CL2020002401A1 - Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas. - Google Patents
Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas.Info
- Publication number
- CL2020002401A1 CL2020002401A1 CL2020002401A CL2020002401A CL2020002401A1 CL 2020002401 A1 CL2020002401 A1 CL 2020002401A1 CL 2020002401 A CL2020002401 A CL 2020002401A CL 2020002401 A CL2020002401 A CL 2020002401A CL 2020002401 A1 CL2020002401 A1 CL 2020002401A1
- Authority
- CL
- Chile
- Prior art keywords
- hsd17b13
- compositions
- methods
- arni
- hydroxysteroid dehydrogenases
- Prior art date
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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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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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
- C12N15/1137—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 against enzymes
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
- C12N2310/3125—Methylphosphonates
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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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- 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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- 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
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
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- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Abstract
Composiciones de ácidos ribonucleicos de hebra doble (ARNhd) dirigidos al gen HSD17B13, así como métodos para inhibir la expresión de HSD17B13, y métodos para tratar a sujetos que pudieran verse beneficiados con una reducción de la expresión de HSD17B13, tales como sujetos que padecen una enfermedad, trastorno o condición asociada con HSD17B13, mediante el uso de dichas composiciones de ARNhd.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862645941P | 2018-03-21 | 2018-03-21 | |
| US201862770298P | 2018-11-21 | 2018-11-21 | |
| US201862775590P | 2018-12-05 | 2018-12-05 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2020002401A1 true CL2020002401A1 (es) | 2021-01-29 |
Family
ID=66001358
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2020002401A CL2020002401A1 (es) | 2018-03-21 | 2020-09-16 | Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas. |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US12359201B2 (es) |
| EP (1) | EP3768838A1 (es) |
| JP (3) | JP7507093B2 (es) |
| KR (2) | KR102873006B1 (es) |
| CN (1) | CN112020556A (es) |
| AU (2) | AU2019239971B2 (es) |
| BR (1) | BR112020018758A2 (es) |
| CA (1) | CA3091146A1 (es) |
| CL (1) | CL2020002401A1 (es) |
| CO (1) | CO2020010342A2 (es) |
| IL (1) | IL277329A (es) |
| MX (2) | MX2020009812A (es) |
| MY (1) | MY207352A (es) |
| PH (1) | PH12020500622A1 (es) |
| SG (1) | SG11202007583SA (es) |
| TW (1) | TWI877108B (es) |
| WO (1) | WO2019183164A1 (es) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
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| JP7458785B2 (ja) | 2017-01-23 | 2024-04-01 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | ヒドロキシステロイド17-βデヒドロゲナーゼ13(HSD17B13)バリアント及びその使用 |
| AU2018250727A1 (en) | 2017-04-11 | 2019-10-31 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (HSD17B) family |
| JP7434151B2 (ja) | 2017-10-11 | 2024-02-20 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Pnpla3 i148mの変異を発現している患者の肝疾患の治療におけるhsd17b13の阻害 |
| EP3728137A4 (en) | 2017-12-22 | 2021-12-08 | North Carolina State University | POLYMERIC FLUOROPHORES, COMPOSITIONS INCLUDING THEM, AND THEIR PREPARATION AND USE PROCESSES |
| SG11202007583SA (en) | 2018-03-21 | 2020-09-29 | Regeneron Pharma | 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| MY209138A (en) * | 2018-09-19 | 2025-06-24 | Arrowhead Pharmaceuticals Inc | Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use |
| US12312584B2 (en) | 2018-10-02 | 2025-05-27 | Exosome Therapeutics, Inc. | cGMP exosome loaded therapeutics for treating sickle cell disease |
| JP7394137B2 (ja) * | 2018-12-21 | 2023-12-07 | アイオニス・ファーマシューティカルズ・インコーポレイテッド | Hsd17b13発現のモジュレータ |
| KR20220112788A (ko) * | 2019-12-06 | 2022-08-11 | 제네반트 사이언시즈 게엠베하 | 간 섬유증 치료용 콘쥬게이트 및 이의 치료 방법 |
| MX2022015169A (es) * | 2020-06-01 | 2023-07-03 | Amgen Inc | Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos. |
| WO2021252649A2 (en) * | 2020-06-09 | 2021-12-16 | Alnylam Pharmaceuticals, Inc. | Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression |
| WO2022098748A1 (en) * | 2020-11-06 | 2022-05-12 | Inipharm, Inc. | Uses for hsd17b13 inhibitors |
| JP2024502282A (ja) | 2020-12-23 | 2024-01-18 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 細胞死誘導dffa様エフェクターb(cideb)阻害剤での肝疾患の治療 |
| US20240279653A1 (en) * | 2021-02-22 | 2024-08-22 | Alnylam Pharmaceuticals, Inc. | FOLLICULIN iRNA COMPOSITIONS AND METHODS THEREOF |
| JP2024515194A (ja) * | 2021-04-22 | 2024-04-05 | 上海拓界生物医薬科技有限公司 | 17β-ヒドロキシステロイドデヒドロゲナーゼの13型を標的とするsiRNA及びsiRNA複合体 |
| IL310864A (en) | 2021-08-20 | 2024-04-01 | Enanta Pharm Inc | 17-beta-hydroxysteroid dehydrogenase type 13 inhibitors and methods of use thereof |
| AU2022343115A1 (en) * | 2021-09-08 | 2024-03-14 | Aligos Therapeutics, Inc. | Modified short interfering nucleic acid (sina) molecules and uses thereof |
| WO2023091644A2 (en) * | 2021-11-19 | 2023-05-25 | Wave Life Sciences Ltd. | Hsd17b13-related double stranded oligonucleotide compositions and methods relating thereto |
| WO2023109935A1 (zh) * | 2021-12-16 | 2023-06-22 | 上海拓界生物医药科技有限公司 | 一种dsRNA、其制备方法及应用 |
| EP4453257A2 (en) | 2021-12-20 | 2024-10-30 | Regeneron Pharmaceuticals, Inc. | Methods of identifying and evaluating liver inflammation and liver fibrosis in a subject by determining a stratified score based on gene expression |
| CN118541487A (zh) * | 2022-01-20 | 2024-08-23 | 上海拓界生物医药科技有限公司 | 一种dsRNA、其应用及制备方法 |
| CN116515835A (zh) * | 2022-04-29 | 2023-08-01 | 北京福元医药股份有限公司 | 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途 |
| WO2023213284A1 (zh) * | 2022-05-06 | 2023-11-09 | 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 |
| US20240002857A1 (en) * | 2022-05-09 | 2024-01-04 | Sanegene Bio Usa Inc. | Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof |
| EP4569113A1 (en) * | 2022-09-15 | 2025-06-18 | Regeneron Pharmaceuticals, Inc. | 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof |
| EP4638742A1 (en) * | 2022-12-22 | 2025-10-29 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of 17beta-hydroxysteroid dehydrogenase type 13 (hsd17b13) |
| EP4677084A1 (en) * | 2023-03-07 | 2026-01-14 | Aligos Therapeutics, Inc. | Modified short inerfering nucleic acid (sina) molecules and uses thereof |
| CN118773194A (zh) * | 2023-06-16 | 2024-10-15 | 施能康医药科技(苏州)有限公司 | 靶向17-β-羟基类固醇脱氢酶13的核酸及其用途 |
| WO2025153088A1 (zh) * | 2024-01-18 | 2025-07-24 | 北京安龙生物医药有限公司 | 靶向第13型17β-羟基类固醇脱氢酶的寡核苷酸及其用途 |
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