CN109069529B - 用于治疗性化合物的靶向配体 - Google Patents
用于治疗性化合物的靶向配体 Download PDFInfo
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- CN109069529B CN109069529B CN201780016137.8A CN201780016137A CN109069529B CN 109069529 B CN109069529 B CN 109069529B CN 201780016137 A CN201780016137 A CN 201780016137A CN 109069529 B CN109069529 B CN 109069529B
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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
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- 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
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- 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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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C07H15/02—Acyclic radicals, not substituted by cyclic structures
- C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- 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
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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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- 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
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- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
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- General Engineering & Computer Science (AREA)
- Plant Pathology (AREA)
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- Biophysics (AREA)
- Crystallography & Structural Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Peptides Or Proteins (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202110938247.8A CN113797348A (zh) | 2016-03-07 | 2017-03-07 | 用于治疗性化合物的靶向配体 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662304652P | 2016-03-07 | 2016-03-07 | |
| US62/304,652 | 2016-03-07 | ||
| US201662370754P | 2016-08-04 | 2016-08-04 | |
| US62/370,754 | 2016-08-04 | ||
| US201662426916P | 2016-11-28 | 2016-11-28 | |
| US62/426,916 | 2016-11-28 | ||
| PCT/US2017/021175 WO2017156012A1 (en) | 2016-03-07 | 2017-03-07 | Targeting ligands for therapeutic compounds |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202110938247.8A Division CN113797348A (zh) | 2016-03-07 | 2017-03-07 | 用于治疗性化合物的靶向配体 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN109069529A CN109069529A (zh) | 2018-12-21 |
| CN109069529B true CN109069529B (zh) | 2021-08-20 |
Family
ID=59723428
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202110938247.8A Pending CN113797348A (zh) | 2016-03-07 | 2017-03-07 | 用于治疗性化合物的靶向配体 |
| CN201780016137.8A Active CN109069529B (zh) | 2016-03-07 | 2017-03-07 | 用于治疗性化合物的靶向配体 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202110938247.8A Pending CN113797348A (zh) | 2016-03-07 | 2017-03-07 | 用于治疗性化合物的靶向配体 |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US10246709B2 (OSRAM) |
| EP (1) | EP3426261A4 (OSRAM) |
| JP (4) | JP6983797B2 (OSRAM) |
| KR (3) | KR102515329B1 (OSRAM) |
| CN (2) | CN113797348A (OSRAM) |
| AU (3) | AU2017229469B2 (OSRAM) |
| BR (1) | BR112018068230B1 (OSRAM) |
| CA (1) | CA3011946A1 (OSRAM) |
| IL (3) | IL296377A (OSRAM) |
| JO (2) | JOP20170057B1 (OSRAM) |
| MX (3) | MX2018009854A (OSRAM) |
| NZ (2) | NZ744357A (OSRAM) |
| TW (3) | TWI783434B (OSRAM) |
| UY (1) | UY37146A (OSRAM) |
| WO (1) | WO2017156012A1 (OSRAM) |
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| TW201620526A (zh) | 2014-06-17 | 2016-06-16 | 愛羅海德研究公司 | 用於抑制α-1抗胰蛋白酶基因表現之組合物及方法 |
| JOP20210043A1 (ar) | 2015-10-01 | 2017-06-16 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| NZ744357A (en) | 2016-03-07 | 2025-09-26 | Arrowhead Pharmaceuticals Inc | Targeting ligands for therapeutic compounds |
| TW202423483A (zh) | 2016-06-06 | 2024-06-16 | 美商愛羅海德製藥公司 | 5’-環-磷酸修飾之核苷酸 |
| JOP20170161A1 (ar) | 2016-08-04 | 2019-01-30 | Arrowhead Pharmaceuticals Inc | عوامل RNAi للعدوى بفيروس التهاب الكبد ب |
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| TWI826365B (zh) | 2017-01-10 | 2023-12-21 | 美商愛羅海德製藥公司 | α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法 |
| CN119548520A (zh) | 2017-01-30 | 2025-03-04 | 箭头药业股份有限公司 | 用于抑制因子xii基因表达的组合物和方法 |
| US10597657B2 (en) | 2017-09-11 | 2020-03-24 | Arrowhead Pharmaceuticals, Inc. | RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3) |
| WO2019055633A1 (en) | 2017-09-14 | 2019-03-21 | Arrowhead Pharmaceuticals, Inc. | RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE |
| EP3697909A4 (en) | 2017-10-17 | 2021-10-13 | Arrowhead Pharmaceuticals, Inc. | RNAI-BASED AGENTS AND COMPOSITIONS INTENDED TO INHIBIT ASIALOGLYCOPROTEIN RECEPTOR 1 EXPRESSION |
| WO2020061177A1 (en) * | 2018-09-19 | 2020-03-26 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use |
| 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 |
| JP7697889B2 (ja) | 2019-06-06 | 2025-06-24 | アローヘッド ファーマシューティカルズ インコーポレイテッド | アルファ-1アンチトリプシン欠乏症(aatd)の治療法 |
| AU2020295796A1 (en) | 2019-06-18 | 2022-02-10 | Janssen Sciences Ireland Unlimited Company | Combination of hepatitis B virus (HBV) vaccines and HBV-targeting rnai |
| US20220363711A1 (en) | 2019-06-25 | 2022-11-17 | Amgen Inc. | Purification methods for carbohydrate-linked oligonucleotides |
| KR20220058578A (ko) | 2019-09-03 | 2022-05-09 | 아크투루스 쎄라퓨틱스, 인크. | 치료 활성 접합체의 아시알로당단백질 수용체 매개 전달 |
| IL296133A (en) * | 2020-03-04 | 2022-11-01 | Verve Therapeutics Inc | Preparations and methods for administering targeted RNA |
| EP4126967A1 (en) * | 2020-03-23 | 2023-02-08 | Amgen Inc. | Monoclonal antibodies to chemically-modified nucleic acids and uses thereof |
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Citations (5)
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| US5885968A (en) * | 1992-08-11 | 1999-03-23 | Rijksuniversiteit Te Leiden | Triantennary cluster glycosides, their preparation and use |
| CN1661044A (zh) * | 1998-10-30 | 2005-08-31 | 第一制药株式会社 | Dds化合物及其测定方法 |
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