucleotidos |
| SE536942C2 (sv) * | 2012-07-24 | 2014-11-11 | Reiz Ismet | Flexibel dragnyckel |
| AU2013299717B2 (en) * | 2012-08-06 | 2018-06-28 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
| US10125369B2 (en) * | 2012-12-05 | 2018-11-13 | Alnylam Pharmaceuticals, Inc. | PCSK9 iRNA compositions and methods of use thereof |
| DK2991656T3 (da) * | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| EP3564374A1 (en) * | 2013-06-21 | 2019-11-06 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulation of target nucleic acids |
| WO2015188194A1 (en) * | 2014-06-06 | 2015-12-10 | Isis Pharmaceuticals, Inc. | Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds |
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| JP2023073507A (ja) | 2023-05-25 |
| EP3019200A2 (en) | 2016-05-18 |
| WO2015006740A3 (en) | 2015-05-07 |
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