AR119061A1 - CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE SCAP Y MÉTODOS DE USO DE LAS MISMAS - Google Patents
CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE SCAP Y MÉTODOS DE USO DE LAS MISMASInfo
- Publication number
- AR119061A1 AR119061A1 ARP200101545A ARP200101545A AR119061A1 AR 119061 A1 AR119061 A1 AR 119061A1 AR P200101545 A ARP200101545 A AR P200101545A AR P200101545 A ARP200101545 A AR P200101545A AR 119061 A1 AR119061 A1 AR 119061A1
- Authority
- AR
- Argentina
- Prior art keywords
- rnai
- expression
- scap
- methods
- patient
- Prior art date
Links
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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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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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
- A61K31/713—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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- 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
- A61P3/04—Anorexiants; Antiobesity agents
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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
- A61P35/00—Antineoplastic agents
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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
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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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
- 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.
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/32—Chemical structure of the sugar
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- 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
- C12N2310/32—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
- 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
- C12N2310/32—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
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
-
- 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
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Child & Adolescent Psychology (AREA)
- Urology & Nephrology (AREA)
- Vascular Medicine (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
La presente se refiere a construcciones de ARNi para reducir la expresión del gen SCAP. También se describen métodos de uso de tales construcciones de ARNi para tratar o prevenir una hepatopatía, la esteatosis hepática no alcohólica (EHNA). Reivindicación 1: Una construcción de ARNi que comprende una hebra sentido y una hebra antisentido, en donde la hebra antisentido comprende una región que tiene al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de una secuencia antisentido enumerada en la Tabla 1 ó 2, y en donde la construcción de ARNi inhibe la expresión de la proteína activadora de la escisión de SREBP (SCAP). Reivindicación 33: Composición farmacéutica que comprende la construcción de ARNi según una cualquiera de las reivindicaciones 1 a 32 y un portador, excipiente o diluyente farmacéuticamente aceptable. Reivindicación 34: Un método para reducir la expresión de SCAP en un paciente que lo necesita que comprende administrar al paciente la construcción de ARNi según una cualquiera de las reivindicaciones 1 a 32.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962854433P | 2019-05-30 | 2019-05-30 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR119061A1 true AR119061A1 (es) | 2021-11-17 |
Family
ID=71833431
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP200101545A AR119061A1 (es) | 2019-05-30 | 2020-06-01 | CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE SCAP Y MÉTODOS DE USO DE LAS MISMAS |
Country Status (17)
Country | Link |
---|---|
US (1) | US20220307022A1 (es) |
EP (1) | EP3976786A2 (es) |
JP (1) | JP2022534402A (es) |
KR (1) | KR20220016138A (es) |
CN (1) | CN113924368A (es) |
AR (1) | AR119061A1 (es) |
AU (1) | AU2020284254A1 (es) |
BR (1) | BR112021024080A2 (es) |
CA (1) | CA3141902A1 (es) |
CL (1) | CL2021003169A1 (es) |
EA (1) | EA202193296A1 (es) |
IL (1) | IL288398A (es) |
MX (1) | MX2021014465A (es) |
SG (1) | SG11202113112WA (es) |
TW (1) | TW202111124A (es) |
UY (1) | UY38733A (es) |
WO (1) | WO2020243702A2 (es) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20230374522A1 (en) * | 2022-04-15 | 2023-11-23 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating scap activity |
TW202413642A (zh) * | 2022-05-25 | 2024-04-01 | 美商安進公司 | 用於抑制scap表現的rnai構建體及其使用方法 |
WO2024015796A1 (en) * | 2022-07-11 | 2024-01-18 | Sanegene Bio Usa Inc. | Optimized 2'- modified ribose derivatives and methods of use |
WO2024023262A2 (en) * | 2022-07-27 | 2024-02-01 | E-Therapeutics Plc | Nucleic acid compounds |
WO2024104416A1 (zh) * | 2022-11-17 | 2024-05-23 | 北京福元医药股份有限公司 | 抑制SCAP基因表达的siRNA、其缀合物和药物组合物及用途 |
Family Cites Families (37)
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US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
US5744101A (en) | 1989-06-07 | 1998-04-28 | Affymax Technologies N.V. | Photolabile nucleoside protecting groups |
US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
US5677195A (en) | 1991-11-22 | 1997-10-14 | Affymax Technologies N.V. | Combinatorial strategies for polymer synthesis |
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 |
US5556752A (en) | 1994-10-24 | 1996-09-17 | Affymetrix, Inc. | Surface-bound, unimolecular, double-stranded DNA |
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 |
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2020
- 2020-06-01 UY UY0001038733A patent/UY38733A/es unknown
- 2020-06-01 WO PCT/US2020/035545 patent/WO2020243702A2/en unknown
- 2020-06-01 BR BR112021024080A patent/BR112021024080A2/pt unknown
- 2020-06-01 AR ARP200101545A patent/AR119061A1/es unknown
- 2020-06-01 SG SG11202113112WA patent/SG11202113112WA/en unknown
- 2020-06-01 JP JP2021570170A patent/JP2022534402A/ja active Pending
- 2020-06-01 CA CA3141902A patent/CA3141902A1/en active Pending
- 2020-06-01 EP EP20746771.3A patent/EP3976786A2/en active Pending
- 2020-06-01 MX MX2021014465A patent/MX2021014465A/es unknown
- 2020-06-01 EA EA202193296A patent/EA202193296A1/ru unknown
- 2020-06-01 KR KR1020217042450A patent/KR20220016138A/ko unknown
- 2020-06-01 TW TW109118339A patent/TW202111124A/zh unknown
- 2020-06-01 US US17/615,520 patent/US20220307022A1/en active Pending
- 2020-06-01 AU AU2020284254A patent/AU2020284254A1/en active Pending
- 2020-06-01 CN CN202080041291.2A patent/CN113924368A/zh active Pending
-
2021
- 2021-11-25 IL IL288398A patent/IL288398A/en unknown
- 2021-11-29 CL CL2021003169A patent/CL2021003169A1/es unknown
Also Published As
Publication number | Publication date |
---|---|
US20220307022A1 (en) | 2022-09-29 |
CN113924368A (zh) | 2022-01-11 |
WO2020243702A2 (en) | 2020-12-03 |
EA202193296A1 (ru) | 2022-03-10 |
MX2021014465A (es) | 2022-01-06 |
EP3976786A2 (en) | 2022-04-06 |
TW202111124A (zh) | 2021-03-16 |
KR20220016138A (ko) | 2022-02-08 |
WO2020243702A3 (en) | 2021-04-22 |
CA3141902A1 (en) | 2020-12-03 |
IL288398A (en) | 2022-01-01 |
JP2022534402A (ja) | 2022-07-29 |
SG11202113112WA (en) | 2021-12-30 |
CL2021003169A1 (es) | 2023-01-20 |
AU2020284254A1 (en) | 2021-12-23 |
BR112021024080A2 (pt) | 2022-02-08 |
UY38733A (es) | 2020-11-30 |
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