CL2023000407A1 - Construcciones de iarn y métodos para inhibir la expresión de marc1 - Google Patents
Construcciones de iarn y métodos para inhibir la expresión de marc1Info
- Publication number
- CL2023000407A1 CL2023000407A1 CL2023000407A CL2023000407A CL2023000407A1 CL 2023000407 A1 CL2023000407 A1 CL 2023000407A1 CL 2023000407 A CL2023000407 A CL 2023000407A CL 2023000407 A CL2023000407 A CL 2023000407A CL 2023000407 A1 CL2023000407 A1 CL 2023000407A1
- Authority
- CL
- Chile
- Prior art keywords
- marc1
- expression
- methods
- constructs
- iarn
- Prior art date
Links
- 101150080343 mtarc1 gene Proteins 0.000 title abstract 2
- 108091030071 RNAI Proteins 0.000 abstract 2
- 230000009368 gene silencing by RNA Effects 0.000 abstract 2
- 206010019708 Hepatic steatosis Diseases 0.000 abstract 1
- 208000019425 cirrhosis of liver Diseases 0.000 abstract 1
- 230000002440 hepatic effect Effects 0.000 abstract 1
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 abstract 1
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 abstract 1
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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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- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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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
- 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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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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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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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6869—Methods for sequencing
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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
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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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- 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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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/332—Abasic residue
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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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- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Analytical Chemistry (AREA)
- Immunology (AREA)
- Cardiology (AREA)
- Obesity (AREA)
- Hematology (AREA)
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- Heart & Thoracic Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente invención se refiere a construcciones de iARN para reducir la expresión del gen MARC1. También se describen métodos para usar dichas construcciones de iARN para tratar o prevenir la fibrosis hepática y las esteatosis hepáticas, tales como la esteatosis hepática no alcohólica y la esteatohepatitis no alcohólica.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063065190P | 2020-08-13 | 2020-08-13 | |
US202163214016P | 2021-06-23 | 2021-06-23 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2023000407A1 true CL2023000407A1 (es) | 2023-09-29 |
Family
ID=77640774
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2023000407A CL2023000407A1 (es) | 2020-08-13 | 2023-02-09 | Construcciones de iarn y métodos para inhibir la expresión de marc1 |
Country Status (16)
Country | Link |
---|---|
US (1) | US20220047621A1 (es) |
EP (1) | EP4196584A2 (es) |
JP (1) | JP2023537943A (es) |
KR (1) | KR20230046319A (es) |
CN (1) | CN116194119A (es) |
AU (1) | AU2021326521A1 (es) |
CA (1) | CA3190868A1 (es) |
CL (1) | CL2023000407A1 (es) |
CO (1) | CO2023002966A2 (es) |
CR (1) | CR20230126A (es) |
IL (1) | IL300360A (es) |
MX (1) | MX2023001786A (es) |
PE (1) | PE20230993A1 (es) |
TW (1) | TW202221124A (es) |
UY (1) | UY39382A (es) |
WO (1) | WO2022036126A2 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2024023262A2 (en) * | 2022-07-27 | 2024-02-01 | E-Therapeutics Plc | Nucleic acid compounds |
Family Cites Families (31)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
US5981505A (en) | 1993-01-26 | 1999-11-09 | The Trustees Of The University Of Pennsylvania | Compositions and methods for delivery of genetic material |
US5837533A (en) | 1994-09-28 | 1998-11-17 | American Home Products Corporation | Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent |
US5840710A (en) | 1994-12-09 | 1998-11-24 | Genzyme Corporation | Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules |
US7422902B1 (en) | 1995-06-07 | 2008-09-09 | The University Of British Columbia | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
US5981501A (en) | 1995-06-07 | 1999-11-09 | Inex Pharmaceuticals Corp. | Methods for encapsulating plasmids in lipid bilayers |
IL122290A0 (en) | 1995-06-07 | 1998-04-05 | Inex Pharmaceuticals Corp | Lipid-nucleic acid complex its preparation and use |
US6217900B1 (en) | 1997-04-30 | 2001-04-17 | American Home Products Corporation | Vesicular complexes and methods of making and using the same |
US6887906B1 (en) | 1997-07-01 | 2005-05-03 | Isispharmaceuticals, Inc. | Compositions and methods for the delivery of oligonucleotides via the alimentary canal |
US7833992B2 (en) | 2001-05-18 | 2010-11-16 | Merck Sharpe & Dohme | Conjugates and compositions for cellular delivery |
US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
US6693187B1 (en) | 2000-10-17 | 2004-02-17 | Lievre Cornu Llc | Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge |
US20030130186A1 (en) | 2001-07-20 | 2003-07-10 | Chandra Vargeese | Conjugates and compositions for cellular delivery |
US7109165B2 (en) | 2001-05-18 | 2006-09-19 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
US9181551B2 (en) | 2002-02-20 | 2015-11-10 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
WO2003093449A2 (en) | 2002-05-06 | 2003-11-13 | Nucleonics, Inc. | Methods for delivery of nucleic acids |
EP1620544B1 (en) | 2003-04-17 | 2018-09-19 | Alnylam Pharmaceuticals Inc. | MODIFIED iRNA AGENTS |
US7851615B2 (en) | 2003-04-17 | 2010-12-14 | Alnylam Pharmaceuticals, Inc. | Lipophilic conjugated iRNA agents |
US8017762B2 (en) | 2003-04-17 | 2011-09-13 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
US7723509B2 (en) | 2003-04-17 | 2010-05-25 | Alnylam Pharmaceuticals | IRNA agents with biocleavable tethers |
US8877917B2 (en) | 2007-04-23 | 2014-11-04 | Alnylam Pharmaceuticals, Inc. | Glycoconjugates of RNA interference agents |
WO2009082607A2 (en) | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
US20110081362A1 (en) * | 2008-01-31 | 2011-04-07 | The Brigham And Women's Hospital, Inc. | Treatment of cancer |
AR090905A1 (es) | 2012-05-02 | 2014-12-17 | Merck Sharp & Dohme | Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica |
IL284593B2 (en) * | 2013-05-01 | 2023-02-01 | Ionis Pharmaceuticals Inc | Compositions and methods for modulation of hbv and ttr expression |
JP6694382B2 (ja) | 2013-06-21 | 2020-05-13 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | 標的核酸を調節するための組成物および方法 |
CA2997444A1 (en) | 2015-09-29 | 2017-04-06 | Amgen Inc. | Asgr inhibitors for reducing cholesterol levels |
US20220049252A1 (en) | 2018-12-10 | 2022-02-17 | Amgen Inc. | CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF |
US20230183707A1 (en) * | 2020-05-21 | 2023-06-15 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting marc1 gene expression |
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2021
- 2021-08-12 IL IL300360A patent/IL300360A/en unknown
- 2021-08-12 PE PE2023000271A patent/PE20230993A1/es unknown
- 2021-08-12 KR KR1020237008366A patent/KR20230046319A/ko unknown
- 2021-08-12 MX MX2023001786A patent/MX2023001786A/es unknown
- 2021-08-12 CR CR20230126A patent/CR20230126A/es unknown
- 2021-08-12 WO PCT/US2021/045784 patent/WO2022036126A2/en active Application Filing
- 2021-08-12 CN CN202180055964.4A patent/CN116194119A/zh active Pending
- 2021-08-12 CA CA3190868A patent/CA3190868A1/en active Pending
- 2021-08-12 AU AU2021326521A patent/AU2021326521A1/en active Pending
- 2021-08-12 US US17/401,114 patent/US20220047621A1/en active Pending
- 2021-08-12 JP JP2023509499A patent/JP2023537943A/ja active Pending
- 2021-08-12 EP EP21766066.1A patent/EP4196584A2/en active Pending
- 2021-08-13 UY UY0001039382A patent/UY39382A/es unknown
- 2021-08-13 TW TW110129910A patent/TW202221124A/zh unknown
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2023
- 2023-02-09 CL CL2023000407A patent/CL2023000407A1/es unknown
- 2023-03-10 CO CONC2023/0002966A patent/CO2023002966A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
PE20230993A1 (es) | 2023-06-23 |
US20220047621A1 (en) | 2022-02-17 |
TW202221124A (zh) | 2022-06-01 |
CN116194119A (zh) | 2023-05-30 |
UY39382A (es) | 2022-02-25 |
WO2022036126A3 (en) | 2022-04-21 |
MX2023001786A (es) | 2023-03-10 |
CA3190868A1 (en) | 2022-02-17 |
WO2022036126A2 (en) | 2022-02-17 |
KR20230046319A (ko) | 2023-04-05 |
CO2023002966A2 (es) | 2023-03-17 |
AU2021326521A1 (en) | 2023-03-09 |
IL300360A (en) | 2023-04-01 |
CR20230126A (es) | 2023-05-03 |
EP4196584A2 (en) | 2023-06-21 |
JP2023537943A (ja) | 2023-09-06 |
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