CO2020010342A2 - Composiciones de arni de 17 β -hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas - Google Patents
Composiciones de arni de 17 β -hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismasInfo
- Publication number
- CO2020010342A2 CO2020010342A2 CONC2020/0010342A CO2020010342A CO2020010342A2 CO 2020010342 A2 CO2020010342 A2 CO 2020010342A2 CO 2020010342 A CO2020010342 A CO 2020010342A CO 2020010342 A2 CO2020010342 A2 CO 2020010342A2
- Authority
- CO
- Colombia
- Prior art keywords
- hsd17b13
- methods
- compositions
- 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
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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
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- 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
- 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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- 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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- 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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- C—CHEMISTRY; METALLURGY
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth 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)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (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)
Abstract
RESUMEN 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 (4)
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 | |
PCT/US2019/023079 WO2019183164A1 (en) | 2018-03-21 | 2019-03-20 | 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2020010342A2 true CO2020010342A2 (es) | 2020-08-31 |
Family
ID=66001358
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2020/0010342A CO2020010342A2 (es) | 2018-03-21 | 2020-08-21 | Composiciones de arni de 17 β -hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas |
Country Status (16)
Country | Link |
---|---|
US (2) | US20210380985A1 (es) |
EP (1) | EP3768838A1 (es) |
JP (2) | JP7507093B2 (es) |
KR (1) | KR20200133373A (es) |
CN (1) | CN112020556A (es) |
AU (1) | AU2019239971A1 (es) |
BR (1) | BR112020018758A2 (es) |
CA (1) | CA3091146A1 (es) |
CL (1) | CL2020002401A1 (es) |
CO (1) | CO2020010342A2 (es) |
IL (1) | IL277329A (es) |
MX (1) | MX2020009812A (es) |
PH (1) | PH12020500622A1 (es) |
SG (1) | SG11202007583SA (es) |
TW (1) | TW202003848A (es) |
WO (1) | WO2019183164A1 (es) |
Families Citing this family (20)
Publication number | Priority date | Publication date | Assignee | Title |
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KR102619197B1 (ko) | 2017-01-23 | 2024-01-03 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
KR20240125690A (ko) | 2017-10-11 | 2024-08-19 | 리제너론 파마슈티칼스 인코포레이티드 | Pnpla3 i148m 변이를 발현하는 환자의 간 질환의 치료에서의 hsd17b13의 저해 |
EP3768838A1 (en) | 2018-03-21 | 2021-01-27 | Regeneron Pharmaceuticals, Inc. | 17ß-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) IRNA COMPOSITIONS AND METHODS OF USE THEREOF |
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 |
US20220273691A1 (en) * | 2018-12-21 | 2022-09-01 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
AU2021284377A1 (en) * | 2020-06-01 | 2022-12-22 | Amgen Inc. | RNAi constructs for inhibiting HSD17B13 expression and methods of use thereof |
WO2022098748A1 (en) * | 2020-11-06 | 2022-05-12 | Inipharm, Inc. | Uses for hsd17b13 inhibitors |
EP4267739A1 (en) | 2020-12-23 | 2023-11-01 | Regeneron Pharmaceuticals, Inc. | Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors |
US20240229037A1 (en) * | 2021-04-22 | 2024-07-11 | Tuojie Biotech(Shanghai) Co., Ltd. | SIRNA TARGETING 17Beta-HYDROXYSTEROID DEHYDROGENASE TYPE 13 AND SIRNA CONJUGATE |
CN118234862A (zh) * | 2021-09-08 | 2024-06-21 | 阿利戈斯治疗公司 | 经修饰的短干扰核酸分子(siNA)及其用途 |
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 |
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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 |
TW202400791A (zh) * | 2022-05-06 | 2024-01-01 | 大陸商蘇州瑞博生物技術股份有限公司 | 一種核酸、含有該核酸的組合物與綴合物及製備方法和用途 |
WO2023220561A1 (en) * | 2022-05-09 | 2023-11-16 | Sanegene Bio Usa Inc. | Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof |
WO2024059165A1 (en) * | 2022-09-15 | 2024-03-21 | Alnylam Pharmaceuticals, Inc. | 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof |
WO2024131916A1 (en) * | 2022-12-22 | 2024-06-27 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of 17beta-hydroxysteroid dehydrogenase type 13 (hsd17b13) |
WO2024187038A1 (en) * | 2023-03-07 | 2024-09-12 | Aligos Therapeutics, Inc. | Modified short inerfering nucleic acid (sina) molecules and uses thereof |
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2019
- 2019-03-20 EP EP19715322.4A patent/EP3768838A1/en active Pending
- 2019-03-20 SG SG11202007583SA patent/SG11202007583SA/en unknown
- 2019-03-20 AU AU2019239971A patent/AU2019239971A1/en active Pending
- 2019-03-20 CN CN201980020643.3A patent/CN112020556A/zh active Pending
- 2019-03-20 BR BR112020018758-5A patent/BR112020018758A2/pt unknown
- 2019-03-20 JP JP2020550112A patent/JP7507093B2/ja active Active
- 2019-03-20 MX MX2020009812A patent/MX2020009812A/es unknown
- 2019-03-20 CA CA3091146A patent/CA3091146A1/en active Pending
- 2019-03-20 US US16/978,947 patent/US20210380985A1/en active Pending
- 2019-03-20 KR KR1020207030127A patent/KR20200133373A/ko not_active Application Discontinuation
- 2019-03-20 WO PCT/US2019/023079 patent/WO2019183164A1/en unknown
- 2019-03-21 TW TW108109810A patent/TW202003848A/zh unknown
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2020
- 2020-08-19 PH PH12020500622A patent/PH12020500622A1/en unknown
- 2020-08-21 CO CONC2020/0010342A patent/CO2020010342A2/es unknown
- 2020-09-14 IL IL277329A patent/IL277329A/en unknown
- 2020-09-16 CL CL2020002401A patent/CL2020002401A1/es unknown
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2021
- 2021-05-14 US US17/321,001 patent/US11180757B1/en active Active
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2024
- 2024-05-01 JP JP2024074408A patent/JP2024105396A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
MX2020009812A (es) | 2021-01-08 |
EP3768838A1 (en) | 2021-01-27 |
IL277329A (en) | 2020-10-29 |
US20210380985A1 (en) | 2021-12-09 |
JP7507093B2 (ja) | 2024-06-27 |
AU2019239971A1 (en) | 2020-09-03 |
CA3091146A1 (en) | 2019-09-26 |
JP2024105396A (ja) | 2024-08-06 |
KR20200133373A (ko) | 2020-11-27 |
PH12020500622A1 (en) | 2021-03-15 |
JP2021518125A (ja) | 2021-08-02 |
TW202003848A (zh) | 2020-01-16 |
US11180757B1 (en) | 2021-11-23 |
BR112020018758A2 (pt) | 2021-01-26 |
CN112020556A (zh) | 2020-12-01 |
CL2020002401A1 (es) | 2021-01-29 |
WO2019183164A1 (en) | 2019-09-26 |
SG11202007583SA (en) | 2020-09-29 |
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