PL3507366T3 - Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna - Google Patents
Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rnaInfo
- Publication number
- PL3507366T3 PL3507366T3 PL17771348T PL17771348T PL3507366T3 PL 3507366 T3 PL3507366 T3 PL 3507366T3 PL 17771348 T PL17771348 T PL 17771348T PL 17771348 T PL17771348 T PL 17771348T PL 3507366 T3 PL3507366 T3 PL 3507366T3
- Authority
- PL
- Poland
- Prior art keywords
- uniconious
- chemically modified
- rna editing
- editing oligonucleotides
- oligonucleotides
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title 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 title 1
Classifications
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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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
- A61P11/06—Antiasthmatics
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- A—HUMAN NECESSITIES
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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- A61P7/06—Antianaemics
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- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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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
- C12N2310/11—Antisense
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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
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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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
- C12N2310/35—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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- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Public Health (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Physical Education & Sports Medicine (AREA)
- Pulmonology (AREA)
- Psychology (AREA)
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Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1614858.7A GB201614858D0 (en) | 2016-09-01 | 2016-09-01 | Modified Bulgeomers |
| GBGB1616374.3A GB201616374D0 (en) | 2016-09-27 | 2016-09-27 | Modified bulgeomers |
| GBGB1621467.8A GB201621467D0 (en) | 2016-12-16 | 2016-12-16 | Modified bulgeomers |
| GBGB1703034.7A GB201703034D0 (en) | 2017-02-24 | 2017-02-24 | Modified bulgeomers |
| GBGB1707508.6A GB201707508D0 (en) | 2017-05-10 | 2017-05-10 | Modified bulgeomers |
| EP17771348.4A EP3507366B1 (en) | 2016-09-01 | 2017-08-31 | Chemically modified single-stranded rna-editing oligonucleotides |
| PCT/EP2017/071912 WO2018041973A1 (en) | 2016-09-01 | 2017-08-31 | Chemically modified single-stranded rna-editing oligonucleotides |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3507366T3 true PL3507366T3 (pl) | 2021-05-04 |
Family
ID=59923385
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL17771348T PL3507366T3 (pl) | 2016-09-01 | 2017-08-31 | Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US10941402B2 (pl) |
| EP (1) | EP3507366B1 (pl) |
| JP (1) | JP7244922B2 (pl) |
| KR (1) | KR102501980B1 (pl) |
| CN (1) | CN110352244B (pl) |
| AU (1) | AU2017320901C1 (pl) |
| CA (1) | CA3035293A1 (pl) |
| DK (1) | DK3507366T3 (pl) |
| ES (1) | ES2837076T3 (pl) |
| IL (1) | IL264923B2 (pl) |
| NZ (1) | NZ751483A (pl) |
| PL (1) | PL3507366T3 (pl) |
| PT (1) | PT3507366T (pl) |
| WO (1) | WO2018041973A1 (pl) |
| ZA (1) | ZA201901016B (pl) |
Families Citing this family (104)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3230445B1 (en) * | 2014-12-12 | 2024-01-24 | Tod M. Woolf | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
| EP3712269A1 (en) | 2014-12-17 | 2020-09-23 | ProQR Therapeutics II B.V. | Targeted rna editing |
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 十分に安定化された非対称ｓｉｒｎａ |
| EP3334499A4 (en) | 2015-08-14 | 2019-04-17 | University of Massachusetts | BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES |
| HK1258758A1 (zh) | 2016-01-31 | 2019-11-22 | University Of Massachusetts | 分支的寡核苷酸 |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| US10941402B2 (en) * | 2016-09-01 | 2021-03-09 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded RNA-editing oligonucleotides |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| GB2574769A (en) | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
| WO2018237245A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| IL316453A (en) * | 2017-10-06 | 2024-12-01 | Univ Oregon Health & Science | RNA editing agents and methods |
| US20210079393A1 (en) | 2018-02-14 | 2021-03-18 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for rna editing |
| CN112601816B (zh) * | 2018-05-11 | 2024-12-17 | 比姆医疗股份有限公司 | 使用可编程碱基编辑器系统遏止病原性突变的方法 |
| KR20250134703A (ko) * | 2018-05-11 | 2025-09-11 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법 |
| AU2019266326A1 (en) | 2018-05-11 | 2020-11-26 | Beam Therapeutics Inc. | Methods of editing single nucleotide polymorphism using programmable base editor systems |
| GB201808146D0 (en) * | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| US20220073915A1 (en) * | 2018-06-29 | 2022-03-10 | Eberhard-Karls-Universität-Tübingen | Artificial nucleic acids for rna editing |
| KR20210093227A (ko) | 2018-08-10 | 2021-07-27 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 변형된 올리고뉴클레오티드 |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| EP3844274A1 (en) | 2018-08-28 | 2021-07-07 | Roche Innovation Center Copenhagen A/S | Neoantigen engineering using splice modulating compounds |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| CA3115864A1 (en) * | 2018-10-12 | 2020-04-16 | Peking University | Methods and compositions for editing rnas |
| EP3911747A4 (en) | 2019-01-18 | 2023-05-24 | University Of Massachusetts | DYNAMIC PHARMACOKINETICS-MODIFYING ANCHORS |
| JP2022519184A (ja) | 2019-01-22 | 2022-03-22 | コロ バイオ， インコーポレイテッド | Ｒｎａ編集オリゴヌクレオチド及びその使用 |
| CN113573717A (zh) * | 2019-01-22 | 2021-10-29 | 科罗生物公司 | Rna编辑的寡核苷酸及其用途 |
| CA3126947A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| EP3918067A1 (en) | 2019-01-28 | 2021-12-08 | ProQR Therapeutics II B.V. | Rna-editing oligonucleotides for the treatment of usher syndrome |
| GB201901873D0 (en) | 2019-02-11 | 2019-04-03 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for nucleic acid editing |
| JP2022528840A (ja) | 2019-03-26 | 2022-06-16 | ユニバーシティ・オブ・マサチューセッツ | 安定性が増加した修飾オリゴヌクレオチド |
| GB201904709D0 (en) | 2019-04-03 | 2019-05-15 | Proqr Therapeutics Ii Bv | Chemically modified oligonucleotides |
| WO2020211780A1 (en) | 2019-04-15 | 2020-10-22 | Edigene Inc. | Methods and compositions for editing rnas |
| GB201905732D0 (en) | 2019-04-24 | 2019-06-05 | Proqr Therapeutics Ii Bv | RNA editing inhibitors and methods of use |
| CA3140438A1 (en) * | 2019-06-05 | 2020-12-10 | Masatora Fukuda | Stable target-editing guide rna having chemically modified nucleic acid introduced thereinto |
| CN113994000B (zh) * | 2019-06-13 | 2025-03-25 | ProQR治疗上市公司Ⅱ | 包括胞苷类似物的反义rna编辑寡核苷酸 |
| WO2021008447A1 (en) | 2019-07-12 | 2021-01-21 | Peking University | Targeted rna editing by leveraging endogenous adar using engineered rnas |
| JPWO2021020550A1 (pl) | 2019-08-01 | 2021-02-04 | ||
| AU2020329155A1 (en) | 2019-08-09 | 2022-03-10 | University Of Massachusetts | Chemically modified oligonucleotides targeting SNPs |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| CA3154768A1 (en) * | 2019-10-06 | 2021-04-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US20240148772A1 (en) * | 2019-11-01 | 2024-05-09 | Tevard Biosciences, Inc. | Methods and compositions for treating a premature termination codon-mediated disorder |
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