JOP20170056B1 - مركبات ترابطية مستهدفة - Google Patents
مركبات ترابطية مستهدفةInfo
- Publication number
- JOP20170056B1 JOP20170056B1 JOP/2017/0056A JOP20170056A JOP20170056B1 JO P20170056 B1 JOP20170056 B1 JO P20170056B1 JO P20170056 A JOP20170056 A JO P20170056A JO P20170056 B1 JOP20170056 B1 JO P20170056B1
- Authority
- JO
- Jordan
- Prior art keywords
- targeting ligands
- compounds
- expression
- target
- therapeutic
- Prior art date
Links
- 239000003446 ligand Substances 0.000 title abstract 5
- 230000008685 targeting Effects 0.000 title abstract 5
- 150000001875 compounds Chemical class 0.000 abstract 6
- 230000014509 gene expression Effects 0.000 abstract 5
- 230000001225 therapeutic effect Effects 0.000 abstract 3
- 230000002401 inhibitory effect Effects 0.000 abstract 2
- 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
- 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
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- 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
- A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
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- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- 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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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- 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
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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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- 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
- A61K47/555—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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
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- 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
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- 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
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- 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
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- 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
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- 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
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H5/00—Compounds containing saccharide radicals in which the hetero bonds to oxygen have been replaced by the same number of hetero bonds to halogen, nitrogen, sulfur, selenium, or tellurium
- C07H5/04—Compounds containing saccharide radicals in which the hetero bonds to oxygen have been replaced by the same number of hetero bonds to halogen, nitrogen, sulfur, selenium, or tellurium to nitrogen
- C07H5/06—Aminosugars
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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 N.A.
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
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Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (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)
Abstract
يتعلق الاختراع الحالي بالكشف عن مركبات ترابطية مستهدفة جديدة يمكن ربطها بمركبات هذه المركبات العلاجية مفيدة في توجيه المركبات الى هدف داخل الجسم الحي. يمكن أن تعمل المركبات الترابطية المستهدفة التي تم الكشف عنها في هذه الوثيقة على استهداف مركبات أوليجومرية لتثبيط التعبير، مثل عوامل RNAi ، على خلايا الكبد لتضمين التعبير الجيني. يمكن استخدام المركبات الترابطية المستهدفة التي تم الكشف عنها في هذه الوثيقة، عندما ترافقها مع مركب علاجي في مجموعة متنوعة من التطبيقات التي تتضمن الاستخدام في تطبيقات علاجية، تشخيصية، التحقق من الهدف، وتطبيقات اكتشاف مجموعة العوامل الوراثية. تكون التركيبات التي تتضمن المركبات الترابطية المستهدفة التي تم الكشف عنها في هذه الوثيقة عند ربطها بمركبات أوليجومرية لتثبيط التعبير قادرة على التعبير عن متواليات حمض نووي مستهدفة في خلايا الكبد، مثل الخلايا الكبدية، التي يمكن أن تكون مفيدة في معالجة الأمراض او الحاليات التي تستجيب لتثبيط التعبير الجيني أو النشاط في خلية، نسيج أو عضو.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662383221P | 2016-09-02 | 2016-09-02 | |
US201762456339P | 2017-02-08 | 2017-02-08 |
Publications (1)
Publication Number | Publication Date |
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JOP20170056B1 true JOP20170056B1 (ar) | 2021-08-17 |
Family
ID=61281611
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JOP/2017/0056A JOP20170056B1 (ar) | 2016-09-02 | 2017-03-07 | مركبات ترابطية مستهدفة |
JOP/2021/0207A JOP20210207A1 (ar) | 2016-09-02 | 2021-08-02 | مركبات ترابطية مستهدفة |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JOP/2021/0207A JOP20210207A1 (ar) | 2016-09-02 | 2021-08-02 | مركبات ترابطية مستهدفة |
Country Status (15)
Country | Link |
---|---|
US (3) | US10294474B2 (ar) |
EP (1) | EP3506913A4 (ar) |
JP (1) | JP6989521B2 (ar) |
KR (3) | KR102557906B1 (ar) |
CN (3) | CN116832169A (ar) |
AU (2) | AU2017320582B2 (ar) |
BR (1) | BR112019004178A2 (ar) |
CA (1) | CA3011668A1 (ar) |
IL (2) | IL300869A (ar) |
JO (2) | JOP20170056B1 (ar) |
MX (3) | MX2018009853A (ar) |
SG (2) | SG10201912835QA (ar) |
TW (2) | TWI775743B (ar) |
UY (1) | UY37145A (ar) |
WO (1) | WO2018044350A1 (ar) |
Families Citing this family (40)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
MX342731B (es) | 2011-06-30 | 2016-10-11 | Arrowhead Res Corp | Composiciones y metodos para inhibir la expresion genica del virus de hepatitis b. |
KR20180038465A (ko) | 2015-08-07 | 2018-04-16 | 애로우헤드 파마슈티컬스 인코포레이티드 | B형 간염 바이러스 감염에 대한 rnai 치료법 |
JOP20170161A1 (ar) | 2016-08-04 | 2019-01-30 | Arrowhead Pharmaceuticals Inc | عوامل RNAi للعدوى بفيروس التهاب الكبد ب |
AU2017320582B2 (en) * | 2016-09-02 | 2023-11-16 | Arrowhead Pharmaceuticals, Inc | Targeting ligands |
JOP20180003B1 (ar) | 2017-01-10 | 2022-09-15 | Arrowhead Pharmaceuticals Inc | عوامل RNAi ألفا-1 مضادة للتريبسين (AAT)، وتركيبات تتضمن عوامل AAT RNAi، وطرق الاستخدام |
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- 2017-03-07 TW TW111129177A patent/TW202320855A/zh unknown
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- 2017-03-07 KR KR1020237024411A patent/KR20230115344A/ko not_active Application Discontinuation
- 2017-03-07 US US15/452,324 patent/US10294474B2/en active Active
- 2017-03-07 CN CN201780042047.6A patent/CN109462981B/zh active Active
- 2017-03-07 WO PCT/US2017/021147 patent/WO2018044350A1/en active Application Filing
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2018
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2019
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2021
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