BR112022006207A2 - Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composição - Google Patents
Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composiçãoInfo
- Publication number
- BR112022006207A2 BR112022006207A2 BR112022006207A BR112022006207A BR112022006207A2 BR 112022006207 A2 BR112022006207 A2 BR 112022006207A2 BR 112022006207 A BR112022006207 A BR 112022006207A BR 112022006207 A BR112022006207 A BR 112022006207A BR 112022006207 A2 BR112022006207 A2 BR 112022006207A2
- Authority
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- Brazil
- Prior art keywords
- oligonucleotide
- composition
- level
- reduce
- cell
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 14
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 7
- 108090000623 proteins and genes Proteins 0.000 title abstract 6
- 239000000203 mixture Substances 0.000 title abstract 5
- 108010016626 Dipeptides Proteins 0.000 title abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 102000004169 proteins and genes Human genes 0.000 title abstract 2
- 238000013518 transcription Methods 0.000 title abstract 2
- 230000035897 transcription Effects 0.000 title abstract 2
- 102000043334 C9orf72 Human genes 0.000 abstract 2
- 108700030955 C9orf72 Proteins 0.000 abstract 2
- 101150014718 C9orf72 gene Proteins 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 201000011240 Frontotemporal dementia 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
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder 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
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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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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/314—Phosphoramidates
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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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- 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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- 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
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
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- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Neurosurgery (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Neurology (AREA)
- Pharmacology & Pharmacy (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Abstract
OLIGONUCLEOTÍDEO, COMPOSIÇÃO DE OLIGONUCLEOTÍDEO, COMPOSIÇÃO FARMACÊUTICA, MÉTODO, MÉTODO PARA DIMINUIR A ATIVIDADE, EXPRESSÃO E/OU NÍVEL DE UM GENE ALVO OU SEU PRODUTO DE GENE EM UMA CÉLULA, MÉTODO PARA REDUZIR FOCOS EM UMA POPULAÇÃO DE CÉLULAS, MÉTODO PARA REDUZIR O NÍVEL DE UMA PROTEÍNA DE REPETIÇÃO DE DIPEPTÍDEO (DPR), MÉTODO PARA KNOCKDOWN PREFERENCIAL DE UMA TRANSCRIÇÃO DE RNA, E MÉTODO PARA PREPARAR UM OLIGONUCLEOTÍDEO OU COMPOSIÇÃO. Entre outros aspectos, a presente revelação fornece oligonucleotídeos C9orf72, composições e métodos de uso dos mesmos. Em algumas modalidades, a presente revelação fornece métodos para tratar afecções, distúrbios ou doenças associados a C9orf72, tais como esclerose lateral amiotrófica e demência frontotemporal.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962911340P | 2019-10-06 | 2019-10-06 | |
| US202062983736P | 2020-03-01 | 2020-03-01 | |
| PCT/US2020/032244 WO2020227691A2 (en) | 2019-05-09 | 2020-05-08 | Oligonucleotide compositions and methods of use thereof |
| US202063069704P | 2020-08-24 | 2020-08-24 | |
| PCT/US2020/054307 WO2021071788A2 (en) | 2019-10-06 | 2020-10-05 | Oligonucleotide compositions and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022006207A2 true BR112022006207A2 (pt) | 2022-07-26 |
Family
ID=75438309
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022006207A BR112022006207A2 (pt) | 2019-10-06 | 2020-10-05 | Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composição |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240175016A1 (pt) |
| EP (1) | EP4037695A4 (pt) |
| JP (1) | JP2022550915A (pt) |
| KR (1) | KR20220070324A (pt) |
| CN (1) | CN114585370A (pt) |
| AU (1) | AU2020363344A1 (pt) |
| BR (1) | BR112022006207A2 (pt) |
| CA (1) | CA3156176A1 (pt) |
| IL (1) | IL291974A (pt) |
| MX (1) | MX2022004102A (pt) |
| TW (1) | TW202126810A (pt) |
| WO (1) | WO2021071788A2 (pt) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| EP4219516A3 (en) | 2012-07-13 | 2024-01-10 | Wave Life Sciences Ltd. | Chiral control |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MA43822A (fr) | 2016-03-13 | 2018-11-28 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
| JP7296882B2 (ja) | 2016-11-23 | 2023-06-23 | ウェイブ ライフ サイエンシズ リミテッド | ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法 |
| JP2020524485A (ja) | 2017-06-02 | 2020-08-20 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物及びその使用方法 |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CN111051281A (zh) | 2017-06-21 | 2020-04-21 | 波涛生命科学有限公司 | 用于合成的化合物、组合物和方法 |
| WO2019032612A1 (en) | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED METHODS |
| JP7472018B2 (ja) | 2017-09-18 | 2024-04-22 | ウェーブ ライフ サイエンシーズ リミテッド | オリゴヌクレオチド調製のための技術 |
| US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2024013361A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2942515A1 (en) * | 2014-03-18 | 2015-09-24 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| ES2791995T3 (es) * | 2015-04-16 | 2020-11-06 | Ionis Pharmaceuticals Inc | Composiciones para modular la expresión de C90RF72 |
| WO2019032612A1 (en) * | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED METHODS |
| CA3139513A1 (en) * | 2019-05-09 | 2020-11-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
-
2020
- 2020-10-05 IL IL291974A patent/IL291974A/en unknown
- 2020-10-05 JP JP2022520976A patent/JP2022550915A/ja active Pending
- 2020-10-05 EP EP20875598.3A patent/EP4037695A4/en active Pending
- 2020-10-05 KR KR1020227015125A patent/KR20220070324A/ko unknown
- 2020-10-05 CA CA3156176A patent/CA3156176A1/en active Pending
- 2020-10-05 MX MX2022004102A patent/MX2022004102A/es unknown
- 2020-10-05 CN CN202080069266.5A patent/CN114585370A/zh active Pending
- 2020-10-05 US US17/766,680 patent/US20240175016A1/en active Pending
- 2020-10-05 WO PCT/US2020/054307 patent/WO2021071788A2/en active Application Filing
- 2020-10-05 AU AU2020363344A patent/AU2020363344A1/en active Pending
- 2020-10-05 BR BR112022006207A patent/BR112022006207A2/pt not_active Application Discontinuation
- 2020-10-06 TW TW109134610A patent/TW202126810A/zh unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021071788A2 (en) | 2021-04-15 |
| KR20220070324A (ko) | 2022-05-30 |
| CN114585370A (zh) | 2022-06-03 |
| EP4037695A2 (en) | 2022-08-10 |
| AU2020363344A1 (en) | 2022-05-26 |
| EP4037695A4 (en) | 2024-03-27 |
| IL291974A (en) | 2022-06-01 |
| JP2022550915A (ja) | 2022-12-05 |
| TW202126810A (zh) | 2021-07-16 |
| US20240175016A1 (en) | 2024-05-30 |
| MX2022004102A (es) | 2022-04-26 |
| WO2021071788A3 (en) | 2021-05-14 |
| CA3156176A1 (en) | 2021-04-15 |
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