JP2020511521A5 - - Google Patents
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- JP2020511521A5 JP2020511521A5 JP2019552611A JP2019552611A JP2020511521A5 JP 2020511521 A5 JP2020511521 A5 JP 2020511521A5 JP 2019552611 A JP2019552611 A JP 2019552611A JP 2019552611 A JP2019552611 A JP 2019552611A JP 2020511521 A5 JP2020511521 A5 JP 2020511521A5
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- JP
- Japan
- Prior art keywords
- composition
- antigen
- therapeutic
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- present
- 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.)
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- 239000000203 mixture Substances 0.000 claims 54
- 230000001225 therapeutic effect Effects 0.000 claims 14
- 239000000427 antigen Substances 0.000 claims 13
- 102000036639 antigens Human genes 0.000 claims 13
- 108091007433 antigens Proteins 0.000 claims 13
- UREBDLICKHMUKA-CXSFZGCWSA-N dexamethasone Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@]2(F)[C@@H]1[C@@H]1C[C@@H](C)[C@@](C(=O)CO)(O)[C@@]1(C)C[C@@H]2O UREBDLICKHMUKA-CXSFZGCWSA-N 0.000 claims 10
- 150000007523 nucleic acids Chemical class 0.000 claims 9
- UFTFJSFQGQCHQW-UHFFFAOYSA-N triformin Chemical compound O=COCC(OC=O)COC=O UFTFJSFQGQCHQW-UHFFFAOYSA-N 0.000 claims 9
- 108020004707 nucleic acids Proteins 0.000 claims 8
- 102000039446 nucleic acids Human genes 0.000 claims 8
- 229950000812 dexamethasone palmitate Drugs 0.000 claims 7
- 239000013604 expression vector Substances 0.000 claims 7
- 238000000034 method Methods 0.000 claims 6
- -1 cationic lipid Chemical class 0.000 claims 3
- 229960003957 dexamethasone Drugs 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000002502 liposome Substances 0.000 claims 3
- 230000007935 neutral effect Effects 0.000 claims 3
- 230000000069 prophylactic effect Effects 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- KSXTUUUQYQYKCR-LQDDAWAPSA-M 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KSXTUUUQYQYKCR-LQDDAWAPSA-M 0.000 claims 2
- 108091033409 CRISPR Proteins 0.000 claims 2
- GZDFHIJNHHMENY-UHFFFAOYSA-N Dimethyl dicarbonate Chemical compound COC(=O)OC(=O)OC GZDFHIJNHHMENY-UHFFFAOYSA-N 0.000 claims 2
- 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 claims 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 2
- 239000007864 aqueous solution Substances 0.000 claims 2
- 239000008121 dextrose Substances 0.000 claims 2
- 150000002632 lipids Chemical class 0.000 claims 2
- 244000052769 pathogen Species 0.000 claims 2
- 230000001717 pathogenic effect Effects 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 239000011780 sodium chloride Substances 0.000 claims 2
- 239000002691 unilamellar liposome Substances 0.000 claims 2
- 238000010354 CRISPR gene editing Methods 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 102000004127 Cytokines Human genes 0.000 claims 1
- 108090000695 Cytokines Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 102000043129 MHC class I family Human genes 0.000 claims 1
- 108091054437 MHC class I family Proteins 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 108091007412 Piwi-interacting RNA Proteins 0.000 claims 1
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 1
- 108020003224 Small Nucleolar RNA Proteins 0.000 claims 1
- 102000042773 Small Nucleolar RNA Human genes 0.000 claims 1
- 108091027967 Small hairpin RNA Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 108091093130 Toxic Small RNA Proteins 0.000 claims 1
- 230000003321 amplification Effects 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 125000004122 cyclic group Chemical group 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 108091070501 miRNA Proteins 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 238000003199 nucleic acid amplification method Methods 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 238000011321 prophylaxis Methods 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 239000002924 silencing RNA Substances 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024093487A JP2024116286A (ja) | 2017-03-23 | 2024-06-10 | ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762475477P | 2017-03-23 | 2017-03-23 | |
| US62/475,477 | 2017-03-23 | ||
| PCT/US2018/024096 WO2018175932A1 (en) | 2017-03-23 | 2018-03-23 | Systems and methods for nucleic acid expression in vivo |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024093487A Division JP2024116286A (ja) | 2017-03-23 | 2024-06-10 | ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020511521A JP2020511521A (ja) | 2020-04-16 |
| JP2020511521A5 true JP2020511521A5 (enExample) | 2021-05-06 |
Family
ID=63585790
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019552611A Pending JP2020511521A (ja) | 2017-03-23 | 2018-03-23 | ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 |
| JP2024093487A Pending JP2024116286A (ja) | 2017-03-23 | 2024-06-10 | ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024093487A Pending JP2024116286A (ja) | 2017-03-23 | 2024-06-10 | ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US11541130B2 (enExample) |
| EP (1) | EP3600428A4 (enExample) |
| JP (2) | JP2020511521A (enExample) |
| CN (2) | CN110678202B (enExample) |
| CA (1) | CA3057320A1 (enExample) |
| WO (1) | WO2018175932A1 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| US10086089B2 (en) | 2015-09-18 | 2018-10-02 | DNARx | Systems and methods for nucleic acid expression in vivo |
| WO2017095967A2 (en) | 2015-11-30 | 2017-06-08 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| EP3600428A4 (en) * | 2017-03-23 | 2020-08-26 | Dnarx | SYSTEMS AND METHODS FOR THE EXPRESSION OF NUCLEIC ACIDS IN VIVO |
| WO2018209270A1 (en) | 2017-05-11 | 2018-11-15 | Northwestern University | Adoptive cell therapy using spherical nucleic acids (snas) |
| MX2019014379A (es) * | 2017-05-31 | 2020-01-23 | Univ North Carolina Chapel Hill | Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso. |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| SG11202108451VA (en) * | 2019-04-03 | 2021-09-29 | Regeneron Pharma | Methods and compositions for insertion of antibody coding sequences into a safe harbor locus |
| WO2021253180A1 (en) | 2020-06-15 | 2021-12-23 | Novartis Ag | Methyl (r) -2- (fluoromethyl) -5-oxo-4-phenyl-4, 5, 6, 7-tetrahydro-1h-cyclopenta [b] pyridine-3-carboxylate and methyl (r) -2- (fluoromethyl) -5-oxo-4-phenyl-1, 4, 5, 7-tetrahydrofuro [3, 4-b] pyridine-3-carboxylate as cav1.2 activators |
| FR3111913A1 (fr) * | 2020-06-30 | 2021-12-31 | Eyevensys | Construction d’adn pour le traitement de pathologies oculaires |
| CN111863118B (zh) * | 2020-07-20 | 2023-09-05 | 湖南莱博赛医用机器人有限公司 | 基于tct制片进行tct和dna倍体分析的方法 |
| US11124568B1 (en) * | 2020-08-19 | 2021-09-21 | Vitruviae LLC | CD3/CD25 antibodies for neuro-immune diseases |
| CA3192229A1 (en) * | 2020-09-25 | 2022-03-31 | DNARx | Systems and methods for expressing biomolecules in a subject |
| US12071633B2 (en) * | 2020-10-13 | 2024-08-27 | Kriya Therapeutics, Inc. | Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer |
| US20230212610A1 (en) * | 2021-12-20 | 2023-07-06 | DNARx | Nucleic acid expression using subcutaneous administration |
| CN115992211B (zh) * | 2022-08-10 | 2025-07-29 | 南京鼓楼医院 | 一种用于狼疮性肾炎诊断的血清外泌体tsRNA标志物、探针及其应用 |
| KR20250107834A (ko) * | 2022-11-11 | 2025-07-14 | 빔 테라퓨틱스, 인크. | 트랜스티레틴 유전자의 염기 편집 |
| CN115851911B (zh) * | 2022-11-25 | 2025-10-24 | 南京鼓楼医院 | 一种用于鉴别狼疮性肾炎的尿液外泌体tsRNA标志物和探针及其应用 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6806084B1 (en) | 1992-06-04 | 2004-10-19 | The Regents Of The University Of California | Methods for compositions for in vivo gene delivery |
| US7279313B2 (en) * | 1995-09-15 | 2007-10-09 | Centelion | Circular DNA molecule having a conditional origin of replication, process for their preparation and their use in gene therapy |
| US6133026A (en) | 1996-05-31 | 2000-10-17 | Sequus Pharmaceuticals, Inc. | Condensed plasmid-liposome complex for transfection |
| AU730771B2 (en) | 1996-08-19 | 2001-03-15 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Novel liposome complexes for increased systemic delivery |
| US6074667A (en) * | 1996-11-20 | 2000-06-13 | Kinnunen; Paavo | Liposomal transfection method |
| WO1999036514A1 (en) * | 1998-01-16 | 1999-07-22 | California Pacific Medical Center Research Institute | Methods and compositions for gene delivery |
| GB9813100D0 (en) | 1998-06-18 | 1998-08-19 | Secr Defence | Method of forming liposomes |
| US20020065236A1 (en) * | 1998-09-09 | 2002-05-30 | Yew Nelson S. | CpG reduced plasmids and viral vectors |
| JP2002524473A (ja) | 1998-09-09 | 2002-08-06 | ジエンザイム コーポレイション | プラスミドベクターのメチル化 |
| WO2001052903A1 (en) * | 2000-01-19 | 2001-07-26 | Genteric, Inc. | Method for nucleic acid transfection of cells |
| AU2001259370A1 (en) | 2000-05-09 | 2001-11-20 | Isis Pharmaceuticals, Inc. | Methods of obtaining active antisense compounds |
| US7083782B2 (en) | 2000-07-19 | 2006-08-01 | Pepgen Corporation | Method of treatment using interferon-tau |
| DE60115070T2 (de) * | 2000-09-18 | 2006-07-27 | Genzyme Corp., Cambridge | Expressionsvektoren mit hybriden ubiquitin-promotoren |
| US20020108132A1 (en) | 2001-02-02 | 2002-08-08 | Avigenics Inc. | Production of a monoclonal antibody by a transgenic chicken |
| FR2821855B1 (fr) | 2001-03-09 | 2004-04-02 | Cayla | Genes synthetiques et plasmides bacteriens depourvus de cpg |
| JP5424521B2 (ja) | 2001-12-21 | 2014-02-26 | ヒューマン ジノーム サイエンシーズ， インコーポレイテッド | アルブミン融合タンパク質 |
| EP2395017A3 (en) * | 2003-05-30 | 2012-12-19 | Merus B.V. | Design and use of paired variable regions of specific binding molecules |
| WO2005084180A2 (en) | 2003-12-19 | 2005-09-15 | University Of Cincinnati | Polyamides and polyamide complexes for delivery of oligonucleotide decoys |
| EP1799859B1 (en) | 2004-09-17 | 2014-07-02 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| US20070087045A1 (en) * | 2005-10-14 | 2007-04-19 | Industrial Technology Research Institute | Lipid carrier and method of preparing the same |
| EP2505646A1 (en) | 2006-05-05 | 2012-10-03 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of CRP |
| EP2061514B1 (en) | 2006-08-29 | 2014-11-26 | Council of Scientific & Industrial Research | An aqueous formulation for possible selective targeting and delivering gene to cancer cells |
| CA2703852A1 (en) * | 2007-11-09 | 2009-05-14 | Northeastern University | Self-assembling micelle-like nanoparticles for systemic gene delivery |
| WO2011008904A1 (en) | 2009-07-17 | 2011-01-20 | Tabor Aaron T | Compositions and methods for genetic modification of cells having cosmetic function to enhance cosmetic appearance |
| CN101812177B (zh) | 2010-01-05 | 2012-01-25 | 中国药科大学 | 类固醇激素修饰的阳离子聚合物及其基因复合物的制备 |
| CN102366411B (zh) | 2011-09-14 | 2012-12-19 | 海南灵康制药有限公司 | 一种地塞米松棕榈酸酯脂质体注射液 |
| EP2638896A1 (en) * | 2012-03-14 | 2013-09-18 | Bioneer A/S | Cationic liposomal drug delivery system for specific targeting of human cd14+ monocytes in whole blood |
| US20140120157A1 (en) | 2012-09-19 | 2014-05-01 | Georgetown University | Targeted liposomes |
| WO2014100073A2 (en) | 2012-12-21 | 2014-06-26 | Merck Sharp & Dohme Corp. | Expression vectors for recombinant protein production in mammalian cells |
| KR20160056869A (ko) * | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| WO2016028682A1 (en) * | 2014-08-17 | 2016-02-25 | The Broad Institute Inc. | Genome editing using cas9 nickases |
| EP3226892A4 (en) * | 2014-12-01 | 2018-07-18 | The Trustees Of The University Of Pennsylvania | Dna antibody constructs and method of using same |
| US10086089B2 (en) | 2015-09-18 | 2018-10-02 | DNARx | Systems and methods for nucleic acid expression in vivo |
| EP3600428A4 (en) | 2017-03-23 | 2020-08-26 | Dnarx | SYSTEMS AND METHODS FOR THE EXPRESSION OF NUCLEIC ACIDS IN VIVO |
| CA3192229A1 (en) | 2020-09-25 | 2022-03-31 | DNARx | Systems and methods for expressing biomolecules in a subject |
-
2018
- 2018-03-23 EP EP18770192.5A patent/EP3600428A4/en active Pending
- 2018-03-23 US US15/934,587 patent/US11541130B2/en active Active
- 2018-03-23 CA CA3057320A patent/CA3057320A1/en active Pending
- 2018-03-23 JP JP2019552611A patent/JP2020511521A/ja active Pending
- 2018-03-23 WO PCT/US2018/024096 patent/WO2018175932A1/en not_active Ceased
- 2018-03-23 CN CN201880027828.2A patent/CN110678202B/zh active Active
- 2018-03-23 CN CN202310347530.2A patent/CN116392609A/zh active Pending
-
2022
- 2022-10-27 US US18/050,389 patent/US20240033376A1/en active Pending
-
2024
- 2024-06-10 JP JP2024093487A patent/JP2024116286A/ja active Pending
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