AR126085A1 - Agonistas de progranulina de oligonucleótido - Google Patents
Agonistas de progranulina de oligonucleótidoInfo
- Publication number
- AR126085A1 AR126085A1 ARP220101492A ARP220101492A AR126085A1 AR 126085 A1 AR126085 A1 AR 126085A1 AR P220101492 A ARP220101492 A AR P220101492A AR P220101492 A ARP220101492 A AR P220101492A AR 126085 A1 AR126085 A1 AR 126085A1
- Authority
- AR
- Argentina
- Prior art keywords
- progranulin
- oligonucleotide
- cell
- nucleotides
- length
- Prior art date
Links
- 108010012809 Progranulins Proteins 0.000 title abstract 8
- 102000019204 Progranulins Human genes 0.000 title abstract 7
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 5
- 239000000556 agonist Substances 0.000 title abstract 3
- 210000004027 cell Anatomy 0.000 abstract 4
- 238000000034 method Methods 0.000 abstract 3
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 108700039691 Genetic Promoter Regions Proteins 0.000 abstract 1
- 101001027324 Homo sapiens Progranulin Proteins 0.000 abstract 1
- 208000012902 Nervous system disease Diseases 0.000 abstract 1
- 208000025966 Neurological disease Diseases 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
- 230000000295 complement effect Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 210000005260 human cell Anatomy 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 210000004962 mammalian cell Anatomy 0.000 abstract 1
Classifications
-
- 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
-
- 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
- C12N2310/323—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
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente invención se refiere a oligonucleótidos que regulan de manera ascendente o restauran la expresión de progranulina en células al actuar sobre la región promotora del gen de progranulina. La invención se refiere además a composiciones farmacéuticas y métodos para el tratamiento de enfermedades asociadas a progranulina, específicamente, haploinsuficiencia de progranulina y trastornos neurológicos. Reivindicación 1: Un agonista de progranulina de oligonucleótido, en donde el oligonucleótido tiene 8 - 40 nucleótidos de longitud y comprende una secuencia contigua de 8 - 40 nucleótidos de longitud que es complementaria del promotor del gen de progranulina humana. Reivindicación 14: Un método in vivo o in vitro para regular de manera ascendente o restaurar la expresión de progranulina en una célula diana, en donde dicho método comprende administrar el agonista de progranulina de oligonucleótido de acuerdo con cualquiera de las reivindicaciones 1 a 12, o la composición farmacéutica de acuerdo con la reivindicación 13, en una cantidad eficaz a dicha célula, en donde la célula es opcionalmente una célula humana o una célula de mamífero.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP21178235 | 2021-06-08 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR126085A1 true AR126085A1 (es) | 2023-09-13 |
Family
ID=76355258
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220101492A AR126085A1 (es) | 2021-06-08 | 2022-06-06 | Agonistas de progranulina de oligonucleótido |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20220403388A1 (es) |
| EP (1) | EP4352222A1 (es) |
| JP (1) | JP2024524874A (es) |
| KR (1) | KR20240019228A (es) |
| CN (1) | CN117441018A (es) |
| AR (1) | AR126085A1 (es) |
| AU (1) | AU2022288115A1 (es) |
| BR (1) | BR112023025676A2 (es) |
| CA (1) | CA3222546A1 (es) |
| TW (1) | TW202313976A (es) |
| WO (1) | WO2022258555A1 (es) |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| AU9063398A (en) | 1997-09-12 | 1999-04-05 | Exiqon A/S | Oligonucleotide analogues |
| DK1152009T4 (en) | 1999-02-12 | 2017-12-11 | Daiichi Sankyo Co Ltd | HIS UNKNOWN NUCLEOSIDES AND OLIGONUCLEOTIDE ANALOGS |
| DK1178999T3 (da) | 1999-05-04 | 2007-08-06 | Santaris Pharma As | L-RIBO-LNA-analoger |
| US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| DK1569661T3 (da) | 2002-11-18 | 2010-01-11 | Santaris Pharma As | Antisense design |
| EP1752536A4 (en) * | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
| EP1984381B1 (en) | 2006-01-27 | 2010-09-29 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| EP2066684B1 (en) | 2006-05-11 | 2012-07-18 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
| WO2008150729A2 (en) | 2007-05-30 | 2008-12-11 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| DK2176280T4 (en) | 2007-07-05 | 2015-07-20 | Isis Pharmaceuticals Inc | 6-Disubstituerede bicykliske nukleinsyreanaloge |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| WO2010036698A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| WO2011017521A2 (en) | 2009-08-06 | 2011-02-10 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| WO2011156202A1 (en) | 2010-06-08 | 2011-12-15 | Isis Pharmaceuticals, Inc. | Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| WO2013154798A1 (en) | 2012-04-09 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| EP2850183A4 (en) * | 2012-05-16 | 2016-02-10 | Rana Therapeutics Inc | COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION |
| MY173826A (en) | 2012-11-15 | 2020-02-24 | Roche Innovation Ct Copenhagen As | Oligonucleotide conjugates |
| JP2017505623A (ja) | 2014-01-30 | 2017-02-23 | エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ | 生物切断性コンジュゲートを有するポリオリゴマー化合物 |
-
2022
- 2022-06-06 AR ARP220101492A patent/AR126085A1/es unknown
- 2022-06-06 WO PCT/EP2022/065298 patent/WO2022258555A1/en not_active Ceased
- 2022-06-06 US US17/833,085 patent/US20220403388A1/en not_active Abandoned
- 2022-06-06 JP JP2023575733A patent/JP2024524874A/ja active Pending
- 2022-06-06 BR BR112023025676A patent/BR112023025676A2/pt not_active Application Discontinuation
- 2022-06-06 EP EP22732985.1A patent/EP4352222A1/en active Pending
- 2022-06-06 CN CN202280040635.7A patent/CN117441018A/zh active Pending
- 2022-06-06 KR KR1020247000172A patent/KR20240019228A/ko active Pending
- 2022-06-06 AU AU2022288115A patent/AU2022288115A1/en active Pending
- 2022-06-06 TW TW111120879A patent/TW202313976A/zh unknown
- 2022-06-06 CA CA3222546A patent/CA3222546A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022258555A1 (en) | 2022-12-15 |
| KR20240019228A (ko) | 2024-02-14 |
| JP2024524874A (ja) | 2024-07-09 |
| TW202313976A (zh) | 2023-04-01 |
| EP4352222A1 (en) | 2024-04-17 |
| CA3222546A1 (en) | 2022-12-15 |
| US20220403388A1 (en) | 2022-12-22 |
| CN117441018A (zh) | 2024-01-23 |
| AU2022288115A1 (en) | 2023-12-07 |
| BR112023025676A2 (pt) | 2024-02-27 |
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