CN112400018A - 肝外递送 - Google Patents
肝外递送 Download PDFInfo
- Publication number
- CN112400018A CN112400018A CN201980045507.XA CN201980045507A CN112400018A CN 112400018 A CN112400018 A CN 112400018A CN 201980045507 A CN201980045507 A CN 201980045507A CN 112400018 A CN112400018 A CN 112400018A
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- CN
- China
- Prior art keywords
- ome
- double
- irna agent
- positions
- stranded irna
- Prior art date
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
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- 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
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- A61K47/55—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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
- A61K47/551—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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
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- 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
- A61K47/554—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 the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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- 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
- A61K47/62—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 a protein, peptide or polyamino acid
- A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
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- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A61P25/16—Anti-Parkinson drugs
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- 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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- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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- C12N2310/315—Phosphorothioates
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/35—Nature of the modification
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- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Psychiatry (AREA)
- Botany (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Oncology (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862668072P | 2018-05-07 | 2018-05-07 | |
| US62/668,072 | 2018-05-07 | ||
| US201862738747P | 2018-09-28 | 2018-09-28 | |
| US62/738,747 | 2018-09-28 | ||
| US201862773082P | 2018-11-29 | 2018-11-29 | |
| US62/773,082 | 2018-11-29 | ||
| PCT/US2019/031170 WO2019217459A1 (en) | 2018-05-07 | 2019-05-07 | Extrahepatic delivery |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112400018A true CN112400018A (zh) | 2021-02-23 |
Family
ID=66669075
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980045507.XA Pending CN112400018A (zh) | 2018-05-07 | 2019-05-07 | 肝外递送 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US12397013B2 (https=) |
| EP (1) | EP3790970A1 (https=) |
| JP (3) | JP7353301B2 (https=) |
| KR (1) | KR20210018267A (https=) |
| CN (1) | CN112400018A (https=) |
| AU (2) | AU2019266207B2 (https=) |
| BR (1) | BR112020022546A8 (https=) |
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| WO2023116607A1 (zh) | 2021-12-21 | 2023-06-29 | 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 |
| WO2024002046A1 (en) * | 2022-06-27 | 2024-01-04 | Ractigen Therapeutics | Oligonucleotide delivery enhancing compounds, pharmaceutical compositions and methods using the same |
| WO2024109722A1 (zh) * | 2022-11-21 | 2024-05-30 | 广州必贝特医药股份有限公司 | 用于抑制CIDEB基因表达的siRNA、药物及其应用 |
| WO2024255761A1 (zh) * | 2023-06-12 | 2024-12-19 | 大睿生物 | 包含糖的寡核苷酸递送配体 |
| CN117417400A (zh) * | 2023-12-18 | 2024-01-19 | 苏州诺维康生物科技有限公司 | 2'-o-丙炔基-5'-二甲氧三苯甲基-n4-乙酰基-胞苷的合成方法 |
| CN117417400B (zh) * | 2023-12-18 | 2024-02-20 | 苏州诺维康生物科技有限公司 | 2'-o-丙炔基-5'-二甲氧三苯甲基-n4-乙酰基-胞苷的合成方法 |
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| IL278382B1 (en) | 2025-11-01 |
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| BR112020022546A8 (pt) | 2022-05-17 |
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| IL323565A (en) | 2025-11-01 |
| US20240358738A1 (en) | 2024-10-31 |
| WO2019217459A1 (en) | 2019-11-14 |
| US20220125823A1 (en) | 2022-04-28 |
| JP2023164585A (ja) | 2023-11-10 |
| SG11202010910QA (en) | 2020-12-30 |
| JP2025071263A (ja) | 2025-05-02 |
| EP3790970A1 (en) | 2021-03-17 |
| US20240009225A1 (en) | 2024-01-11 |
| MX2020011570A (es) | 2020-11-24 |
| AU2019266207A1 (en) | 2020-12-17 |
| KR20210018267A (ko) | 2021-02-17 |
| JP7641339B2 (ja) | 2025-03-06 |
| CA3098623A1 (en) | 2019-11-14 |
| IL278382A (en) | 2020-12-31 |
| AU2019266207B2 (en) | 2025-04-24 |
| BR112020022546A2 (pt) | 2021-02-02 |
| JP2021522810A (ja) | 2021-09-02 |
| US12397013B2 (en) | 2025-08-26 |
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