CO2024000239A2 - Composiciones de arni de gen diana asociado con trastorno metabólico y sus métodos de uso - Google Patents
Composiciones de arni de gen diana asociado con trastorno metabólico y sus métodos de usoInfo
- Publication number
- CO2024000239A2 CO2024000239A2 CONC2024/0000239A CO2024000239A CO2024000239A2 CO 2024000239 A2 CO2024000239 A2 CO 2024000239A2 CO 2024000239 A CO2024000239 A CO 2024000239A CO 2024000239 A2 CO2024000239 A2 CO 2024000239A2
- Authority
- CO
- Colombia
- Prior art keywords
- metabolic disorder
- methods
- target gene
- compositions
- gene rnai
- Prior art date
Links
- 208000030159 metabolic disease Diseases 0.000 title abstract 4
- 108091030071 RNAI Proteins 0.000 title abstract 3
- 230000009368 gene silencing by RNA Effects 0.000 title abstract 3
- 108090000623 proteins and genes Proteins 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 2
- 102100034135 Activin receptor type-1C Human genes 0.000 abstract 2
- 101000799193 Homo sapiens Activin receptor type-1C Proteins 0.000 abstract 2
- 101001054832 Homo sapiens Inhibin beta C chain Proteins 0.000 abstract 2
- 101001054830 Homo sapiens Inhibin beta E chain Proteins 0.000 abstract 2
- 102100026812 Inhibin beta C chain Human genes 0.000 abstract 2
- 102000017795 Perilipin-1 Human genes 0.000 abstract 2
- 108010067162 Perilipin-1 Proteins 0.000 abstract 2
- 102000010861 Type 3 Cyclic Nucleotide Phosphodiesterases Human genes 0.000 abstract 2
- 108010037543 Type 3 Cyclic Nucleotide Phosphodiesterases Proteins 0.000 abstract 2
- 102100026818 Inhibin beta E chain Human genes 0.000 abstract 1
- 208000001145 Metabolic Syndrome Diseases 0.000 abstract 1
- 201000000690 abdominal obesity-metabolic syndrome Diseases 0.000 abstract 1
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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- 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
- A61P3/00—Drugs for disorders of the metabolism
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/1138—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 receptors or cell surface proteins
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04017—3',5'-Cyclic-nucleotide phosphodiesterase (3.1.4.17)
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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
- C12N2310/10—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
- C12N2310/312—Phosphonates
- C12N2310/3125—Methylphosphonates
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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
- C12N2310/315—Phosphorothioates
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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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- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Virology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (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)
Abstract
La presente invención se refiere a agentes de ARNi, p. ej., agentes de ARN bicatenario (ARNbc), que se dirigen a un gen diana asociado con un trastorno metabólico, p. ej., gen de subunidad de inhibina beta E (INHBE), receptor de activina A tipo 1C (ACVR1C), perilipina 1 (PLIN1), fosfodiesterasa 3B (PDE3B), o subunidad de inhibina beta C (INHBC). La invención se refiere además a los métodos de uso de tales agentes de ARNi para inhibir la expresión de un gen diana asociado al trastorno metabólico y a los métodos para tratar o prevenir un trastorno metabólico, p. ej., síndrome metabólico.
Applications Claiming Priority (7)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163223995P | 2021-07-21 | 2021-07-21 | |
US202163278126P | 2021-11-11 | 2021-11-11 | |
US202163285143P | 2021-12-02 | 2021-12-02 | |
US202163287578P | 2021-12-09 | 2021-12-09 | |
US202263321799P | 2022-03-21 | 2022-03-21 | |
US202263323543P | 2022-03-25 | 2022-03-25 | |
PCT/US2022/037658 WO2023003922A1 (en) | 2021-07-21 | 2022-07-20 | Metabolic disorder-associated target gene irna compositions and methods of use thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2024000239A2 true CO2024000239A2 (es) | 2024-02-05 |
Family
ID=84979577
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2024/0000239A CO2024000239A2 (es) | 2021-07-21 | 2024-01-12 | Composiciones de arni de gen diana asociado con trastorno metabólico y sus métodos de uso |
Country Status (9)
Country | Link |
---|---|
EP (1) | EP4373937A1 (es) |
JP (1) | JP2024526890A (es) |
KR (1) | KR20240036041A (es) |
AU (1) | AU2022314619A1 (es) |
CA (1) | CA3226887A1 (es) |
CO (1) | CO2024000239A2 (es) |
IL (1) | IL309897A (es) |
MX (1) | MX2024000981A (es) |
WO (1) | WO2023003922A1 (es) |
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2022
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- 2022-07-20 JP JP2024503574A patent/JP2024526890A/ja active Pending
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- 2022-07-20 WO PCT/US2022/037658 patent/WO2023003922A1/en active Application Filing
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WO2023003922A1 (en) | 2023-01-26 |
IL309897A (en) | 2024-03-01 |
EP4373937A1 (en) | 2024-05-29 |
CA3226887A1 (en) | 2023-01-26 |
JP2024526890A (ja) | 2024-07-19 |
MX2024000981A (es) | 2024-02-12 |
AU2022314619A1 (en) | 2024-01-04 |
KR20240036041A (ko) | 2024-03-19 |
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