JPWO2020102285A5 - - Google Patents
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- Publication number
- JPWO2020102285A5 JPWO2020102285A5 JP2021525757A JP2021525757A JPWO2020102285A5 JP WO2020102285 A5 JPWO2020102285 A5 JP WO2020102285A5 JP 2021525757 A JP2021525757 A JP 2021525757A JP 2021525757 A JP2021525757 A JP 2021525757A JP WO2020102285 A5 JPWO2020102285 A5 JP WO2020102285A5
- Authority
- JP
- Japan
- Prior art keywords
- mir
- sequence
- copies
- target
- recombinant dna
- 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
- 108020004511 Recombinant DNA Proteins 0.000 claims description 59
- 238000003776 cleavage reaction Methods 0.000 claims description 54
- 230000007017 scission Effects 0.000 claims description 54
- 102000053602 DNA Human genes 0.000 claims description 51
- 108091070501 miRNA Proteins 0.000 claims description 43
- 239000002679 microRNA Substances 0.000 claims description 41
- 108700011259 MicroRNAs Proteins 0.000 claims description 36
- 108020005004 Guide RNA Proteins 0.000 claims description 32
- 230000003612 virological effect Effects 0.000 claims description 29
- 239000000427 antigen Substances 0.000 claims description 22
- 102000036639 antigens Human genes 0.000 claims description 22
- 108091007433 antigens Proteins 0.000 claims description 22
- 102000053642 Catalytic RNA Human genes 0.000 claims description 20
- 108090000994 Catalytic RNA Proteins 0.000 claims description 20
- 108091092562 ribozyme Proteins 0.000 claims description 20
- 102000040430 polynucleotide Human genes 0.000 claims description 19
- 108091033319 polynucleotide Proteins 0.000 claims description 19
- 239000002157 polynucleotide Substances 0.000 claims description 19
- 210000004027 cell Anatomy 0.000 claims description 16
- 230000001225 therapeutic effect Effects 0.000 claims description 16
- 108091070946 miR-128 stem-loop Proteins 0.000 claims description 15
- 241000700605 Viruses Species 0.000 claims description 13
- 102000004169 proteins and genes Human genes 0.000 claims description 13
- 108090000623 proteins and genes Proteins 0.000 claims description 13
- 108010001857 Cell Surface Receptors Proteins 0.000 claims description 12
- 206010028980 Neoplasm Diseases 0.000 claims description 12
- 102000006240 membrane receptors Human genes 0.000 claims description 12
- 108091007780 MiR-122 Proteins 0.000 claims description 10
- 108091027966 Mir-137 Proteins 0.000 claims description 10
- 108091027766 Mir-143 Proteins 0.000 claims description 10
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 10
- 108091028606 miR-1 stem-loop Proteins 0.000 claims description 10
- 108091051828 miR-122 stem-loop Proteins 0.000 claims description 10
- 108091056924 miR-124 stem-loop Proteins 0.000 claims description 10
- 108091031479 miR-204 stem-loop Proteins 0.000 claims description 10
- 108091032382 miR-204-1 stem-loop Proteins 0.000 claims description 10
- 108091085803 miR-204-2 stem-loop Proteins 0.000 claims description 10
- 108091089766 miR-204-3 stem-loop Proteins 0.000 claims description 10
- 108091073500 miR-204-4 stem-loop Proteins 0.000 claims description 10
- 108091053626 miR-204-5 stem-loop Proteins 0.000 claims description 10
- 108091035328 miR-217 stem-loop Proteins 0.000 claims description 10
- 108091039135 miR-217-1 stem-loop Proteins 0.000 claims description 10
- 108091029206 miR-217-2 stem-loop Proteins 0.000 claims description 10
- 108091063841 miR-219 stem-loop Proteins 0.000 claims description 10
- 108091070636 miR-219a stem-loop Proteins 0.000 claims description 10
- 150000002632 lipids Chemical class 0.000 claims description 9
- 108010012236 Chemokines Proteins 0.000 claims description 8
- 102000019034 Chemokines Human genes 0.000 claims description 8
- 108090000695 Cytokines Proteins 0.000 claims description 8
- 102000004127 Cytokines Human genes 0.000 claims description 8
- 108091028066 Mir-126 Proteins 0.000 claims description 8
- 241000709664 Picornaviridae Species 0.000 claims description 8
- 241000837158 Senecavirus A Species 0.000 claims description 8
- 239000003446 ligand Substances 0.000 claims description 8
- 230000010076 replication Effects 0.000 claims description 8
- 241000114864 ssRNA viruses Species 0.000 claims description 7
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 6
- 230000002401 inhibitory effect Effects 0.000 claims description 6
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 239000002105 nanoparticle Substances 0.000 claims description 5
- 150000007523 nucleic acids Chemical group 0.000 claims description 5
- 239000002245 particle Substances 0.000 claims description 5
- 102100036846 C-C motif chemokine 21 Human genes 0.000 claims description 4
- 102100032367 C-C motif chemokine 5 Human genes 0.000 claims description 4
- 102100025248 C-X-C motif chemokine 10 Human genes 0.000 claims description 4
- 241000709687 Coxsackievirus Species 0.000 claims description 4
- 102100036466 Delta-like protein 3 Human genes 0.000 claims description 4
- 101150084967 EPCAM gene Proteins 0.000 claims description 4
- 102100020715 Fms-related tyrosine kinase 3 ligand protein Human genes 0.000 claims description 4
- 101710162577 Fms-related tyrosine kinase 3 ligand protein Proteins 0.000 claims description 4
- 101000713085 Homo sapiens C-C motif chemokine 21 Proteins 0.000 claims description 4
- 101000797762 Homo sapiens C-C motif chemokine 5 Proteins 0.000 claims description 4
- 101000858088 Homo sapiens C-X-C motif chemokine 10 Proteins 0.000 claims description 4
- 101000914324 Homo sapiens Carcinoembryonic antigen-related cell adhesion molecule 5 Proteins 0.000 claims description 4
- 101000914321 Homo sapiens Carcinoembryonic antigen-related cell adhesion molecule 7 Proteins 0.000 claims description 4
- 101000928513 Homo sapiens Delta-like protein 3 Proteins 0.000 claims description 4
- 101000617725 Homo sapiens Pregnancy-specific beta-1-glycoprotein 2 Proteins 0.000 claims description 4
- 108010002350 Interleukin-2 Proteins 0.000 claims description 4
- 102000009572 RNA Polymerase II Human genes 0.000 claims description 4
- 108010009460 RNA Polymerase II Proteins 0.000 claims description 4
- 229940127276 delta-like ligand 3 Drugs 0.000 claims description 4
- 230000002255 enzymatic effect Effects 0.000 claims description 4
- 102000034287 fluorescent proteins Human genes 0.000 claims description 4
- 108091006047 fluorescent proteins Proteins 0.000 claims description 4
- 230000002101 lytic effect Effects 0.000 claims description 4
- 239000013612 plasmid Substances 0.000 claims description 4
- 208000015181 infectious disease Diseases 0.000 claims description 3
- 230000002458 infectious effect Effects 0.000 claims description 3
- 206010058467 Lung neoplasm malignant Diseases 0.000 claims description 2
- 201000011510 cancer Diseases 0.000 claims description 2
- 230000001524 infective effect Effects 0.000 claims description 2
- 230000002601 intratumoral effect Effects 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 201000007270 liver cancer Diseases 0.000 claims description 2
- 208000014018 liver neoplasm Diseases 0.000 claims description 2
- 201000005202 lung cancer Diseases 0.000 claims description 2
- 208000020816 lung neoplasm Diseases 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 4
- 241000710929 Alphavirus Species 0.000 claims 2
- 108091060568 Mir-133 microRNA precursor family Proteins 0.000 claims 2
- 108091028684 Mir-145 Proteins 0.000 claims 2
- 108091023685 miR-133 stem-loop Proteins 0.000 claims 2
- 108091063796 miR-206 stem-loop Proteins 0.000 claims 2
- 239000002086 nanomaterial Substances 0.000 claims 1
- 230000004614 tumor growth Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 12
- 101000941029 Homo sapiens Endoplasmic reticulum junction formation protein lunapark Proteins 0.000 description 10
- 102000037978 Immune checkpoint receptors Human genes 0.000 description 4
- 108091008028 Immune checkpoint receptors Proteins 0.000 description 4
- -1 cationic lipid Chemical class 0.000 description 4
- 229920001223 polyethylene glycol Polymers 0.000 description 4
- 230000007935 neutral effect Effects 0.000 description 3
- 229920000642 polymer Polymers 0.000 description 3
- KWVJHCQQUFDPLU-YEUCEMRASA-N 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KWVJHCQQUFDPLU-YEUCEMRASA-N 0.000 description 2
- ZLGYVWRJIZPQMM-HHHXNRCGSA-N 2-azaniumylethyl [(2r)-2,3-di(dodecanoyloxy)propyl] phosphate Chemical compound CCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)CCCCCCCCCCC ZLGYVWRJIZPQMM-HHHXNRCGSA-N 0.000 description 2
- LYCAIKOWRPUZTN-UHFFFAOYSA-N Ethylene glycol Chemical compound OCCO LYCAIKOWRPUZTN-UHFFFAOYSA-N 0.000 description 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 description 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 description 1
- IYMAXBFPHPZYIK-BQBZGAKWSA-N Arg-Gly-Asp Chemical compound NC(N)=NCCC[C@H](N)C(=O)NCC(=O)N[C@@H](CC(O)=O)C(O)=O IYMAXBFPHPZYIK-BQBZGAKWSA-N 0.000 description 1
- 239000002202 Polyethylene glycol Substances 0.000 description 1
- NRLNQCOGCKAESA-KWXKLSQISA-N [(6z,9z,28z,31z)-heptatriaconta-6,9,28,31-tetraen-19-yl] 4-(dimethylamino)butanoate Chemical compound CCCCC\C=C/C\C=C/CCCCCCCCC(OC(=O)CCCN(C)C)CCCCCCCC\C=C/C\C=C/CCCCC NRLNQCOGCKAESA-KWXKLSQISA-N 0.000 description 1
- 108010072041 arginyl-glycyl-aspartic acid Proteins 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 235000012000 cholesterol Nutrition 0.000 description 1
- KIUKXJAPPMFGSW-MNSSHETKSA-N hyaluronan Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)C1O[C@H]1[C@H](O)[C@@H](O)[C@H](O[C@H]2[C@@H](C(O[C@H]3[C@@H]([C@@H](O)[C@H](O)[C@H](O3)C(O)=O)O)[C@H](O)[C@@H](CO)O2)NC(C)=O)[C@@H](C(O)=O)O1 KIUKXJAPPMFGSW-MNSSHETKSA-N 0.000 description 1
- 229920002674 hyaluronan Polymers 0.000 description 1
- 229940099552 hyaluronan Drugs 0.000 description 1
- WGCNASOHLSPBMP-UHFFFAOYSA-N hydroxyacetaldehyde Natural products OCC=O WGCNASOHLSPBMP-UHFFFAOYSA-N 0.000 description 1
- 230000002934 lysing effect Effects 0.000 description 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862760422P | 2018-11-13 | 2018-11-13 | |
| US62/760,422 | 2018-11-13 | ||
| PCT/US2019/061093 WO2020102285A1 (en) | 2018-11-13 | 2019-11-13 | Encapsulated polynucleotides and methods of use |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022507269A JP2022507269A (ja) | 2022-01-18 |
| JPWO2020102285A5 true JPWO2020102285A5 (https=) | 2022-11-07 |
| JP2022507269A5 JP2022507269A5 (https=) | 2022-11-07 |
Family
ID=70731661
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021525757A Pending JP2022507269A (ja) | 2018-11-13 | 2019-11-13 | カプセル化ポリヌクレオチド及び使用方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20210403950A1 (https=) |
| EP (1) | EP3880812A4 (https=) |
| JP (1) | JP2022507269A (https=) |
| KR (1) | KR20210093285A (https=) |
| CN (1) | CN113348246A (https=) |
| AU (1) | AU2019381698A1 (https=) |
| BR (1) | BR112021009226A2 (https=) |
| CA (1) | CA3117924A1 (https=) |
| IL (1) | IL282992A (https=) |
| MX (1) | MX2021005448A (https=) |
| SG (1) | SG11202104887UA (https=) |
| WO (1) | WO2020102285A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11130787B2 (en) * | 2020-06-11 | 2021-09-28 | MBF Therapeutics, Inc. | Alphaherpesvirus glycoprotein d-encoding nucleic acid constructs and methods |
| MX2023009345A (es) * | 2021-02-10 | 2023-10-24 | Oncorus Inc | Compuestos, composiciones y metodos de uso de estos. |
| IT202200016386A1 (it) * | 2022-08-02 | 2024-02-02 | Univ Degli Studi Roma La Sapienza | Nanoparticelle lipidiche multicomponenti ad elevata fusogenicità cellulare per la veicolazione di acidi nucleici e relativo processo di preparazione |
| EP4658782A2 (en) * | 2023-02-02 | 2025-12-10 | The Regents of the University of California | Self-amplifying non-coding rna for targeted gene knockdown |
| EP4683655A1 (en) * | 2023-03-20 | 2026-01-28 | Dimberg, Anna | Tumor blood vessel-targeted aav therapy for cancer treatment |
| CN117487762A (zh) * | 2023-11-06 | 2024-02-02 | 中科佰乘(深圳)生物科技有限公司 | 一种细胞外囊泡、制备方法、药物组合物及应用 |
| CN119242712A (zh) * | 2024-09-27 | 2025-01-03 | 南京师范大学 | 特异性靶向骨骼肌细胞的携带miRNA-486基因的腺相关病毒载体、药物组合物 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1092777A1 (en) * | 1999-10-15 | 2001-04-18 | Aventis Pharma S.A. | Self-cleaving RNA sequences and their use for the control of protein synthesis. |
| AU2002953436A0 (en) * | 2002-12-18 | 2003-01-09 | The University Of Newcastle Research Associates Limited | A method of treating a malignancy in a subject via direct picornaviral-mediated oncolysis |
| KR20060026854A (ko) * | 2003-05-28 | 2006-03-24 | 위스콘신 얼럼나이 리서어치 화운데이션 | ＰｏｌⅡ 프로모터 및 리보자임을 갖는 재조합 인플루엔자벡터 |
| WO2005030139A2 (en) * | 2003-09-26 | 2005-04-07 | Novartis Ag | Seneca valley virus based compositions and methods for treating disease |
| WO2006074526A1 (en) * | 2005-01-17 | 2006-07-20 | Viralytics Limited | Method and composition for treatment of neoplasms |
| US9006487B2 (en) * | 2005-06-15 | 2015-04-14 | Massachusetts Institute Of Technology | Amine-containing lipids and uses thereof |
| WO2016191684A1 (en) * | 2015-05-28 | 2016-12-01 | Finer Mitchell H | Genome editing vectors |
| EP3307308A2 (en) * | 2015-06-10 | 2018-04-18 | Hookipa Biotech AG | Hpv vaccines |
| RU2749050C2 (ru) * | 2016-01-27 | 2021-06-03 | Онкорус, Инк. | Онколитические вирусные векторы и их применение |
| RU2021127872A (ru) * | 2016-06-30 | 2021-11-09 | Онкорус, Инк. | Доставка терапевтических полипептидов посредством псевдотипированных онколитических вирусов |
| EP3472318B1 (en) * | 2016-07-08 | 2023-09-06 | Exuma Biotech Corp. | Methods and compositions for transducing lymphocytes and regulating the activity thereof |
| EP3538515B1 (en) * | 2016-11-08 | 2022-07-20 | Ramot at Tel-Aviv University Ltd. | Cationic lipids for nucleic acid delivery and preparation thereof |
| AU2018301701A1 (en) * | 2017-07-14 | 2020-02-27 | Elevatebio Technologies, Inc. | Encapsulated polynucleotides and methods of use |
| CA3157063A1 (en) * | 2019-10-10 | 2021-04-15 | Oncorus, Inc. | Dual viruses and dual oncolytic viruses and methods of treatment |
-
2019
- 2019-11-13 EP EP19884039.9A patent/EP3880812A4/en not_active Withdrawn
- 2019-11-13 CN CN201980085641.2A patent/CN113348246A/zh active Pending
- 2019-11-13 WO PCT/US2019/061093 patent/WO2020102285A1/en not_active Ceased
- 2019-11-13 JP JP2021525757A patent/JP2022507269A/ja active Pending
- 2019-11-13 US US17/293,153 patent/US20210403950A1/en not_active Abandoned
- 2019-11-13 AU AU2019381698A patent/AU2019381698A1/en not_active Abandoned
- 2019-11-13 MX MX2021005448A patent/MX2021005448A/es unknown
- 2019-11-13 SG SG11202104887UA patent/SG11202104887UA/en unknown
- 2019-11-13 CA CA3117924A patent/CA3117924A1/en active Pending
- 2019-11-13 BR BR112021009226-9A patent/BR112021009226A2/pt not_active IP Right Cessation
- 2019-11-13 KR KR1020217017630A patent/KR20210093285A/ko not_active Ceased
-
2021
- 2021-05-06 IL IL282992A patent/IL282992A/en unknown
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