AR129073A1 - Composiciones y métodos para modular la actividad de scap - Google Patents
Composiciones y métodos para modular la actividad de scapInfo
- Publication number
- AR129073A1 AR129073A1 ARP230100922A ARP230100922A AR129073A1 AR 129073 A1 AR129073 A1 AR 129073A1 AR P230100922 A ARP230100922 A AR P230100922A AR P230100922 A ARP230100922 A AR P230100922A AR 129073 A1 AR129073 A1 AR 129073A1
- Authority
- AR
- Argentina
- Prior art keywords
- scap
- activity
- sense strand
- antisense strand
- srebp
- Prior art date
Links
- 230000000694 effects Effects 0.000 title abstract 5
- 238000000034 method Methods 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title abstract 2
- 108091034117 Oligonucleotide Proteins 0.000 abstract 7
- 102000009822 Sterol Regulatory Element Binding Proteins Human genes 0.000 abstract 6
- 108010020396 Sterol Regulatory Element Binding Proteins Proteins 0.000 abstract 6
- 102100027223 Sterol regulatory element-binding protein cleavage-activating protein Human genes 0.000 abstract 6
- 101710184555 Sterol regulatory element-binding protein cleavage-activating protein Proteins 0.000 abstract 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 6
- 108091030071 RNAI Proteins 0.000 abstract 4
- 108091081021 Sense strand Proteins 0.000 abstract 4
- 230000000692 anti-sense effect Effects 0.000 abstract 4
- 230000009368 gene silencing by RNA Effects 0.000 abstract 4
- 201000010099 disease Diseases 0.000 abstract 3
- 102000004169 proteins and genes Human genes 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 206010003210 Arteriosclerosis Diseases 0.000 abstract 2
- 201000001320 Atherosclerosis Diseases 0.000 abstract 2
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 abstract 2
- 208000035475 disorder Diseases 0.000 abstract 2
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 208000032928 Dyslipidaemia Diseases 0.000 abstract 1
- 208000017170 Lipid metabolism disease Diseases 0.000 abstract 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical group CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 abstract 1
- 230000003213 activating effect Effects 0.000 abstract 1
- 238000003776 cleavage reaction Methods 0.000 abstract 1
- 229940124447 delivery agent Drugs 0.000 abstract 1
- 239000003937 drug carrier Substances 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 abstract 1
- 239000000546 pharmaceutical excipient Substances 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
- 230000007017 scission Effects 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
- 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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- 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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- 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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- 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
- C12N2310/312—Phosphonates
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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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- 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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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
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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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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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
Landscapes
- 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)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se describen oligonucleótidos y composiciones que los incluyen que modulan (por ejemplo, inhiben, limitan o reducen) la actividad de la proteína activadora de escisión de la proteína de unión al elemento regulador de esterol (SREBP) (SCAP). También se describen métodos para fabricar y usar los oligonucleótidos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociadas con la actividad de SCAP, como la enfermedad del hígado graso no alcohólico (NAFLD), (NASH), dislipidemia, enfermedad cardiovascular aterosclerótica (ASCVD) y/u otras afecciones, enfermedades y/o trastornos asociados con SCAP. Reivindicación 64: Un oligonucleótido de iARN para modular la actividad de la proteína activadora de la escisión de la proteína de unión al elemento regulador de esterol (SREBP) (SCAP), donde el oligonucleótido comprende una cadena sentido y una cadena antisentido, donde la cadena sentido y la cadena antisentido forman una región dúplex, donde todos los nucleótidos que comprenden la cadena sentido y la cadena antisentido están modificados, donde la cadena antisentido comprende una región de complementariedad con una secuencia objetivo de ARNm de SCAP de cualquiera de las SEQ ID Nº 777 a 783, y donde la región de complementariedad tiene una longitud de al menos 15 nucleótidos contiguos. Reivindicación 82: El oligonucleótido de iARN de la reivindicación 81, donde una secuencia de 5-GCAGCCGAAAGGCUGC-3 (SEQ ID Nº 784) en la cadena sentido forma una estructura de tallo-bucle, y en el que los nucleótidos AAA en una secuencia de 5-GAAA-3 en el bucle están cada uno conjugados con un resto de GalNAc monovalente. Reivindicación 92: Una composición farmacéutica que comprende: el oligonucleótido de iARN de cualquiera de las reivindicaciones 1 a 91, o una sal farmacéuticamente aceptable de este; y un portador, agente de administración o excipiente farmacéuticamente aceptable. Reivindicación 94: Un método para tratar a un individuo que tiene una enfermedad, trastorno o afección asociada con la actividad de la proteína activadora de la escisión de la proteína de unión al elemento regulador de esterol (SREBP) (SCAP), donde el método comprende la etapa de: administrarle al individuo una cantidad terapéuticamente eficaz del oligonucleótido de iARN de cualquiera de las reivindicaciones 1 a 91, o la composición farmacéutica de la reivindicación 92, mediante lo cual se trata al individuo.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263363091P | 2022-04-15 | 2022-04-15 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR129073A1 true AR129073A1 (es) | 2024-07-10 |
Family
ID=86330765
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP230100922A AR129073A1 (es) | 2022-04-15 | 2023-04-14 | Composiciones y métodos para modular la actividad de scap |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20230374522A1 (es) |
| AR (1) | AR129073A1 (es) |
| AU (1) | AU2023254798A1 (es) |
| TW (1) | TW202345873A (es) |
| WO (1) | WO2023201043A1 (es) |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6469158B1 (en) | 1992-05-14 | 2002-10-22 | Ribozyme Pharmaceuticals, Incorporated | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| US5804683A (en) | 1992-05-14 | 1998-09-08 | Ribozyme Pharmaceuticals, Inc. | Deprotection of RNA with alkylamine |
| US5977343A (en) | 1992-05-14 | 1999-11-02 | Ribozyme Pharmaceuticals, Inc. | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| DK0748382T3 (da) | 1993-09-02 | 2003-02-17 | Ribozyme Pharm Inc | Enzymatisk nukleinsyre indeholdende ikke-nukleotid |
| US5889136A (en) | 1995-06-09 | 1999-03-30 | The Regents Of The University Of Colorado | Orthoester protecting groups in RNA synthesis |
| US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
| US6111086A (en) | 1998-02-27 | 2000-08-29 | Scaringe; Stephen A. | Orthoester protecting groups |
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| JP7535047B2 (ja) * | 2018-12-12 | 2024-08-15 | ディセルナ ファーマシューティカルズ インコーポレイテッド | トリループを含む二本鎖核酸インヒビター分子 |
| TW202111124A (zh) * | 2019-05-30 | 2021-03-16 | 美商安進公司 | 用於抑制ｓｃａｐ表現之ｒｎａｉ構建體及其使用方法 |
| KR20220142445A (ko) * | 2020-01-15 | 2022-10-21 | 다이서나 파마수이티컬, 인크. | 4'-o-메틸렌 포스포네이트 핵산 및 이의 유사체 |
| CN116096889A (zh) * | 2020-03-18 | 2023-05-09 | 迪克纳制药公司 | 用于抑制angptl3表达的组合物和方法 |
| KR20230043912A (ko) * | 2020-08-05 | 2023-03-31 | 다이서나 파마수이티컬, 인크. | Lpa 발현을 저해하기 위한 조성물 및 방법 |
| AU2021356639A1 (en) * | 2020-10-08 | 2023-05-18 | Dicerna Pharmaceuticals, Inc. | Selective delivery of oligonucleotides to glial cells |
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2023
- 2023-04-14 US US18/300,937 patent/US20230374522A1/en active Pending
- 2023-04-14 AR ARP230100922A patent/AR129073A1/es unknown
- 2023-04-14 WO PCT/US2023/018657 patent/WO2023201043A1/en active Application Filing
- 2023-04-14 AU AU2023254798A patent/AU2023254798A1/en active Pending
- 2023-04-14 TW TW112113992A patent/TW202345873A/zh unknown
Also Published As
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|---|---|
| WO2023201043A1 (en) | 2023-10-19 |
| TW202345873A (zh) | 2023-12-01 |
| US20230374522A1 (en) | 2023-11-23 |
| AU2023254798A1 (en) | 2024-09-19 |
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