JPWO2020041769A5 - - Google Patents
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- Publication number
- JPWO2020041769A5 JPWO2020041769A5 JP2021509991A JP2021509991A JPWO2020041769A5 JP WO2020041769 A5 JPWO2020041769 A5 JP WO2020041769A5 JP 2021509991 A JP2021509991 A JP 2021509991A JP 2021509991 A JP2021509991 A JP 2021509991A JP WO2020041769 A5 JPWO2020041769 A5 JP WO2020041769A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotides
- optionally
- oligonucleotide
- positions
- antisense strand
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 239000002773 nucleotide Substances 0.000 claims description 150
- 125000003729 nucleotide group Chemical group 0.000 claims description 150
- 229920000272 Oligonucleotide Polymers 0.000 claims description 122
- 230000000692 anti-sense Effects 0.000 claims description 76
- 230000004048 modification Effects 0.000 claims description 61
- 238000006011 modification reaction Methods 0.000 claims description 61
- 150000007523 nucleic acids Chemical group 0.000 claims description 59
- 229920000972 Sense strand Polymers 0.000 claims description 55
- 108020004707 nucleic acids Proteins 0.000 claims description 52
- 150000001875 compounds Chemical class 0.000 claims description 27
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 23
- 201000010099 disease Diseases 0.000 claims description 8
- 230000002209 hydrophobic Effects 0.000 claims description 8
- 125000005647 linker group Chemical group 0.000 claims description 8
- 229920000160 (ribonucleotides)n+m Polymers 0.000 claims description 4
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims description 4
- 108020004999 Messenger RNA Proteins 0.000 claims description 4
- 125000000217 alkyl group Chemical group 0.000 claims description 4
- -1 alkylene phosphonate Chemical compound 0.000 claims description 4
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 4
- 229920002106 messenger RNA Polymers 0.000 claims description 4
- 235000021317 phosphate Nutrition 0.000 claims description 4
- 230000003612 virological Effects 0.000 claims description 4
- ABLZXFCXXLZCGV-UHFFFAOYSA-L CHEBI:8154 Chemical class [O-]P([O-])=O ABLZXFCXXLZCGV-UHFFFAOYSA-L 0.000 claims description 2
- 229940088594 Vitamin Drugs 0.000 claims description 2
- BQMQLJQPTQPEOV-UHFFFAOYSA-M [O-]P(=O)OC=C Chemical compound [O-]P(=O)OC=C BQMQLJQPTQPEOV-UHFFFAOYSA-M 0.000 claims description 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K [O-]P([O-])([O-])=O Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims description 2
- 125000003342 alkenyl group Chemical group 0.000 claims description 2
- 150000001408 amides Chemical class 0.000 claims description 2
- 150000001413 amino acids Chemical class 0.000 claims description 2
- 125000003118 aryl group Chemical group 0.000 claims description 2
- OKTJSMMVPCPJKN-UHFFFAOYSA-N carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims description 2
- 150000001841 cholesterols Chemical class 0.000 claims description 2
- 230000000295 complement Effects 0.000 claims description 2
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims description 2
- 150000002148 esters Chemical class 0.000 claims description 2
- LYCAIKOWRPUZTN-UHFFFAOYSA-N glycol Chemical group OCCO LYCAIKOWRPUZTN-UHFFFAOYSA-N 0.000 claims description 2
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 2
- 229910052757 nitrogen Inorganic materials 0.000 claims description 2
- 125000004433 nitrogen atoms Chemical group N* 0.000 claims description 2
- 125000004430 oxygen atoms Chemical group O* 0.000 claims description 2
- 239000010452 phosphate Substances 0.000 claims description 2
- 150000008298 phosphoramidates Chemical class 0.000 claims description 2
- 150000003013 phosphoric acid derivatives Chemical class 0.000 claims description 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 2
- 230000001743 silencing Effects 0.000 claims description 2
- 150000003852 triazoles Chemical class 0.000 claims description 2
- 239000011782 vitamin Substances 0.000 claims description 2
- 235000013343 vitamin Nutrition 0.000 claims description 2
- 150000003722 vitamin derivatives Chemical class 0.000 claims description 2
- 229930003231 vitamins Natural products 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims 3
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 description 4
- 239000003937 drug carrier Substances 0.000 description 2
- 239000000203 mixture Substances 0.000 description 2
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862721993P | 2018-08-23 | 2018-08-23 | |
| US62/721,993 | 2018-08-23 | ||
| PCT/US2019/048027 WO2020041769A1 (en) | 2018-08-23 | 2019-08-23 | O-methyl rich fully stabilized oligonucleotides |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021533804A JP2021533804A (ja) | 2021-12-09 |
| JPWO2020041769A5 true JPWO2020041769A5 (ru) | 2022-08-31 |
Family
ID=69591135
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021509991A Pending JP2021533804A (ja) | 2018-08-23 | 2019-08-23 | Ｏ−メチルリッチ完全安定化オリゴヌクレオチド |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US11279930B2 (ru) |
| EP (1) | EP3840759A4 (ru) |
| JP (1) | JP2021533804A (ru) |
| WO (1) | WO2020041769A1 (ru) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| JP2019503394A (ja) | 2016-01-31 | 2019-02-07 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 分岐オリゴヌクレオチド |
| US11753638B2 (en) | 2016-08-12 | 2023-09-12 | University Of Massachusetts | Conjugated oligonucleotides |
| EP3642341A4 (en) | 2017-06-23 | 2021-06-16 | University Of Massachusetts | TWO-DAY SELF-RELEASING SIRNA AND RELATED PROCEDURES |
| AU2020329155A1 (en) | 2019-08-09 | 2022-03-10 | University Of Massachusetts | Chemically modified oligonucleotides targeting SNPs |
| WO2021041247A1 (en) * | 2019-08-23 | 2021-03-04 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| EP4110913A4 (en) * | 2020-02-28 | 2024-07-24 | Univ Massachusetts | OLIGONUCLEOTIDES FOR PRNP MODULATION |
| CN115552006A (zh) * | 2020-03-18 | 2022-12-30 | 马萨诸塞大学 | 用于mapt调节的寡核苷酸 |
| CN117677699A (zh) * | 2021-06-23 | 2024-03-08 | 马萨诸塞大学 | 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物 |
| WO2023014654A2 (en) * | 2021-08-02 | 2023-02-09 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| WO2023087062A1 (en) * | 2021-11-18 | 2023-05-25 | Resonance Health Analysis Services Pty Ltd | Method for treating cyclophilin b associated diseases |
| AU2023234185A1 (en) * | 2022-03-16 | 2024-10-03 | Daiichi Sankyo Company, Limited | siRNA FOR SUPPRESSING EXPRESSION OF TRANSFERRIN RECEPTOR-2 |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5985558A (en) * | 1997-04-14 | 1999-11-16 | Isis Pharmaceuticals Inc. | Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos |
| US6291438B1 (en) | 1993-02-24 | 2001-09-18 | Jui H. Wang | Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use |
| US5858988A (en) | 1993-02-24 | 1999-01-12 | Wang; Jui H. | Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use |
| US8273866B2 (en) * | 2002-02-20 | 2012-09-25 | Merck Sharp & Dohme Corp. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA) |
| WO2002081628A2 (en) | 2001-04-05 | 2002-10-17 | Ribozyme Pharmaceuticals, Incorporated | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies |
| US20060009409A1 (en) * | 2002-02-01 | 2006-01-12 | Woolf Tod M | Double-stranded oligonucleotides |
| ES2864206T3 (es) | 2003-06-02 | 2021-10-13 | Univ Massachusetts | Métodos y composiciones para mejorar la eficacia y la especificidad del ARNi |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| US7425544B2 (en) * | 2003-09-18 | 2008-09-16 | Eli Lilly And Company | Modulation of eIF4E expression |
| KR101147147B1 (ko) | 2004-04-01 | 2012-05-25 | 머크 샤프 앤드 돔 코포레이션 | Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드 |
| WO2005121372A2 (en) | 2004-06-03 | 2005-12-22 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| DK1931780T3 (en) * | 2005-08-29 | 2016-01-25 | Regulus Therapeutics Inc | Antisense-forbindelser med forøget anti-microrna-aktivitet |
| US20110046206A1 (en) * | 2007-06-22 | 2011-02-24 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| CA2732229C (en) * | 2008-07-25 | 2023-10-17 | Alnylam Pharmaceuticals, Inc. | Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand |
| EP3336188B1 (en) * | 2008-09-22 | 2020-05-06 | Phio Pharmaceuticals Corp. | Reduced size self-delivering rnai compounds |
| WO2011084345A1 (en) * | 2009-12-17 | 2011-07-14 | Merck Sharp & Dohme Corp. | A method for rapidly evaluating performance of short interfering rna with novel chemical modifications |
| US9862350B2 (en) | 2013-08-12 | 2018-01-09 | Tk Holdings Inc. | Dual chambered passenger airbag |
| SG10201903290YA (en) * | 2014-08-20 | 2019-05-30 | Alnylam Pharmaceuticals Inc | Modified double-stranded rna agents |
| JOP20200092A1 (ar) * | 2014-11-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها |
| US9803205B2 (en) * | 2015-03-17 | 2017-10-31 | Arrowhead Pharmaceuticals, Inc. | Compositions and methods for inhibiting gene expression of factor XII |
| EP3277811B1 (en) * | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric sirna |
| WO2016161378A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
| LT3277814T (lt) | 2015-04-03 | 2020-10-26 | University Of Massachusetts | Oligonukleotidų junginiai, skirti nukreipimui į hantingtino mrnr |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| IL295755A (en) | 2015-10-09 | 2022-10-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods thereof |
| JP2019503394A (ja) | 2016-01-31 | 2019-02-07 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 分岐オリゴヌクレオチド |
| US11753638B2 (en) * | 2016-08-12 | 2023-09-12 | University Of Massachusetts | Conjugated oligonucleotides |
| PL3607069T3 (pl) * | 2017-04-05 | 2023-03-06 | Silence Therapeutics Gmbh | Produkty i kompozycje |
| EP3550021A1 (en) * | 2018-04-05 | 2019-10-09 | Silence Therapeutics GmbH | Products and compositions for inhibiting expression of a target gene |
| WO2021041247A1 (en) * | 2019-08-23 | 2021-03-04 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
-
2019
- 2019-08-23 JP JP2021509991A patent/JP2021533804A/ja active Pending
- 2019-08-23 WO PCT/US2019/048027 patent/WO2020041769A1/en unknown
- 2019-08-23 EP EP19852320.1A patent/EP3840759A4/en active Pending
- 2019-08-23 US US16/550,076 patent/US11279930B2/en active Active
-
2022
- 2022-01-20 US US17/580,269 patent/US20220251555A1/en active Pending
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