AR125992A1 - Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1) - Google Patents
Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1)Info
- Publication number
- AR125992A1 AR125992A1 ARP220101411A ARP220101411A AR125992A1 AR 125992 A1 AR125992 A1 AR 125992A1 AR P220101411 A ARP220101411 A AR P220101411A AR P220101411 A ARP220101411 A AR P220101411A AR 125992 A1 AR125992 A1 AR 125992A1
- Authority
- AR
- Argentina
- Prior art keywords
- marc1
- expression
- adema
- galnac
- inhibiting
- Prior art date
Links
- 230000002401 inhibitory effect Effects 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 230000002438 mitochondrial effect Effects 0.000 title 1
- 102100033255 Mitochondrial amidoxime-reducing component 1 Human genes 0.000 abstract 4
- 101150080343 mtarc1 gene Proteins 0.000 abstract 4
- 108091034117 Oligonucleotide Proteins 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 3
- 108091030071 RNAI Proteins 0.000 abstract 2
- 108091081021 Sense strand Proteins 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 230000009368 gene silencing by RNA Effects 0.000 abstract 2
- 238000012986 modification Methods 0.000 abstract 2
- 230000004048 modification Effects 0.000 abstract 2
- 239000002342 ribonucleoside Substances 0.000 abstract 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 1
- 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 1
- 229940124447 delivery agent Drugs 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 239000003937 drug carrier Substances 0.000 abstract 1
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 239000000546 pharmaceutical excipient Substances 0.000 abstract 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 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
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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
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- 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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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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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
- C12N2310/312—Phosphonates
- C12N2310/3125—Methylphosphonates
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- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
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Abstract
En la presente descripción se proporcionan oligonucleótidos que inhiben la expresión de MARC1. Además, se proporcionan composiciones que incluyen los mismos y usos de estos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociados con la expresión de MARC1. Reivindicación 1: Un oligonucleótido de ARNi bicatenario (ARNbci) para inhibir la expresión de MARC1, en donde dicho ARNbc comprende una hebra sentido y una hebra antisentido, la hebra antisentido comprende una región de complementariedad con un transcrito de ARN de MARC1, en donde la hebra sentido comprende la secuencia y todas las modificaciones de 5-mGs-mG-mC-mU-mA-mG-mA-fG-fA-fA-fG-mA-mA-mA-mG-mU-mU-mA-mA-mA-mG-mC-mA-mG-mC-mC-mG-[ademA-GalNAc]-[ademA-GalNAc]-[ademA-GalNAc]-mG-mG-mC-mU-mG-mC-3 (SEQ ID Nº 1615), y en donde la hebra antisentido comprende la secuencia y todas las modificaciones de 5-MeFosfonato-4O-mUs-fUs-fUs-fA-fA-mC-fU-mU-mU-fC-mU-mU-mC-fU-mC-mU-mA-mG-mC-mCs-mGs-mG-3 (SEQ ID Nº 1651), en donde mC, mA, mG, mU = 2-OMe ribonucleósidos; fA, fC, fG, fU = 2F ribonucleósidos; s = fosforotioato, y en donde ademA-GalNAc = fórmula (1). Reivindicación 13: Una composición farmacéutica que comprende el oligonucleótido de ARNi de acuerdo con cualquiera de las reivindicaciones 1 - 12, y un portador, agente de suministro o excipiente farmacéuticamente aceptables.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163194395P | 2021-05-28 | 2021-05-28 | |
| EP21183860 | 2021-07-06 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR125992A1 true AR125992A1 (es) | 2023-08-30 |
Family
ID=82067447
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220101411A AR125992A1 (es) | 2021-05-28 | 2022-05-27 | Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1) |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US11655473B2 (es) |
| EP (1) | EP4347820A1 (es) |
| JP (2) | JP7463621B2 (es) |
| KR (1) | KR102662071B1 (es) |
| AR (1) | AR125992A1 (es) |
| AU (1) | AU2022282579A1 (es) |
| BR (1) | BR112023023807A2 (es) |
| CA (1) | CA3215836A1 (es) |
| CL (1) | CL2023003363A1 (es) |
| CO (1) | CO2023016136A2 (es) |
| IL (1) | IL308418A (es) |
| MX (1) | MX2023013504A (es) |
| TW (1) | TWI831225B (es) |
| WO (1) | WO2022248665A1 (es) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024192379A1 (en) * | 2023-03-16 | 2024-09-19 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of mitochondrial amidoxime reducing component 1 (marc1), pharmaceutical compositions thereof, and methods of use |
Family Cites Families (27)
| 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 |
| JPH09502092A (ja) | 1993-09-02 | 1997-03-04 | リボザイム・ファーマシューティカルズ・インコーポレイテッド | 非ヌクレオチドを含有する酵素性核酸 |
| 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 |
| DK1309726T4 (en) | 2000-03-30 | 2019-01-28 | Whitehead Inst Biomedical Res | RNA Sequence-Specific Mediators of RNA Interference |
| CZ308053B6 (cs) | 2000-12-01 | 2019-11-27 | Max Planck Gesellschaft | Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití |
| US20050159378A1 (en) | 2001-05-18 | 2005-07-21 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA) |
| WO2003040395A2 (en) | 2001-11-07 | 2003-05-15 | Applera Corporation | Universal nucleotides for nucleic acid analysis |
| DK2284266T3 (da) | 2002-11-14 | 2014-01-13 | Thermo Fisher Scient Biosciences Inc | sIRNA-MOLEKYLE MOD TP53 |
| WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
| US20070265220A1 (en) | 2004-03-15 | 2007-11-15 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
| NL1026610C2 (nl) * | 2004-07-08 | 2006-01-10 | Wp Suspension B V | Schokbreker met drukondersteunde verstelling. |
| US20090018097A1 (en) | 2005-09-02 | 2009-01-15 | Mdrna, Inc | Modification of double-stranded ribonucleic acid molecules |
| ES2708944T3 (es) | 2008-09-22 | 2019-04-12 | Dicerna Pharmaceuticals Inc | Composiciones y métodos para la inhibición específica de la expresión de genes por DSRNA que tenga modificaciones |
| US8513207B2 (en) | 2008-12-18 | 2013-08-20 | Dicerna Pharmaceuticals, Inc. | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| WO2010093788A2 (en) | 2009-02-11 | 2010-08-19 | Dicerna Pharmaceuticals, Inc. | Multiplex dicer substrate rna interference molecules having joining sequences |
| US8927513B2 (en) | 2009-07-07 | 2015-01-06 | Alnylam Pharmaceuticals, Inc. | 5′ phosphate mimics |
| US9725479B2 (en) | 2010-04-22 | 2017-08-08 | Ionis Pharmaceuticals, Inc. | 5′-end derivatives |
| RU2014119787A (ru) | 2011-10-25 | 2015-12-10 | Айсис Фармасьютикалс, Инк. | Антисмысловая регуляция экспрессии gccr |
| CA2970801C (en) | 2014-12-15 | 2024-02-13 | Dicerna Pharmaceuticals, Inc. | Ligand-modified double-stranded nucleic acids |
| CN110072530A (zh) | 2016-09-02 | 2019-07-30 | 迪克纳制药公司 | 4′-磷酸酯类似物和包含其的寡核苷酸 |
| CN110799192A (zh) * | 2017-03-27 | 2020-02-14 | 加利福尼亚大学董事会 | 治疗癌症的组合物和方法 |
| US10835581B2 (en) | 2017-11-28 | 2020-11-17 | University of Pittsburgh—of the Commonwealth System of Higher Education | Method of treating insulin resistance |
| US20200241005A1 (en) | 2019-01-25 | 2020-07-30 | Viscient Biosciences, Inc. | Compositions and methods for the diagnosis and treatment of diseases of the liver |
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2022
- 2022-05-27 AR ARP220101411A patent/AR125992A1/es unknown
- 2022-05-27 JP JP2023523030A patent/JP7463621B2/ja active Active
- 2022-05-27 BR BR112023023807A patent/BR112023023807A2/pt unknown
- 2022-05-27 WO PCT/EP2022/064408 patent/WO2022248665A1/en active Application Filing
- 2022-05-27 EP EP22730852.5A patent/EP4347820A1/en active Pending
- 2022-05-27 KR KR1020237015531A patent/KR102662071B1/ko active IP Right Grant
- 2022-05-27 AU AU2022282579A patent/AU2022282579A1/en active Pending
- 2022-05-27 TW TW111119898A patent/TWI831225B/zh active
- 2022-05-27 CA CA3215836A patent/CA3215836A1/en active Pending
- 2022-05-27 US US17/826,211 patent/US11655473B2/en active Active
- 2022-05-27 MX MX2023013504A patent/MX2023013504A/es unknown
- 2022-05-27 IL IL308418A patent/IL308418A/en unknown
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2023
- 2023-04-14 US US18/134,585 patent/US20230340490A1/en active Pending
- 2023-11-10 CL CL2023003363A patent/CL2023003363A1/es unknown
- 2023-11-29 CO CONC2023/0016136A patent/CO2023016136A2/es unknown
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2024
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| Publication number | Publication date |
|---|---|
| EP4347820A1 (en) | 2024-04-10 |
| MX2023013504A (es) | 2024-02-23 |
| US20230042451A1 (en) | 2023-02-09 |
| JP2024071571A (ja) | 2024-05-24 |
| IL308418A (en) | 2024-01-01 |
| CO2023016136A2 (es) | 2024-02-15 |
| JP2024508069A (ja) | 2024-02-22 |
| KR102662071B1 (ko) | 2024-05-03 |
| CL2023003363A1 (es) | 2024-06-21 |
| TW202307206A (zh) | 2023-02-16 |
| BR112023023807A2 (pt) | 2024-02-20 |
| US20230340490A1 (en) | 2023-10-26 |
| AU2022282579A1 (en) | 2023-11-02 |
| KR20230130609A (ko) | 2023-09-12 |
| JP7463621B2 (ja) | 2024-04-08 |
| US11655473B2 (en) | 2023-05-23 |
| TWI831225B (zh) | 2024-02-01 |
| AU2022282579A9 (en) | 2023-11-16 |
| WO2022248665A1 (en) | 2022-12-01 |
| CA3215836A1 (en) | 2022-12-01 |
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