IL302558A - Oligonucleotide compositions and methods thereof - Google Patents
Oligonucleotide compositions and methods thereofInfo
- Publication number
- IL302558A IL302558A IL302558A IL30255823A IL302558A IL 302558 A IL302558 A IL 302558A IL 302558 A IL302558 A IL 302558A IL 30255823 A IL30255823 A IL 30255823A IL 302558 A IL302558 A IL 302558A
- Authority
- IL
- Israel
- Prior art keywords
- oligonucleotide
- nucleic acid
- target
- composition
- oligonucleotides
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title claims 204
- 239000000203 mixture Substances 0.000 title claims 64
- 238000000034 method Methods 0.000 title claims 21
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 88
- 108020004707 nucleic acids Proteins 0.000 claims 60
- 102000039446 nucleic acids Human genes 0.000 claims 60
- 150000007523 nucleic acids Chemical class 0.000 claims 60
- 230000004048 modification Effects 0.000 claims 57
- 238000012986 modification Methods 0.000 claims 57
- 235000000346 sugar Nutrition 0.000 claims 49
- 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 claims 48
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 44
- 229960005305 adenosine Drugs 0.000 claims 44
- 239000002777 nucleoside Substances 0.000 claims 33
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 32
- 150000008163 sugars Chemical class 0.000 claims 17
- 230000000295 complement effect Effects 0.000 claims 16
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims 13
- 102000055025 Adenosine deaminases Human genes 0.000 claims 13
- 150000008300 phosphoramidites Chemical class 0.000 claims 11
- 229910052698 phosphorus Inorganic materials 0.000 claims 11
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 10
- 239000011574 phosphorus Substances 0.000 claims 10
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 8
- 150000003839 salts Chemical group 0.000 claims 8
- 239000008186 active pharmaceutical agent Substances 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 208000035475 disorder Diseases 0.000 claims 4
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims 4
- 229920001184 polypeptide Polymers 0.000 claims 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 4
- 239000002253 acid Substances 0.000 claims 3
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical group [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims 3
- 229910052760 oxygen Inorganic materials 0.000 claims 3
- 239000001301 oxygen Substances 0.000 claims 3
- 101000865408 Homo sapiens Double-stranded RNA-specific adenosine deaminase Proteins 0.000 claims 2
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims 2
- 229930010555 Inosine Natural products 0.000 claims 2
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims 2
- 125000001931 aliphatic group Chemical group 0.000 claims 2
- 125000002619 bicyclic group Chemical group 0.000 claims 2
- 229910052799 carbon Inorganic materials 0.000 claims 2
- 238000006243 chemical reaction Methods 0.000 claims 2
- 229960003786 inosine Drugs 0.000 claims 2
- 125000002950 monocyclic group Chemical group 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 229910052757 nitrogen Inorganic materials 0.000 claims 2
- 125000004433 nitrogen atom Chemical group N* 0.000 claims 2
- 125000003835 nucleoside group Chemical group 0.000 claims 2
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 2
- 125000003367 polycyclic group Chemical group 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 2
- 229930024421 Adenine Natural products 0.000 claims 1
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 1
- 101100460700 Aspergillus sp. (strain MF297-2) notG gene Proteins 0.000 claims 1
- 102100029791 Double-stranded RNA-specific adenosine deaminase Human genes 0.000 claims 1
- 101100001674 Emericella variicolor andI gene Proteins 0.000 claims 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 1
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims 1
- 229960000643 adenine Drugs 0.000 claims 1
- 125000004429 atom Chemical group 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- 230000008878 coupling Effects 0.000 claims 1
- 238000010168 coupling process Methods 0.000 claims 1
- 238000005859 coupling reaction Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000005842 heteroatom Chemical group 0.000 claims 1
- 229910052739 hydrogen Inorganic materials 0.000 claims 1
- 239000001257 hydrogen Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 1
- -1 ora phosphoramidite Chemical class 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 229920006395 saturated elastomer Polymers 0.000 claims 1
- 229910052717 sulfur Inorganic materials 0.000 claims 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
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/322—2'-R Modification
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- 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
- C12N2320/33—Alteration of splicing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- 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)
Landscapes
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063111079P | 2020-11-08 | 2020-11-08 | |
| US202163175036P | 2021-04-14 | 2021-04-14 | |
| US202163188415P | 2021-05-13 | 2021-05-13 | |
| US202163196178P | 2021-06-02 | 2021-06-02 | |
| US202163248520P | 2021-09-26 | 2021-09-26 | |
| PCT/US2021/058495 WO2022099159A1 (fr) | 2020-11-08 | 2021-11-08 | Compositions d'oligonucléotides et procédés associés |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL302558A true IL302558A (en) | 2023-07-01 |
Family
ID=81456778
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL302558A IL302558A (en) | 2020-11-08 | 2021-11-08 | Oligonucleotide compositions and methods thereof |
Country Status (8)
| Country | Link |
|---|---|
| EP (1) | EP4240849A1 (fr) |
| JP (1) | JP2023548584A (fr) |
| KR (1) | KR20230118716A (fr) |
| AU (1) | AU2021373062A1 (fr) |
| CA (1) | CA3197311A1 (fr) |
| IL (1) | IL302558A (fr) |
| MX (1) | MX2023005326A (fr) |
| WO (1) | WO2022099159A1 (fr) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2017281497B2 (en) | 2016-06-22 | 2023-04-06 | Proqr Therapeutics Ii B.V. | Single-stranded RNA-editing oligonucleotides |
| EP3544987A4 (fr) | 2016-11-23 | 2020-11-18 | Wave Life Sciences Ltd. | Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides |
| TW201904587A (zh) | 2017-06-02 | 2019-02-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
| WO2018237194A1 (fr) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Composés, compositions et procédés de synthèse |
| AU2018316159A1 (en) | 2017-08-08 | 2020-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3684755A4 (fr) | 2017-09-18 | 2022-03-02 | Wave Life Sciences Ltd. | Technologies de préparation d'oligonucléotides |
| EP4363574A1 (fr) * | 2021-06-29 | 2024-05-08 | Korro Bio, Inc. | Procédés et compositions pour édition médiée par adar |
| AU2022349529A1 (en) * | 2021-09-26 | 2024-05-02 | Wave Life Sciences Ltd. | Compositions for editing mecp2 transcripts and methods thereof |
| WO2023069603A1 (fr) * | 2021-10-22 | 2023-04-27 | Korro Bio, Inc. | Procédés et compositions pour perturber l'interaction de la protéine nrf2-keap1 par l'édition d'arn à médiation adar |
| WO2023152371A1 (fr) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie |
| WO2024013361A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides pour édition d'arn médiée par adar et leur utilisation |
| WO2024013360A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar |
| WO2024017817A1 (fr) * | 2022-07-18 | 2024-01-25 | F. Hoffmann-La Roche Ag | Oligonucléotide d'édition |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| WO2024110565A1 (fr) | 2022-11-24 | 2024-05-30 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement de l'hémochromatose hfe héréditaire |
| WO2024114908A1 (fr) | 2022-11-30 | 2024-06-06 | Eberhard Karls Universität Tübingen | Oligonucléotides antisens chimiquement modifiés (asos) et compositions les comprenant pour l'édition d'arn |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| WO2024121373A1 (fr) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire |
| CN116444593B (zh) * | 2023-04-28 | 2023-12-26 | 南通大学 | 一种7-脱氮鸟苷亚磷酰胺单体的合成方法 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014074597A1 (fr) * | 2012-11-06 | 2014-05-15 | President And Fellows Of Harvard College | Compositions et procédés se rapportant à des nanostructures d'acides nucléiques complexes |
| NZ751483A (en) * | 2016-09-01 | 2022-07-01 | Proqr Therapeutics Ii Bv | Chemically modified single-stranded rna-editing oligonucleotides |
| CA3063739A1 (fr) * | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systemes, procedes et compositions d'edition ciblee d'acides nucleiques |
| WO2018237194A1 (fr) * | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Composés, compositions et procédés de synthèse |
-
2021
- 2021-11-08 CA CA3197311A patent/CA3197311A1/fr active Pending
- 2021-11-08 AU AU2021373062A patent/AU2021373062A1/en active Pending
- 2021-11-08 JP JP2023527250A patent/JP2023548584A/ja active Pending
- 2021-11-08 IL IL302558A patent/IL302558A/en unknown
- 2021-11-08 WO PCT/US2021/058495 patent/WO2022099159A1/fr active Application Filing
- 2021-11-08 MX MX2023005326A patent/MX2023005326A/es unknown
- 2021-11-08 KR KR1020237019215A patent/KR20230118716A/ko unknown
- 2021-11-08 EP EP21890258.3A patent/EP4240849A1/fr active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4240849A1 (fr) | 2023-09-13 |
| AU2021373062A1 (en) | 2023-06-08 |
| CA3197311A1 (fr) | 2022-05-12 |
| KR20230118716A (ko) | 2023-08-11 |
| MX2023005326A (es) | 2023-07-31 |
| JP2023548584A (ja) | 2023-11-17 |
| WO2022099159A1 (fr) | 2022-05-12 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL302558A (en) | Oligonucleotide compositions and methods thereof | |
| US5672697A (en) | Nucleoside 5'-methylene phosphonates | |
| JP2020522510A5 (fr) | ||
| US5434257A (en) | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages | |
| KR0155574B1 (ko) | 주쇄가 변성된 올리고뉴클레오티드 유사체 | |
| DE69934227T2 (de) | Auf Beta-Arabinose und dessen Analoga basierte Antisense-Oligonukleotide | |
| CA2416631C (fr) | Composes de fixation d'acides nucleiques contenant des analogues pyrazolo[3,4-d]pyrimidine de purine 2,6-diamine et leurs utilisations | |
| CN1118140A (zh) | 为提高在酸性pH的稳定性而改性的低聚核苷酸 | |
| Thibaudeau et al. | Quantitation of the pD Dependent Thermodynamics of the N⇄ S Pseudorotational Equilibrium of the Pentofuranose Moiety in Nucleosides Gives a Direct Measurement of the Strength of the Tunable Anomeric Effect and the p K a of the Nucleobase | |
| EP0679657A2 (fr) | Nucléosides et oligonucléotides contenant des groupes 2'-éther | |
| JPWO2021071858A5 (fr) | ||
| RU2016146819A (ru) | Композиции и способы модулирования экспрессии рецептора гормона роста | |
| EP0476071A1 (fr) | Oligonucleotides resistant a l'exonuclease, et procedes pour leur preparation | |
| DE102008054480A1 (de) | Direktsequenzierung durch ATP-Freisetzung | |
| PL216525B1 (pl) | 5'-O-[(N-acylo)amidoditiofosforano] nukleozydy oraz sposób wytwarzania 5'-O-[(N-acylo)amidofosforano]-,5'-O-[(N-acylo)amidotiofosforano]-, 5'-O-[(N-acylo)amidoditiofosforano]nukleozydów | |
| JP2021090458A5 (fr) | ||
| JP2015513561A (ja) | 三環ヌクレオシドおよびこれらから調製したオリゴマー化合物 | |
| RU2019113673A (ru) | Новые бициклические нуклеозиды и олигомеры, полученные из них | |
| Brito-Arias | N-glycosides | |
| Seela et al. | Synthesis of 4‐Substituted 1H‐Benzimidazole 2′‐Deoxyribonucleosides and Utility of the 4‐Nitro Compound as Universal Base | |
| WO2006013203A2 (fr) | Promedicaments actives par des polymerases d'adn dependantes de l'arn | |
| Edgell et al. | Evidence of independent gene duplications during the evolution of archaeal and eukaryotic family B DNA polymerases. | |
| Beck et al. | A double-headed nucleotide with two cytosines: DNA with condensed information and improved duplex stability | |
| JP2013520395A (ja) | 治療、診断、ｇ‐テトラド形成オリゴヌクレオシド及びアプタマーといった生物学的応用のための新規修飾を取り入れたアラ‐２’‐ｏ‐メチル‐ヌクレオシド、当該ホスホラミダイト及びオリゴヌクレオチドの合成 | |
| Kaiser et al. | Acyclic Nucleoside Phosphonates Bearing (R)‐or (S)‐9‐[3‐Hydroxy‐2‐(phosphonoethoxy) propyl](HPEP) Moiety as Monomers for the Synthesis of Modified Oligonucleotides |