JPWO2020123083A5 - - Google Patents
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- Publication number
- JPWO2020123083A5 JPWO2020123083A5 JP2021534224A JP2021534224A JPWO2020123083A5 JP WO2020123083 A5 JPWO2020123083 A5 JP WO2020123083A5 JP 2021534224 A JP2021534224 A JP 2021534224A JP 2021534224 A JP2021534224 A JP 2021534224A JP WO2020123083 A5 JPWO2020123083 A5 JP WO2020123083A5
- Authority
- JP
- Japan
- Prior art keywords
- double
- nucleic acid
- stranded nucleic
- acid inhibitor
- inhibitor molecule
- 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.)
- Granted
Links
- 108020004707 nucleic acids Proteins 0.000 claims description 30
- 150000007523 nucleic acids Chemical class 0.000 claims description 30
- 102000039446 nucleic acids Human genes 0.000 claims description 30
- 239000003112 inhibitor Substances 0.000 claims description 29
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 15
- 239000008194 pharmaceutical composition Substances 0.000 claims description 13
- 230000014509 gene expression Effects 0.000 claims description 3
- 235000000346 sugar Nutrition 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims 22
- 125000003729 nucleotide group Chemical group 0.000 claims 22
- 150000002632 lipids Chemical class 0.000 claims 12
- 108091081021 Sense strand Proteins 0.000 claims 10
- 230000000692 anti-sense effect Effects 0.000 claims 8
- 229910052799 carbon Inorganic materials 0.000 claims 4
- -1 cationic lipid Chemical class 0.000 claims 4
- 125000002619 bicyclic group Chemical group 0.000 claims 3
- 125000003843 furanosyl group Chemical group 0.000 claims 3
- 239000003446 ligand Substances 0.000 claims 3
- 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 2
- 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 2
- 108091034117 Oligonucleotide Proteins 0.000 claims 2
- 229930182558 Sterol Natural products 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 125000005647 linker group Chemical group 0.000 claims 2
- 239000002105 nanoparticle Substances 0.000 claims 2
- 230000007935 neutral effect Effects 0.000 claims 2
- 150000003432 sterols Chemical class 0.000 claims 2
- 235000003702 sterols Nutrition 0.000 claims 2
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical group [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 1
- 101150046432 Tril gene Proteins 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 125000004122 cyclic group Chemical group 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 125000006239 protecting group Chemical group 0.000 claims 1
- 239000000203 mixture Substances 0.000 description 5
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 3
- DNIAPMSPPWPWGF-UHFFFAOYSA-N Propylene glycol Chemical compound CC(O)CO DNIAPMSPPWPWGF-UHFFFAOYSA-N 0.000 description 3
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 2
- 238000009472 formulation Methods 0.000 description 2
- 238000000034 method Methods 0.000 description 2
- 108090000623 proteins and genes Proteins 0.000 description 2
- GUBGYTABKSRVRQ-XLOQQCSPSA-N Alpha-Lactose Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1O[C@@H]1[C@@H](CO)O[C@H](O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-XLOQQCSPSA-N 0.000 description 1
- 229920002261 Corn starch Polymers 0.000 description 1
- 108010010803 Gelatin Proteins 0.000 description 1
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 1
- 206010020751 Hypersensitivity Diseases 0.000 description 1
- GUBGYTABKSRVRQ-QKKXKWKRSA-N Lactose Natural products OC[C@H]1O[C@@H](O[C@H]2[C@H](O)[C@@H](O)C(O)O[C@@H]2CO)[C@H](O)[C@@H](O)[C@H]1O GUBGYTABKSRVRQ-QKKXKWKRSA-N 0.000 description 1
- GXCLVBGFBYZDAG-UHFFFAOYSA-N N-[2-(1H-indol-3-yl)ethyl]-N-methylprop-2-en-1-amine Chemical compound CN(CCC1=CNC2=C1C=CC=C2)CC=C GXCLVBGFBYZDAG-UHFFFAOYSA-N 0.000 description 1
- 235000019483 Peanut oil Nutrition 0.000 description 1
- 239000002202 Polyethylene glycol Substances 0.000 description 1
- 235000019485 Safflower oil Nutrition 0.000 description 1
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 1
- 229920002472 Starch Polymers 0.000 description 1
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 1
- 229930006000 Sucrose Natural products 0.000 description 1
- 240000008042 Zea mays Species 0.000 description 1
- 235000005824 Zea mays ssp. parviglumis Nutrition 0.000 description 1
- 235000002017 Zea mays subsp mays Nutrition 0.000 description 1
- 230000002411 adverse Effects 0.000 description 1
- WNROFYMDJYEPJX-UHFFFAOYSA-K aluminium hydroxide Chemical compound [OH-].[OH-].[OH-].[Al+3] WNROFYMDJYEPJX-UHFFFAOYSA-K 0.000 description 1
- 239000007864 aqueous solution Substances 0.000 description 1
- 239000008135 aqueous vehicle Substances 0.000 description 1
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 description 1
- 239000000872 buffer Substances 0.000 description 1
- 239000001913 cellulose Substances 0.000 description 1
- 229920002678 cellulose Polymers 0.000 description 1
- 229940110456 cocoa butter Drugs 0.000 description 1
- 235000019868 cocoa butter Nutrition 0.000 description 1
- 235000005822 corn Nutrition 0.000 description 1
- 235000005687 corn oil Nutrition 0.000 description 1
- 239000002285 corn oil Substances 0.000 description 1
- 235000012343 cottonseed oil Nutrition 0.000 description 1
- 239000002385 cottonseed oil Substances 0.000 description 1
- 239000002552 dosage form Substances 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 235000019441 ethanol Nutrition 0.000 description 1
- 229920000159 gelatin Polymers 0.000 description 1
- 239000008273 gelatin Substances 0.000 description 1
- 235000019322 gelatine Nutrition 0.000 description 1
- 235000011852 gelatine desserts Nutrition 0.000 description 1
- 239000008103 glucose Substances 0.000 description 1
- 230000007794 irritation Effects 0.000 description 1
- 239000008101 lactose Substances 0.000 description 1
- VTHJTEIRLNZDEV-UHFFFAOYSA-L magnesium dihydroxide Chemical compound [OH-].[OH-].[Mg+2] VTHJTEIRLNZDEV-UHFFFAOYSA-L 0.000 description 1
- 239000000347 magnesium hydroxide Substances 0.000 description 1
- 229910001862 magnesium hydroxide Inorganic materials 0.000 description 1
- 239000000463 material Substances 0.000 description 1
- 231100000252 nontoxic Toxicity 0.000 description 1
- 230000003000 nontoxic effect Effects 0.000 description 1
- 239000003921 oil Substances 0.000 description 1
- 235000019198 oils Nutrition 0.000 description 1
- 239000004006 olive oil Substances 0.000 description 1
- 235000008390 olive oil Nutrition 0.000 description 1
- 239000006174 pH buffer Substances 0.000 description 1
- 239000000312 peanut oil Substances 0.000 description 1
- 229920001223 polyethylene glycol Polymers 0.000 description 1
- 229920005862 polyol Polymers 0.000 description 1
- 150000003077 polyols Chemical class 0.000 description 1
- 229920001592 potato starch Polymers 0.000 description 1
- 235000005713 safflower oil Nutrition 0.000 description 1
- 239000003813 safflower oil Substances 0.000 description 1
- 239000008159 sesame oil Substances 0.000 description 1
- 235000011803 sesame oil Nutrition 0.000 description 1
- 239000000243 solution Substances 0.000 description 1
- 239000003549 soybean oil Substances 0.000 description 1
- 235000012424 soybean oil Nutrition 0.000 description 1
- 230000000087 stabilizing effect Effects 0.000 description 1
- 235000019698 starch Nutrition 0.000 description 1
- 230000001954 sterilising effect Effects 0.000 description 1
- 238000004659 sterilization and disinfection Methods 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 239000005720 sucrose Substances 0.000 description 1
- 150000008163 sugars Chemical class 0.000 description 1
- 239000000829 suppository Substances 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 231100000419 toxicity Toxicity 0.000 description 1
- 230000001988 toxicity Effects 0.000 description 1
- 239000001993 wax Substances 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024070175A JP7748500B2 (ja) | 2018-12-12 | 2024-04-24 | トリループを含む二本鎖核酸インヒビター分子 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862778759P | 2018-12-12 | 2018-12-12 | |
| US62/778,759 | 2018-12-12 | ||
| PCT/US2019/061241 WO2020123083A1 (en) | 2018-12-12 | 2019-11-13 | Double-stranded nucleic acid inhibitor molecules containing a triloop |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024070175A Division JP7748500B2 (ja) | 2018-12-12 | 2024-04-24 | トリループを含む二本鎖核酸インヒビター分子 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022521877A JP2022521877A (ja) | 2022-04-13 |
| JPWO2020123083A5 true JPWO2020123083A5 (https=) | 2022-11-24 |
| JP2022521877A5 JP2022521877A5 (https=) | 2022-11-24 |
| JP7535047B2 JP7535047B2 (ja) | 2024-08-15 |
Family
ID=71076672
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021534224A Active JP7535047B2 (ja) | 2018-12-12 | 2019-11-13 | トリループを含む二本鎖核酸インヒビター分子 |
| JP2024070175A Active JP7748500B2 (ja) | 2018-12-12 | 2024-04-24 | トリループを含む二本鎖核酸インヒビター分子 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024070175A Active JP7748500B2 (ja) | 2018-12-12 | 2024-04-24 | トリループを含む二本鎖核酸インヒビター分子 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220064640A1 (https=) |
| EP (1) | EP3894560A4 (https=) |
| JP (2) | JP7535047B2 (https=) |
| KR (1) | KR20210127917A (https=) |
| CN (1) | CN113454221A (https=) |
| AU (1) | AU2019397247A1 (https=) |
| BR (1) | BR112021011294A2 (https=) |
| CA (1) | CA3122930A1 (https=) |
| CL (1) | CL2021001552A1 (https=) |
| IL (1) | IL283775A (https=) |
| MX (1) | MX2021007001A (https=) |
| SG (1) | SG11202106046XA (https=) |
| WO (1) | WO2020123083A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2019397247A1 (en) * | 2018-12-12 | 2021-06-24 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules containing a triloop |
| WO2020226960A1 (en) | 2019-05-03 | 2020-11-12 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules with shortened sense strands |
| MX2023001450A (es) * | 2020-08-04 | 2023-04-14 | Dicerna Pharmaceuticals Inc | Composiciones y metodos para la inhibicion de la expresion de plp1. |
| CA3226019A1 (en) | 2021-07-20 | 2023-01-26 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their preparation, and uses |
| WO2023144127A1 (en) | 2022-01-31 | 2023-08-03 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their biodistribution upon administration, and uses |
| KR20250005108A (ko) * | 2022-04-15 | 2025-01-09 | 다이서나 파마수이티컬, 인크. | Scap 활성을 조절하기 위한 조성물 및 방법 |
| WO2023232976A1 (en) | 2022-06-03 | 2023-12-07 | Ags Therapeutics Sas | Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses |
| CN121038801A (zh) | 2022-10-24 | 2025-11-28 | Ags治疗简易股份公司 | 来自微藻的细胞外囊泡、它们在鼻内施用时的生物分布及其用途 |
| WO2025176847A1 (en) | 2024-02-21 | 2025-08-28 | Ags Therapeutics Sas | Ocular delivery of active agents via microalgae extracellular vesicles |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8067572B2 (en) * | 2005-05-25 | 2011-11-29 | The University Of York | Hybrid interfering RNA |
| 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 |
| EP2363467B1 (en) * | 2008-10-23 | 2015-12-16 | The University of Tokyo | Method for inhibiting function of micro-rna |
| EP3358014B1 (en) * | 2010-04-19 | 2020-12-23 | TAGCyx Biotechnologies Inc. | Method for stabilizing functional nucleic acids |
| EP2781598B1 (en) * | 2011-11-16 | 2018-08-08 | Osaka City University | Nucleic acid molecule for inhibiting activity of rnai molecule |
| 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 |
| JP6987084B2 (ja) * | 2016-03-16 | 2021-12-22 | ディセルナ ファーマシューティカルズ インコーポレイテッド | β−カテニン関連疾患又は障害の治療のための組成物及び方法 |
| KR102350647B1 (ko) * | 2016-09-02 | 2022-01-14 | 다이서나 파마수이티컬, 인크. | 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드 |
| KR20200023427A (ko) * | 2017-06-29 | 2020-03-04 | 다이서나 파마수이티컬, 인크. | Hmgb1 발현을 억제하기 위한 조성물 및 방법 |
| CA3094008A1 (en) * | 2018-04-13 | 2019-10-17 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides |
| AU2019397247A1 (en) | 2018-12-12 | 2021-06-24 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules containing a triloop |
-
2019
- 2019-11-13 AU AU2019397247A patent/AU2019397247A1/en not_active Abandoned
- 2019-11-13 CA CA3122930A patent/CA3122930A1/en active Pending
- 2019-11-13 WO PCT/US2019/061241 patent/WO2020123083A1/en not_active Ceased
- 2019-11-13 JP JP2021534224A patent/JP7535047B2/ja active Active
- 2019-11-13 BR BR112021011294-4A patent/BR112021011294A2/pt not_active IP Right Cessation
- 2019-11-13 CN CN201980091916.3A patent/CN113454221A/zh not_active Withdrawn
- 2019-11-13 KR KR1020217021547A patent/KR20210127917A/ko not_active Ceased
- 2019-11-13 SG SG11202106046XA patent/SG11202106046XA/en unknown
- 2019-11-13 MX MX2021007001A patent/MX2021007001A/es unknown
- 2019-11-13 US US17/311,949 patent/US20220064640A1/en not_active Abandoned
- 2019-11-13 EP EP19895099.0A patent/EP3894560A4/en active Pending
-
2021
- 2021-06-07 IL IL283775A patent/IL283775A/en unknown
- 2021-06-11 CL CL2021001552A patent/CL2021001552A1/es unknown
-
2024
- 2024-04-24 JP JP2024070175A patent/JP7748500B2/ja active Active
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