BR112023025676A2 - Agonistas de oligonucleotídeos de progranulina - Google Patents
Agonistas de oligonucleotídeos de progranulinaInfo
- Publication number
- BR112023025676A2 BR112023025676A2 BR112023025676A BR112023025676A BR112023025676A2 BR 112023025676 A2 BR112023025676 A2 BR 112023025676A2 BR 112023025676 A BR112023025676 A BR 112023025676A BR 112023025676 A BR112023025676 A BR 112023025676A BR 112023025676 A2 BR112023025676 A2 BR 112023025676A2
- Authority
- BR
- Brazil
- Prior art keywords
- progranulin
- oligonucleotide agonists
- agonists
- oligonucleotide
- relates
- Prior art date
Links
- 108010012809 Progranulins Proteins 0.000 title abstract 6
- 102000019204 Progranulins Human genes 0.000 title abstract 5
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 3
- 239000000556 agonist Substances 0.000 title abstract 2
- 108700039691 Genetic Promoter Regions 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
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 230000008685 targeting Effects 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)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
agonistas de oligonucleotídeos de progranulina. a presente invenção refere-se a oligonucleotídeos que regulam positivamente ou restauram a expressão de progranulina em células, visando a região promotora do gene da progranulina. a invenção refere-se ainda a composições farmacêuticas e métodos para o tratamento de doenças associadas à progranulina, especificamente haploinsuficiência de progranulina e distúrbios neurológicos.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP21178235 | 2021-06-08 | ||
PCT/EP2022/065298 WO2022258555A1 (en) | 2021-06-08 | 2022-06-06 | Oligonucleotide progranulin agonists |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112023025676A2 true BR112023025676A2 (pt) | 2024-02-27 |
Family
ID=76355258
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112023025676A BR112023025676A2 (pt) | 2021-06-08 | 2022-06-06 | Agonistas de oligonucleotídeos de progranulina |
Country Status (10)
Country | Link |
---|---|
US (1) | US20220403388A1 (pt) |
EP (1) | EP4352222A1 (pt) |
KR (1) | KR20240019228A (pt) |
CN (1) | CN117441018A (pt) |
AR (1) | AR126085A1 (pt) |
AU (1) | AU2022288115A1 (pt) |
BR (1) | BR112023025676A2 (pt) |
CA (1) | CA3222546A1 (pt) |
TW (1) | TW202313976A (pt) |
WO (1) | WO2022258555A1 (pt) |
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 |
EP2341058A3 (en) | 1997-09-12 | 2011-11-23 | Exiqon A/S | Oligonucleotide Analogues |
NZ513402A (en) | 1999-02-12 | 2003-06-30 | Sankyo Co | Novel nucleosides and oligonucleotide analogues |
PT1178999E (pt) | 1999-05-04 | 2007-06-26 | Santaris Pharma As | Análogos de l-ribo-lna |
US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
DK2284269T3 (en) | 2002-11-18 | 2017-10-23 | Roche Innovation Ct Copenhagen 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 |
JP5342881B2 (ja) | 2006-01-27 | 2013-11-13 | アイシス ファーマシューティカルズ, インコーポレーテッド | 6−修飾された二環式核酸類似体 |
AU2007249349B2 (en) | 2006-05-11 | 2012-03-08 | Isis Pharmaceuticals, Inc. | 5'-Modified bicyclic nucleic acid analogs |
US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
US8278425B2 (en) | 2007-05-30 | 2012-10-02 | 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 |
CN101796062B (zh) | 2007-07-05 | 2014-07-30 | Isis制药公司 | 6-双取代双环核酸类似物 |
WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
DK2356129T3 (da) | 2008-09-24 | 2013-05-13 | Isis Pharmaceuticals Inc | Substituerede alpha-L-bicykliske nukleosider |
EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
US8846637B2 (en) | 2010-06-08 | 2014-09-30 | Isis Pharmaceuticals, Inc. | Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
JP2016528873A (ja) * | 2012-05-16 | 2016-09-23 | ラナ セラピューティクス インコーポレイテッド | 遺伝子発現を調節するための組成物及び方法 |
JP2016503300A (ja) | 2012-11-15 | 2016-02-04 | ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブRoche Innovation Center Copenhagen A/S | 抗ApoBアンチセンス複合体化合物 |
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 US US17/833,085 patent/US20220403388A1/en active Pending
- 2022-06-06 KR KR1020247000172A patent/KR20240019228A/ko unknown
- 2022-06-06 BR BR112023025676A patent/BR112023025676A2/pt unknown
- 2022-06-06 TW TW111120879A patent/TW202313976A/zh unknown
- 2022-06-06 AR ARP220101492A patent/AR126085A1/es unknown
- 2022-06-06 CN CN202280040635.7A patent/CN117441018A/zh active Pending
- 2022-06-06 WO PCT/EP2022/065298 patent/WO2022258555A1/en active Application Filing
- 2022-06-06 EP EP22732985.1A patent/EP4352222A1/en active Pending
- 2022-06-06 CA CA3222546A patent/CA3222546A1/en active Pending
- 2022-06-06 AU AU2022288115A patent/AU2022288115A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
AR126085A1 (es) | 2023-09-13 |
TW202313976A (zh) | 2023-04-01 |
EP4352222A1 (en) | 2024-04-17 |
WO2022258555A1 (en) | 2022-12-15 |
AU2022288115A1 (en) | 2023-12-07 |
US20220403388A1 (en) | 2022-12-22 |
KR20240019228A (ko) | 2024-02-14 |
CA3222546A1 (en) | 2022-12-15 |
CN117441018A (zh) | 2024-01-23 |
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