JPWO2020065602A5 - - Google Patents
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- Publication number
- JPWO2020065602A5 JPWO2020065602A5 JP2021517420A JP2021517420A JPWO2020065602A5 JP WO2020065602 A5 JPWO2020065602 A5 JP WO2020065602A5 JP 2021517420 A JP2021517420 A JP 2021517420A JP 2021517420 A JP2021517420 A JP 2021517420A JP WO2020065602 A5 JPWO2020065602 A5 JP WO2020065602A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- nucleotides
- construct
- acid portion
- moieties
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims 279
- 108020004707 nucleic acids Proteins 0.000 claims 258
- 102000039446 nucleic acids Human genes 0.000 claims 258
- 125000003729 nucleotide group Chemical group 0.000 claims 95
- 239000002773 nucleotide Substances 0.000 claims 74
- 230000004048 modification Effects 0.000 claims 43
- 238000012986 modification Methods 0.000 claims 43
- 108090000623 proteins and genes Proteins 0.000 claims 20
- 230000008685 targeting Effects 0.000 claims 20
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 12
- 230000000295 complement effect Effects 0.000 claims 12
- 238000003776 cleavage reaction Methods 0.000 claims 10
- 230000007017 scission Effects 0.000 claims 10
- 239000003446 ligand Substances 0.000 claims 8
- 238000001727 in vivo Methods 0.000 claims 7
- 238000000034 method Methods 0.000 claims 7
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 7
- 150000001720 carbohydrates Chemical group 0.000 claims 6
- 235000014633 carbohydrates Nutrition 0.000 claims 6
- NAGJZTKCGNOGPW-UHFFFAOYSA-K dioxido-sulfanylidene-sulfido-$l^{5}-phosphane Chemical compound [O-]P([O-])([S-])=S NAGJZTKCGNOGPW-UHFFFAOYSA-K 0.000 claims 6
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 5
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims 4
- 229910052799 carbon Inorganic materials 0.000 claims 4
- 210000004027 cell Anatomy 0.000 claims 4
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical group CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims 3
- 238000000338 in vitro Methods 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 claims 2
- 229910019142 PO4 Inorganic materials 0.000 claims 2
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims 2
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- NAGJZTKCGNOGPW-UHFFFAOYSA-N dithiophosphoric acid Chemical group OP(O)(S)=S NAGJZTKCGNOGPW-UHFFFAOYSA-N 0.000 claims 2
- ZTWTYVWXUKTLCP-UHFFFAOYSA-L ethenyl-dioxido-oxo-$l^{5}-phosphane Chemical compound [O-]P([O-])(=O)C=C ZTWTYVWXUKTLCP-UHFFFAOYSA-L 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 2
- 239000010452 phosphate Substances 0.000 claims 2
- 208000035657 Abasia Diseases 0.000 claims 1
- 108091023037 Aptamer Chemical group 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical group OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 1
- 210000000170 cell membrane Anatomy 0.000 claims 1
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol group Chemical group [C@@H]1(CC[C@H]2[C@@H]3CC=C4C[C@@H](O)CC[C@]4(C)[C@H]3CC[C@]12C)[C@H](C)CCCC(C)C HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 1
- 238000010276 construction Methods 0.000 claims 1
- 239000002537 cosmetic Substances 0.000 claims 1
- 238000009792 diffusion process Methods 0.000 claims 1
- 150000002016 disaccharides Chemical class 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 230000009977 dual effect Effects 0.000 claims 1
- 230000030279 gene silencing Effects 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 150000004676 glycans Chemical class 0.000 claims 1
- GUBGYTABKSRVRQ-QKKXKWKRSA-N lactose group Chemical group OC1[C@H](O)[C@@H](O)[C@H](O[C@H]2[C@H](O)[C@@H](O)[C@@H](O)[C@H](O2)CO)[C@H](O1)CO GUBGYTABKSRVRQ-QKKXKWKRSA-N 0.000 claims 1
- 125000005647 linker group Chemical group 0.000 claims 1
- 125000003473 lipid group Chemical group 0.000 claims 1
- 125000001921 locked nucleotide group Chemical group 0.000 claims 1
- 125000000311 mannosyl group Chemical group C1([C@@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 150000002772 monosaccharides Chemical class 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- ONKSSDKXDIVIHK-UHFFFAOYSA-N n,n-didecyldodecanamide Chemical group CCCCCCCCCCCC(=O)N(CCCCCCCCCC)CCCCCCCCCC ONKSSDKXDIVIHK-UHFFFAOYSA-N 0.000 claims 1
- 229920001542 oligosaccharide Polymers 0.000 claims 1
- 150000002482 oligosaccharides Chemical class 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims 1
- 229920001282 polysaccharide Polymers 0.000 claims 1
- 239000005017 polysaccharide Substances 0.000 claims 1
- 108090000765 processed proteins & peptides Chemical group 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 230000002194 synthesizing effect Effects 0.000 claims 1
- 150000004044 tetrasaccharides Chemical class 0.000 claims 1
- 150000004043 trisaccharides Chemical class 0.000 claims 1
- 239000011782 vitamin Chemical group 0.000 claims 1
- 229930003231 vitamin Chemical group 0.000 claims 1
- 229940088594 vitamin Drugs 0.000 claims 1
- 235000013343 vitamin Nutrition 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862738222P | 2018-09-28 | 2018-09-28 | |
| US62/738,222 | 2018-09-28 | ||
| PCT/IB2019/058221 WO2020065602A2 (en) | 2018-09-28 | 2019-09-27 | Products and compositions |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022503850A JP2022503850A (ja) | 2022-01-12 |
| JP2022503850A5 JP2022503850A5 (https=) | 2022-09-28 |
| JPWO2020065602A5 true JPWO2020065602A5 (https=) | 2022-09-28 |
Family
ID=69951906
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021517420A Pending JP2022503850A (ja) | 2018-09-28 | 2019-09-27 | 産物及び組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210371861A1 (https=) |
| EP (1) | EP3856200A4 (https=) |
| JP (1) | JP2022503850A (https=) |
| KR (1) | KR20210093851A (https=) |
| CN (1) | CN113164507A (https=) |
| AU (1) | AU2019346148A1 (https=) |
| BR (1) | BR112021005777A2 (https=) |
| CA (1) | CA3110300A1 (https=) |
| IL (1) | IL281684A (https=) |
| WO (1) | WO2020065602A2 (https=) |
| ZA (1) | ZA202101298B (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20250000892A1 (en) * | 2021-10-14 | 2025-01-02 | Arizona Board Of Regents On Behalf Of Arizona State University | Programmable delivery platforms for rna structures |
| WO2023069707A2 (en) * | 2021-10-22 | 2023-04-27 | Sirnaomics, Inc. | Products and compositions |
| WO2023240249A1 (en) * | 2022-06-11 | 2023-12-14 | Sirnaomics, Inc. | Products and compositions |
| WO2024059873A2 (en) * | 2022-09-16 | 2024-03-21 | Sirnaomics, Inc. | Products and compositions |
| WO2024081922A1 (en) * | 2022-10-14 | 2024-04-18 | Arizona Board Of Regents On Behalf Of Arizona State University | Modular rna delivery platforms and methods of their use |
| WO2024164001A2 (en) * | 2023-02-03 | 2024-08-08 | Sirnaomics, Inc. | Products and compositions |
| WO2024216267A2 (en) * | 2023-04-14 | 2024-10-17 | Sirnaomics, Inc. | Products and compositions |
| WO2024229229A2 (en) * | 2023-05-02 | 2024-11-07 | Sirnaomics, Inc. | Products and compositions |
| TW202526017A (zh) | 2023-09-14 | 2025-07-01 | 美商Ionis製藥公司 | 用於減少ａｐｏｃｉｉｉ表現的化合物及方法 |
| WO2025064821A2 (en) | 2023-09-21 | 2025-03-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for inhibiting lpa |
| WO2025259875A1 (en) * | 2024-06-12 | 2025-12-18 | Sirnaomics, Inc. | Novel antibody oligonucleotide drug conjugates containing gemcitibine for pharmaceutical compositions and related methods of use and treatment |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1989307B1 (en) * | 2006-02-08 | 2012-08-08 | Quark Pharmaceuticals, Inc. | NOVEL TANDEM siRNAS |
| US8252526B2 (en) * | 2006-11-09 | 2012-08-28 | Gradalis, Inc. | ShRNA molecules and methods of use thereof |
| EP2318528A1 (en) * | 2008-07-24 | 2011-05-11 | Rxi Pharmaceuticals Corporation | Rnai constructs and uses therof |
| EP2395085B1 (en) * | 2009-02-04 | 2015-06-10 | Sungkyunkwan University Foundation for Corporate Collaboration | Small interference rna complex with increased intracellular transmission capacity |
| ES2804764T3 (es) * | 2009-06-01 | 2021-02-09 | Halo Bio Rnai Therapeutics Inc | Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos |
| WO2015164818A1 (en) * | 2014-04-25 | 2015-10-29 | Strike Bio, Inc. | Multiple targeted rnai for the treatment of cancers |
| EP4365291A3 (en) * | 2015-06-12 | 2024-08-14 | Alnylam Pharmaceuticals, Inc. | Complement component c5 irna compositions and methods of use thereof |
| IL316159A (en) * | 2015-06-15 | 2024-12-01 | Mpeg La Llc | Defined Oligonucleotide Multimers and Methods for Manufacture Thereof |
| US10689654B2 (en) * | 2016-10-18 | 2020-06-23 | Augusta University Research Institute, Inc. | Bivalent siRNA chimeras and methods of use thereof |
| CN108342386B (zh) * | 2017-01-22 | 2022-04-15 | 广州市锐博生物科技有限公司 | 一种多聚寡核酸分子及其在多靶标干扰中的应用 |
| WO2020014948A1 (zh) * | 2018-07-20 | 2020-01-23 | 广州市锐博生物科技有限公司 | 核酸单元及其聚合核酸与应用 |
-
2019
- 2019-09-27 CN CN201980063688.9A patent/CN113164507A/zh active Pending
- 2019-09-27 CA CA3110300A patent/CA3110300A1/en active Pending
- 2019-09-27 EP EP19866965.7A patent/EP3856200A4/en active Pending
- 2019-09-27 AU AU2019346148A patent/AU2019346148A1/en not_active Abandoned
- 2019-09-27 WO PCT/IB2019/058221 patent/WO2020065602A2/en not_active Ceased
- 2019-09-27 JP JP2021517420A patent/JP2022503850A/ja active Pending
- 2019-09-27 KR KR1020217009902A patent/KR20210093851A/ko not_active Withdrawn
- 2019-09-27 BR BR112021005777A patent/BR112021005777A2/pt not_active IP Right Cessation
-
2021
- 2021-02-25 ZA ZA2021/01298A patent/ZA202101298B/en unknown
- 2021-03-21 IL IL281684A patent/IL281684A/en unknown
- 2021-03-29 US US17/215,964 patent/US20210371861A1/en active Pending
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