MX2018009854A - Ligandos dirigidos para compuestos terapeuticos. - Google Patents
Ligandos dirigidos para compuestos terapeuticos.Info
- Publication number
- MX2018009854A MX2018009854A MX2018009854A MX2018009854A MX2018009854A MX 2018009854 A MX2018009854 A MX 2018009854A MX 2018009854 A MX2018009854 A MX 2018009854A MX 2018009854 A MX2018009854 A MX 2018009854A MX 2018009854 A MX2018009854 A MX 2018009854A
- Authority
- MX
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- Prior art keywords
- targeting ligands
- expression
- compounds
- target
- inhibiting oligomeric
- Prior art date
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- C—CHEMISTRY; METALLURGY
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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
- 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
- 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
- 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
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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
- 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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- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/31—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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- C—CHEMISTRY; METALLURGY
- 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
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- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- 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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- 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
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- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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
- C12N2310/3521—Methyl
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- C—CHEMISTRY; METALLURGY
- 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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- 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
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- 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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (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)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
La presente invención se relaciona con nuevos ligandos de dirección que pueden enlazarse a compuestos, tales como compuestos terapéuticos que son útiles en la dirección de compuestos al blanco in vivo. Los ligandos de dirección descritos en la presente pueden servir para los compuestos oligoméricos de inhibición de la expresión blanco, tales como agente de ARNi, a células hepáticas para modular la expresión génica. Los ligandos de dirección descritos en la presente, cuando se conjugan a un oligomérico que inhibe la expresión, pueden usarse en una variedad de aplicaciones, que incluyen el uso en la validación terapéutica, diagnóstico, blanco y aplicaciones de descubrimiento genómico. Las composiciones, que incluyen los ligandos de dirección descritos en la presente, cuando se enlazan a compuestos oligoméricos que inhiben la expresión son capaces de mediar la expresión de secuencias de ácidos nucleicos blanco en las células hepáticas, tales como hepatocitos, que podrían ser útiles en el tratamiento de enfermedades o afecciones que responden a la inhibición de la expresión o actividad génica en una célula, tejido u organismo.
Applications Claiming Priority (4)
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 | |
PCT/US2017/021175 WO2017156012A1 (en) | 2016-03-07 | 2017-03-07 | Targeting ligands for therapeutic compounds |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2018009854A true MX2018009854A (es) | 2018-11-09 |
Family
ID=59723428
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2018009854A MX2018009854A (es) | 2016-03-07 | 2017-03-07 | Ligandos dirigidos para compuestos terapeuticos. |
MX2023006334A MX2023006334A (es) | 2016-03-07 | 2018-08-14 | Ligandos dirigidos para compuestos terapeuticos. |
MX2023006332A MX2023006332A (es) | 2016-03-07 | 2018-08-14 | Ligandos dirigidos para compuestos terapeuticos. |
Family Applications After (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2023006334A MX2023006334A (es) | 2016-03-07 | 2018-08-14 | Ligandos dirigidos para compuestos terapeuticos. |
MX2023006332A MX2023006332A (es) | 2016-03-07 | 2018-08-14 | Ligandos dirigidos para compuestos terapeuticos. |
Country Status (14)
Country | Link |
---|---|
US (3) | US10246709B2 (es) |
EP (1) | EP3426261A4 (es) |
JP (2) | JP6983797B2 (es) |
KR (3) | KR102515329B1 (es) |
CN (2) | CN113797348A (es) |
AU (2) | AU2017229469B2 (es) |
BR (1) | BR112018068230B1 (es) |
CA (1) | CA3011946A1 (es) |
IL (3) | IL296377A (es) |
JO (2) | JOP20170057B1 (es) |
MX (3) | MX2018009854A (es) |
TW (2) | TWI783434B (es) |
UY (1) | UY37146A (es) |
WO (1) | WO2017156012A1 (es) |
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JOP20210043A1 (ar) | 2015-10-01 | 2017-06-16 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
UY37146A (es) * | 2016-03-07 | 2017-09-29 | Arrowhead Pharmaceuticals Inc | Ligandos de direccionamiento para compuestos terapéuticos |
JOP20170161A1 (ar) | 2016-08-04 | 2019-01-30 | Arrowhead Pharmaceuticals Inc | عوامل RNAi للعدوى بفيروس التهاب الكبد ب |
KR102557906B1 (ko) * | 2016-09-02 | 2023-07-20 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
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 |
CN111107853A (zh) | 2017-09-11 | 2020-05-05 | 箭头药业股份有限公司 | 用于抑制载脂蛋白C-III (APOC3)的表达的RNAi试剂和组合物 |
TN2020000038A1 (en) | 2017-09-14 | 2021-10-04 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of angiopoietin-like 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 |
AU2019342117A1 (en) * | 2018-09-19 | 2021-03-04 | 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 |
US20220305046A1 (en) | 2019-06-06 | 2022-09-29 | 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 |
AU2021244329A1 (en) | 2020-03-23 | 2022-09-29 | Amgen Inc. | Monoclonal antibodies to chemically-modified nucleic acids and uses thereof |
JP2023519246A (ja) | 2020-03-26 | 2023-05-10 | 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 | PNPLA3の発現を阻害するためのRNAi薬、その医薬組成物、及び使用方法 |
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WO2021206182A1 (ja) * | 2020-04-10 | 2021-10-14 | 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 |
WO2021254360A1 (zh) * | 2020-06-16 | 2021-12-23 | 上海拓界生物医药科技有限公司 | 一种碳水化合物分子簇及其制备方法和医药用途 |
JP2023541170A (ja) | 2020-09-15 | 2023-09-28 | 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 | Z-aatタンパク質のレベルを低下させる方法 |
MX2023005198A (es) | 2020-11-05 | 2023-05-16 | Amgen Inc | Metodos para tratar la enfermedad cardiovascular aterosclerotica con constructos de iarn dirigidos a lpa. |
US20240175020A1 (en) | 2020-12-23 | 2024-05-30 | Flagship Pioneering Innovations Vi, Llc | Compositions of modified trems and uses thereof |
KR20240028335A (ko) | 2021-04-23 | 2024-03-05 | 간엔에이 바이오, 인크. | 글리칸 변형 핵산, 제조 방법 및 치료 용도 |
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KR20240028502A (ko) * | 2021-07-02 | 2024-03-05 | 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 | 핵산 리간드 및 이의 접합체, 및 이를 위한 제조 방법 및 이의 용도 |
CN114703184A (zh) * | 2022-03-11 | 2022-07-05 | 厦门甘宝利生物医药有限公司 | Lpa抑制剂及其用途 |
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TW202406926A (zh) * | 2022-05-02 | 2024-02-16 | 美商安進公司 | 用於製備碳水化合物靶向部分及其中間體之方法 |
WO2023233290A1 (en) | 2022-05-31 | 2023-12-07 | Janssen Sciences Ireland Unlimited Company | Rnai agents targeting pd-l1 |
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