JPWO2019075419A5 - - Google Patents
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- JPWO2019075419A5 JPWO2019075419A5 JP2020542046A JP2020542046A JPWO2019075419A5 JP WO2019075419 A5 JPWO2019075419 A5 JP WO2019075419A5 JP 2020542046 A JP2020542046 A JP 2020542046A JP 2020542046 A JP2020542046 A JP 2020542046A JP WO2019075419 A5 JPWO2019075419 A5 JP WO2019075419A5
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- JP
- Japan
- Prior art keywords
- composition
- positions
- oligonucleotide
- antisense strand
- phosphate
- Prior art date
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- 108091034117 Oligonucleotide Proteins 0.000 claims description 29
- 125000003729 nucleotide group Chemical group 0.000 claims description 21
- 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 description 15
- 239000002773 nucleotide Substances 0.000 claims description 14
- 230000000692 anti-sense effect Effects 0.000 claims description 10
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims description 7
- 239000002953 phosphate buffered saline Substances 0.000 claims description 7
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 22
- 239000000546 pharmaceutical excipient Substances 0.000 claims 7
- 108091081021 Sense strand Proteins 0.000 claims 6
- 102100034671 L-lactate dehydrogenase A chain Human genes 0.000 claims 4
- 108010088350 Lactate Dehydrogenase 5 Proteins 0.000 claims 4
- 208000004777 Primary Hyperoxaluria Diseases 0.000 claims 4
- 208000008852 Hyperoxaluria Diseases 0.000 claims 2
- MUBZPKHOEPUJKR-UHFFFAOYSA-N Oxalic acid Chemical compound OC(=O)C(O)=O MUBZPKHOEPUJKR-UHFFFAOYSA-N 0.000 claims 2
- 230000002440 hepatic effect Effects 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 11
- 108091028664 Ribonucleotide Proteins 0.000 description 8
- 230000004048 modification Effects 0.000 description 8
- 238000012986 modification Methods 0.000 description 8
- 150000003013 phosphoric acid derivatives Chemical class 0.000 description 8
- 239000002336 ribonucleotide Substances 0.000 description 8
- 239000005547 deoxyribonucleotide Substances 0.000 description 7
- 229910019142 PO4 Inorganic materials 0.000 description 5
- 229910052799 carbon Inorganic materials 0.000 description 5
- 210000003494 hepatocyte Anatomy 0.000 description 5
- 239000010452 phosphate Substances 0.000 description 5
- 125000002652 ribonucleotide group Chemical group 0.000 description 5
- 238000006467 substitution reaction Methods 0.000 description 5
- 239000002253 acid Substances 0.000 description 4
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 4
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 description 3
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 description 3
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 description 3
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 description 3
- 230000015556 catabolic process Effects 0.000 description 3
- 210000004027 cell Anatomy 0.000 description 3
- 238000006731 degradation reaction Methods 0.000 description 3
- 238000009396 hybridization Methods 0.000 description 3
- 108020004707 nucleic acids Proteins 0.000 description 3
- 102000039446 nucleic acids Human genes 0.000 description 3
- 150000007523 nucleic acids Chemical class 0.000 description 3
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical class [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 description 3
- 229930024421 Adenine Natural products 0.000 description 2
- UYTPUPDQBNUYGX-UHFFFAOYSA-N Guanine Natural products O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 description 2
- 102000008088 Hepatocyte Nuclear Factors Human genes 0.000 description 2
- 108010049606 Hepatocyte Nuclear Factors Proteins 0.000 description 2
- 229960000643 adenine Drugs 0.000 description 2
- 125000004429 atom Chemical group 0.000 description 2
- 238000007385 chemical modification Methods 0.000 description 2
- 230000000295 complement effect Effects 0.000 description 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Natural products NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 2
- 229940104302 cytosine Drugs 0.000 description 2
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 2
- 210000004185 liver Anatomy 0.000 description 2
- 125000004430 oxygen atom Chemical group O* 0.000 description 2
- 150000002972 pentoses Chemical class 0.000 description 2
- 239000008363 phosphate buffer Substances 0.000 description 2
- 239000000126 substance Substances 0.000 description 2
- MGRVRXRGTBOSHW-UHFFFAOYSA-N (aminomethyl)phosphonic acid Chemical compound NCP(O)(O)=O MGRVRXRGTBOSHW-UHFFFAOYSA-N 0.000 description 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 1
- ASJSAQIRZKANQN-CRCLSJGQSA-N 2-deoxy-D-ribose Chemical compound OC[C@@H](O)[C@@H](O)CC=O ASJSAQIRZKANQN-CRCLSJGQSA-N 0.000 description 1
- 102000009027 Albumins Human genes 0.000 description 1
- 108010088751 Albumins Proteins 0.000 description 1
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 description 1
- 108010039209 Blood Coagulation Factors Proteins 0.000 description 1
- 102000015081 Blood Coagulation Factors Human genes 0.000 description 1
- 102000002004 Cytochrome P-450 Enzyme System Human genes 0.000 description 1
- 108010015742 Cytochrome P-450 Enzyme System Proteins 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 108010049003 Fibrinogen Proteins 0.000 description 1
- 102000008946 Fibrinogen Human genes 0.000 description 1
- 102100029115 Fumarylacetoacetase Human genes 0.000 description 1
- 101150111020 GLUL gene Proteins 0.000 description 1
- 101150003775 HNF1A gene Proteins 0.000 description 1
- 101150068639 Hnf4a gene Proteins 0.000 description 1
- 101000780643 Homo sapiens Protein argonaute-2 Proteins 0.000 description 1
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 description 1
- 101100114688 Mus musculus Cyp3a11 gene Proteins 0.000 description 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical group CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 description 1
- 101710163270 Nuclease Proteins 0.000 description 1
- 108091028043 Nucleic acid sequence Proteins 0.000 description 1
- 102000004160 Phosphoric Monoester Hydrolases Human genes 0.000 description 1
- 108090000608 Phosphoric Monoester Hydrolases Proteins 0.000 description 1
- 102000007584 Prealbumin Human genes 0.000 description 1
- 108010071690 Prealbumin Proteins 0.000 description 1
- 102100034207 Protein argonaute-2 Human genes 0.000 description 1
- 108010094028 Prothrombin Proteins 0.000 description 1
- 102100027378 Prothrombin Human genes 0.000 description 1
- 108091030071 RNAI Proteins 0.000 description 1
- 101100114680 Rattus norvegicus Cyp3a2 gene Proteins 0.000 description 1
- 102000007562 Serum Albumin Human genes 0.000 description 1
- 108010071390 Serum Albumin Proteins 0.000 description 1
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Natural products O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 description 1
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Natural products O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 1
- VFRROHXSMXFLSN-KCDKBNATSA-N aldehydo-D-galactose 6-phosphate Chemical compound OP(=O)(O)OC[C@@H](O)[C@H](O)[C@H](O)[C@@H](O)C=O VFRROHXSMXFLSN-KCDKBNATSA-N 0.000 description 1
- 125000004202 aminomethyl group Chemical group [H]N([H])C([H])([H])* 0.000 description 1
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 description 1
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 239000003114 blood coagulation factor Substances 0.000 description 1
- 229940012952 fibrinogen Drugs 0.000 description 1
- 108010022687 fumarylacetoacetase Proteins 0.000 description 1
- 230000009368 gene silencing by RNA Effects 0.000 description 1
- 102000005396 glutamine synthetase Human genes 0.000 description 1
- 108020002326 glutamine synthetase Proteins 0.000 description 1
- 229910052739 hydrogen Inorganic materials 0.000 description 1
- 239000001257 hydrogen Substances 0.000 description 1
- 230000005847 immunogenicity Effects 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 230000003993 interaction Effects 0.000 description 1
- CAAULPUQFIIOTL-UHFFFAOYSA-N methyl dihydrogen phosphate Chemical group COP(O)(O)=O CAAULPUQFIIOTL-UHFFFAOYSA-N 0.000 description 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 1
- 230000003278 mimic effect Effects 0.000 description 1
- 229910052757 nitrogen Inorganic materials 0.000 description 1
- 125000004433 nitrogen atom Chemical group N* 0.000 description 1
- 125000000371 nucleobase group Chemical group 0.000 description 1
- 229940039716 prothrombin Drugs 0.000 description 1
- 229910052717 sulfur Inorganic materials 0.000 description 1
- 125000004434 sulfur atom Chemical group 0.000 description 1
- 125000004055 thiomethyl group Chemical group [H]SC([H])([H])* 0.000 description 1
- 229940104230 thymidine Drugs 0.000 description 1
- 229940035893 uracil Drugs 0.000 description 1
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 description 1
- 229940045145 uridine Drugs 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023109290A JP7674420B2 (ja) | 2017-10-13 | 2023-07-03 | Ｌｄｈａの発現を阻害するための方法及び組成物 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762572403P | 2017-10-13 | 2017-10-13 | |
| US201762572398P | 2017-10-13 | 2017-10-13 | |
| US62/572,403 | 2017-10-13 | ||
| US62/572,398 | 2017-10-13 | ||
| US201862726950P | 2018-09-04 | 2018-09-04 | |
| US62/726,950 | 2018-09-04 | ||
| PCT/US2018/055735 WO2019075419A1 (en) | 2017-10-13 | 2018-10-12 | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023109290A Division JP7674420B2 (ja) | 2017-10-13 | 2023-07-03 | Ｌｄｈａの発現を阻害するための方法及び組成物 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2020536587A JP2020536587A (ja) | 2020-12-17 |
| JP2020536587A5 JP2020536587A5 (https=) | 2023-03-01 |
| JPWO2019075419A5 true JPWO2019075419A5 (https=) | 2023-03-01 |
| JP7308213B2 JP7308213B2 (ja) | 2023-07-13 |
Family
ID=66101691
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020542046A Active JP7308213B2 (ja) | 2017-10-13 | 2018-10-12 | Ｌｄｈａの発現を阻害するための方法及び組成物 |
| JP2023109290A Active JP7674420B2 (ja) | 2017-10-13 | 2023-07-03 | Ｌｄｈａの発現を阻害するための方法及び組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023109290A Active JP7674420B2 (ja) | 2017-10-13 | 2023-07-03 | Ｌｄｈａの発現を阻害するための方法及び組成物 |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US11286488B2 (https=) |
| EP (2) | EP4265261A3 (https=) |
| JP (2) | JP7308213B2 (https=) |
| KR (2) | KR102609396B1 (https=) |
| CN (3) | CN118530989A (https=) |
| AU (1) | AU2018346971B2 (https=) |
| CA (1) | CA3078933A1 (https=) |
| ES (1) | ES2955045T3 (https=) |
| HR (1) | HRP20231063T1 (https=) |
| HU (1) | HUE063026T2 (https=) |
| IL (1) | IL273875B2 (https=) |
| MX (1) | MX2020003836A (https=) |
| PL (1) | PL3679141T3 (https=) |
| RS (1) | RS64483B1 (https=) |
| WO (1) | WO2019075419A1 (https=) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2015330670B2 (en) | 2014-10-10 | 2022-01-06 | Novo Nordisk Health Care Ag | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| EP3277811B1 (en) | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric sirna |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| HRP20231063T1 (hr) | 2017-10-13 | 2023-12-22 | Novo Nordisk Health Care Ag | Postupci i pripravci namijenjeni inhibiranju eksprimiranja ldha |
| SI3684377T1 (sl) * | 2017-10-20 | 2023-05-31 | Dicerna Pharmaceuticals, Inc. | Postopki zdravljenja okužbe s hepatitisom B |
| WO2019143621A1 (en) | 2018-01-16 | 2019-07-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting aldh2 expression |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| MX2022001710A (es) | 2019-08-09 | 2022-05-10 | Univ Massachusetts | Oligonucleótidos modificados químicamente dirigidos a los snp. |
| JP2022545118A (ja) * | 2019-08-23 | 2022-10-25 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| WO2021067744A1 (en) * | 2019-10-02 | 2021-04-08 | Dicerna Pharmaceuticals, Inc. | Chemical modifications of small interfering rna with minimal fluorine content |
| EP4121534A1 (en) | 2020-03-18 | 2023-01-25 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant |
| US20250205203A1 (en) * | 2020-05-18 | 2025-06-26 | Chinook Therapeutics Canada, Inc. | Substituted pyrazolyl compounds and methods of use thereof |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| EP4244356A1 (en) * | 2020-11-13 | 2023-09-20 | Dicerna Pharmaceuticals, Inc. | Chemical modifications for inhibiting expression of aldh2 |
| CA3207897A1 (en) | 2021-03-19 | 2022-09-22 | Ralf Eisenhuth | Improved oligonucleotide synthesis |
| US20250282813A1 (en) | 2021-04-09 | 2025-09-11 | Bachem Holding Ag | Pseudo solid phase protecting group and methods for the synthesis of oligonucleotides and oligonucleotide conjugates |
| WO2022223515A2 (en) * | 2021-04-19 | 2022-10-27 | Novo Nordisk A/S | Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression |
| US20240287512A1 (en) * | 2021-05-11 | 2024-08-29 | Dicerna Pharmaceuticals, Inc. | Lipid conjugation for targeting neurons of the central nervous system |
| JP2024523237A (ja) | 2021-06-18 | 2024-06-28 | ホンジーン バイオテック コーポレイション | 官能化されたｎ－アセチルガラクトサミンヌクレオシド |
| WO2023003805A1 (en) | 2021-07-19 | 2023-01-26 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder |
| JP2024532019A (ja) * | 2021-08-25 | 2024-09-05 | ノボ ノルディスク エー／エス | アルファ－１アンチトリプシン発現を阻害するための組成物及び方法 |
| JP2024532271A (ja) | 2021-08-30 | 2024-09-05 | ホンジーン バイオテック コーポレイション | 官能化ｎ－アセチルガラクトサミンアナログ |
| AR127260A1 (es) * | 2021-10-05 | 2024-01-03 | Dicerna Pharmaceuticals Inc | Composiciones y métodos para inhibir el receptor de melanocortina 2 y la expresión del citocromo p450 11b |
| WO2023102469A2 (en) * | 2021-12-01 | 2023-06-08 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating apoc3 expression |
| WO2023114746A1 (en) | 2021-12-15 | 2023-06-22 | Hongene Biotech Corporation | Functionalized n-acetylgalactosamine analogs |
| US20260001902A1 (en) | 2022-09-19 | 2026-01-01 | Bachem Holding Ag | Improved oligonucleotide synthesis |
| JP2025531341A (ja) * | 2022-09-19 | 2025-09-19 | カイロンノヴァ （シャーメン） バイオファーマ カンパニー，リミテッド | 炭水化物－オリゴヌクレオチド複合体、薬物組成物および治療への応用 |
| WO2024083746A1 (en) | 2022-10-17 | 2024-04-25 | Bachem Holding Ag | Method and composition for oligonucleotide synthesis |
| WO2024118503A1 (en) | 2022-11-28 | 2024-06-06 | Hongene Biotech Corporation | Functionalized n-acetylgalactosamine analogs |
| EP4695265A1 (en) | 2023-10-16 | 2026-02-18 | Bachem Holding AG | Method and composition for oligonucleotide synthesis |
| TW202602463A (zh) | 2024-03-22 | 2026-01-16 | 日商武田藥品工業股份有限公司 | 用於抑制細胞色素ｐ４５０家族７次家族ａ成員１（ｃｙｐ７ａ１）表現之組成物及方法 |
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| AU2005222965B8 (en) | 2004-03-15 | 2010-07-01 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
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| JP2012504389A (ja) | 2008-09-22 | 2012-02-23 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法 |
| US20110288147A1 (en) | 2008-09-22 | 2011-11-24 | Bob Dale Brown | Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop |
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| AU2010223919B2 (en) | 2009-03-13 | 2016-03-31 | Les Laboratoires Servier | Methods and compositions for cell-proliferation-related disorders |
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| PL2576580T3 (pl) | 2010-05-28 | 2017-03-31 | F.Hoffmann-La Roche Ag | Zmniejszenie poziomu mleczanu i zwiększenie wytwarzania polipeptydu przez obniżenie poziomu ekspresji dehydrogenazy mleczanowej i kinazy dehydrogenazy pirogronianowej |
| JP2014518626A (ja) | 2011-05-18 | 2014-08-07 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | 被験者が慢性腎疾患を有する、または発症するリスクがあるかどうかを決定するための方法 |
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| AR090906A1 (es) | 2012-05-02 | 2014-12-17 | Merck Sharp & Dohme | Conjugados que contienen tetragalnac y procedimientos para la administracion de oligonucleotidos |
| AU2015330670B2 (en) | 2014-10-10 | 2022-01-06 | Novo Nordisk Health Care Ag | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| JP7105065B2 (ja) | 2014-12-15 | 2022-07-22 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | リガンド修飾二本鎖核酸 |
| JP2018500905A (ja) | 2014-12-18 | 2018-01-18 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | ナノチューブを使用した核酸ポリメラーゼ立体構造変化の検出 |
| HK1252852A1 (zh) * | 2015-05-29 | 2019-06-06 | Arrowhead Pharmaceuticals, Inc. | 阻止HIF2α基因表达的组合物及方法 |
| HRP20231063T1 (hr) | 2017-10-13 | 2023-12-22 | Novo Nordisk Health Care Ag | Postupci i pripravci namijenjeni inhibiranju eksprimiranja ldha |
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2018
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- 2018-10-12 EP EP23172235.6A patent/EP4265261A3/en active Pending
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- 2018-10-12 KR KR1020237041035A patent/KR20230169413A/ko active Pending
- 2018-10-12 WO PCT/US2018/055735 patent/WO2019075419A1/en not_active Ceased
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2020
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