JOP20170057B1 - مركبات ترابطية مستهدفة كمركبات علاجية - Google Patents
مركبات ترابطية مستهدفة كمركبات علاجيةInfo
- Publication number
- JOP20170057B1 JOP20170057B1 JOP/2017/0057A JOP20170057A JOP20170057B1 JO P20170057 B1 JOP20170057 B1 JO P20170057B1 JO P20170057 A JOP20170057 A JO P20170057A JO P20170057 B1 JOP20170057 B1 JO P20170057B1
- Authority
- JO
- Jordan
- Prior art keywords
- targeting ligands
- expression
- compounds
- target
- inhibiting oligomeric
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 7
- 239000003446 ligand Substances 0.000 title abstract 5
- 230000008685 targeting Effects 0.000 title abstract 5
- 230000001225 therapeutic effect Effects 0.000 title abstract 3
- 230000014509 gene expression Effects 0.000 abstract 6
- 230000002401 inhibitory effect Effects 0.000 abstract 3
- 210000005229 liver cell Anatomy 0.000 abstract 2
- 108091030071 RNAI Proteins 0.000 abstract 1
- -1 RNAi agents Chemical class 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000009368 gene silencing by RNA Effects 0.000 abstract 1
- 210000003494 hepatocyte Anatomy 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 230000005764 inhibitory process Effects 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 1
- 238000010200 validation analysis Methods 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
- 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
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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
-
- 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
- C12N15/113—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
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
-
- 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
-
- 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
- C12N2310/32—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
-
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Crystallography & Structural Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (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)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662304652P | 2016-03-07 | 2016-03-07 | |
| US201662370754P | 2016-08-04 | 2016-08-04 | |
| US201662426916P | 2016-11-28 | 2016-11-28 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| JOP20170057B1 true JOP20170057B1 (ar) | 2022-03-14 |
Family
ID=59723428
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JOP/2017/0057A JOP20170057B1 (ar) | 2016-03-07 | 2017-03-07 | مركبات ترابطية مستهدفة كمركبات علاجية |
| JOP/2021/0231A JOP20210231A1 (ar) | 2016-03-07 | 2021-08-19 | مركبات ترابطية مستهدفة كمركبات علاجية |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JOP/2021/0231A JOP20210231A1 (ar) | 2016-03-07 | 2021-08-19 | مركبات ترابطية مستهدفة كمركبات علاجية |
Country Status (14)
| Country | Link |
|---|---|
| US (4) | US10246709B2 (enExample) |
| EP (1) | EP3426261A4 (enExample) |
| JP (4) | JP6983797B2 (enExample) |
| KR (3) | KR102348425B1 (enExample) |
| CN (2) | CN109069529B (enExample) |
| AU (3) | AU2017229469B2 (enExample) |
| BR (1) | BR112018068230B1 (enExample) |
| CA (1) | CA3011946A1 (enExample) |
| IL (3) | IL296377A (enExample) |
| JO (2) | JOP20170057B1 (enExample) |
| MX (3) | MX2018009854A (enExample) |
| TW (3) | TWI783434B (enExample) |
| UY (1) | UY37146A (enExample) |
| WO (1) | WO2017156012A1 (enExample) |
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| JOP20160211B1 (ar) | 2015-10-01 | 2021-08-17 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| JOP20170057B1 (ar) * | 2016-03-07 | 2022-03-14 | Arrowhead Pharmaceuticals Inc | مركبات ترابطية مستهدفة كمركبات علاجية |
| TWI912633B (zh) | 2016-06-06 | 2026-01-21 | 美商愛羅海德製藥公司 | 5'-環-磷酸修飾之核苷酸 |
| JOP20190015A1 (ar) | 2016-08-04 | 2019-02-04 | Arrowhead Pharmaceuticals Inc | عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب |
| JOP20170056B1 (ar) | 2016-09-02 | 2021-08-17 | Arrowhead Pharmaceuticals Inc | مركبات ترابطية مستهدفة |
| US10450565B2 (en) | 2017-01-10 | 2019-10-22 | Arrowhead Pharmaceuticals, Inc. | Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use |
| CN110234326B (zh) | 2017-01-30 | 2024-11-12 | 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 |
| SG11201912178VA (en) | 2017-09-11 | 2020-01-30 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
| MX2020001912A (es) | 2017-09-14 | 2020-03-24 | Arrowhead Pharmaceuticals Inc | Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso. |
| CN111212909A (zh) | 2017-10-17 | 2020-05-29 | 箭头药业股份有限公司 | 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物 |
| US12534728B2 (en) * | 2018-09-19 | 2026-01-27 | 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 |
| AU2020286462B2 (en) | 2019-06-06 | 2026-07-02 | Arrowhead Pharmaceuticals, Inc. | Methods for the treatment of alpha-1 antitrypsin deficiency (AATD) |
| JP2022536945A (ja) | 2019-06-18 | 2022-08-22 | ヤンセン・サイエンシズ・アイルランド・アンリミテッド・カンパニー | Ｂ型肝炎ウイルス（ＨＢＶ）ワクチンおよびＨＢＶを標的化するＲＮＡｉの組合せ |
| CA3143047A1 (en) | 2019-06-25 | 2020-12-30 | Amgen Inc. | Purification methods for carbohydrate-linked oligonucleotides |
| IL290766B2 (en) | 2019-09-03 | 2026-03-01 | Arcturus Therapeutics Inc | Administration of therapeutically active conjugates mediated by asialoglycoprotein receptor |
| CA3174708A1 (en) * | 2020-03-04 | 2021-09-10 | Verve Therapeutics, Inc. | Compositions and methods for targeted rna delivery |
| EP4126967A1 (en) | 2020-03-23 | 2023-02-08 | Amgen Inc. | Monoclonal antibodies to chemically-modified nucleic acids and uses thereof |
| KR20220158011A (ko) | 2020-03-26 | 2022-11-29 | 애로우헤드 파마슈티컬스 인코포레이티드 | PNPLA3의 발현을 억제하기 위한 RNAi 작용제, 이의 약제학적 조성물, 및 사용 방법 |
| US20230250125A1 (en) | 2020-04-01 | 2023-08-10 | Janssen Biopharma, Inc. | Nucleic acid polymers |
| EP4132951A4 (en) | 2020-04-09 | 2025-06-11 | Verve Therapeutics, Inc. | CHEMICALLY MODIFIED GENE EDITING GUIDE RNAS WITH CAS12B |
| JP7875529B2 (ja) * | 2020-04-10 | 2026-06-18 | 学校法人 聖マリアンナ医科大学 | 化合物、造影剤、及び化合物の製造方法 |
| TW202214301A (zh) * | 2020-06-16 | 2022-04-16 | 大陸商上海拓界生物醫藥科技有限公司 | 一種碳水化合物分子簇及其製備方法和醫藥用途 |
| WO2022060721A2 (en) | 2020-09-15 | 2022-03-24 | Arrowhead Pharmaceuticals, Inc. | Methods for the reduction of z-aat protein levels |
| KR20230104200A (ko) | 2020-11-05 | 2023-07-07 | 암젠 인크 | LPA-표적화된 RNAi 작제물을 이용하여 죽상경화성 심혈관질환을 치료하는 방법 |
| KR20230135585A (ko) | 2020-12-23 | 2023-09-25 | 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 | 변형된 trem의 조성물 및 이의 용도 |
| IL307885A (en) | 2021-04-23 | 2023-12-01 | Ganna Bio Inc | Nucleic acids with different glycans, preparation methods and therapeutic uses |
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| TW202317197A (zh) * | 2021-07-02 | 2023-05-01 | 大陸商上海拓界生物醫藥科技有限公司 | 一種核酸配體及其綴合物、其製備方法和用途 |
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| WO2025021831A1 (en) | 2023-07-24 | 2025-01-30 | Astrazeneca Ab | Multivalent cargo-carrying complexes and uses thereof |
| GB202311324D0 (en) | 2023-07-24 | 2023-09-06 | Astrazeneca Ab | Multivalent cargo-carrying complexes and uses thereof |
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