JPWO2019161059A5 - - Google Patents
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- Publication number
- JPWO2019161059A5 JPWO2019161059A5 JP2020543344A JP2020543344A JPWO2019161059A5 JP WO2019161059 A5 JPWO2019161059 A5 JP WO2019161059A5 JP 2020543344 A JP2020543344 A JP 2020543344A JP 2020543344 A JP2020543344 A JP 2020543344A JP WO2019161059 A5 JPWO2019161059 A5 JP WO2019161059A5
- Authority
- JP
- Japan
- Prior art keywords
- antibody
- cedna vector
- vector according
- nucleotide sequence
- heterologous nucleotide
- 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 35
- 125000003729 nucleotide group Chemical group 0.000 claims description 35
- 102000037865 fusion proteins Human genes 0.000 claims description 25
- 108020001507 fusion proteins Proteins 0.000 claims description 25
- 238000000034 method Methods 0.000 claims description 18
- 230000001105 regulatory effect Effects 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 8
- 230000001225 therapeutic effect Effects 0.000 claims description 8
- 238000006467 substitution reaction Methods 0.000 claims description 7
- 108010003723 Single-Domain Antibodies Proteins 0.000 claims description 6
- 150000002632 lipids Chemical class 0.000 claims description 6
- 239000000203 mixture Substances 0.000 claims description 6
- 108020003175 receptors Proteins 0.000 claims description 6
- 102000005962 receptors Human genes 0.000 claims description 6
- 230000003248 secreting effect Effects 0.000 claims description 6
- 238000011144 upstream manufacturing Methods 0.000 claims description 6
- 241000700605 Viruses Species 0.000 claims description 4
- 239000002299 complementary DNA Substances 0.000 claims description 3
- 230000003612 virological effect Effects 0.000 claims description 3
- 102100021886 Activin receptor type-2A Human genes 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 108010084313 CD58 Antigens Proteins 0.000 claims description 2
- 102000008203 CTLA-4 Antigen Human genes 0.000 claims description 2
- 108010021064 CTLA-4 Antigen Proteins 0.000 claims description 2
- 229940045513 CTLA4 antagonist Drugs 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 101000970954 Homo sapiens Activin receptor type-2A Proteins 0.000 claims description 2
- 101000851018 Homo sapiens Vascular endothelial growth factor receptor 1 Proteins 0.000 claims description 2
- 101000851007 Homo sapiens Vascular endothelial growth factor receptor 2 Proteins 0.000 claims description 2
- 102100039880 Interleukin-1 receptor accessory protein Human genes 0.000 claims description 2
- 101710180389 Interleukin-1 receptor accessory protein Proteins 0.000 claims description 2
- 241000125945 Protoparvovirus Species 0.000 claims description 2
- 108060008683 Tumor Necrosis Factor Receptor Proteins 0.000 claims description 2
- 102100033178 Vascular endothelial growth factor receptor 1 Human genes 0.000 claims description 2
- 102100033177 Vascular endothelial growth factor receptor 2 Human genes 0.000 claims description 2
- 230000015556 catabolic process Effects 0.000 claims description 2
- 238000012258 culturing Methods 0.000 claims description 2
- 238000006731 degradation reaction Methods 0.000 claims description 2
- 230000001419 dependent effect Effects 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims description 2
- 239000002105 nanoparticle Substances 0.000 claims description 2
- 102000003298 tumor necrosis factor receptor Human genes 0.000 claims description 2
- 208000007056 sickle cell anemia Diseases 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 9
- 210000004027 cell Anatomy 0.000 description 9
- 238000012217 deletion Methods 0.000 description 8
- 230000037430 deletion Effects 0.000 description 8
- 238000003780 insertion Methods 0.000 description 4
- 230000037431 insertion Effects 0.000 description 4
- 206010028980 Neoplasm Diseases 0.000 description 3
- 208000023275 Autoimmune disease Diseases 0.000 description 2
- 201000011510 cancer Diseases 0.000 description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 2
- 210000003527 eukaryotic cell Anatomy 0.000 description 2
- 208000015122 neurodegenerative disease Diseases 0.000 description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 1
- 241000649045 Adeno-associated virus 10 Species 0.000 description 1
- 241000649046 Adeno-associated virus 11 Species 0.000 description 1
- 241000649047 Adeno-associated virus 12 Species 0.000 description 1
- 208000024827 Alzheimer disease Diseases 0.000 description 1
- 208000011231 Crohn disease Diseases 0.000 description 1
- 206010012438 Dermatitis atopic Diseases 0.000 description 1
- 208000031220 Hemophilia Diseases 0.000 description 1
- 208000009292 Hemophilia A Diseases 0.000 description 1
- 208000035150 Hypercholesterolemia Diseases 0.000 description 1
- 206010021143 Hypoxia Diseases 0.000 description 1
- 206010025323 Lymphomas Diseases 0.000 description 1
- 201000004681 Psoriasis Diseases 0.000 description 1
- 206010039491 Sarcoma Diseases 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 230000001154 acute effect Effects 0.000 description 1
- 238000007792 addition Methods 0.000 description 1
- 208000006673 asthma Diseases 0.000 description 1
- 201000008937 atopic dermatitis Diseases 0.000 description 1
- 201000010099 disease Diseases 0.000 description 1
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- 230000001146 hypoxic effect Effects 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 230000028709 inflammatory response Effects 0.000 description 1
- 208000032839 leukemia Diseases 0.000 description 1
- 230000001404 mediated effect Effects 0.000 description 1
- 201000006417 multiple sclerosis Diseases 0.000 description 1
- 102000039446 nucleic acids Human genes 0.000 description 1
- 108020004707 nucleic acids Proteins 0.000 description 1
- 150000007523 nucleic acids Chemical class 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 208000001685 postmenopausal osteoporosis Diseases 0.000 description 1
- 230000001124 posttranscriptional effect Effects 0.000 description 1
- 230000005855 radiation Effects 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 206010039073 rheumatoid arthritis Diseases 0.000 description 1
- 150000003384 small molecules Chemical class 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024141968A JP2024167281A (ja) | 2018-02-14 | 2024-08-23 | 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用 |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862630676P | 2018-02-14 | 2018-02-14 | |
| US201862630670P | 2018-02-14 | 2018-02-14 | |
| US62/630,676 | 2018-02-14 | ||
| US62/630,670 | 2018-02-14 | ||
| US201862680087P | 2018-06-04 | 2018-06-04 | |
| US201862680092P | 2018-06-04 | 2018-06-04 | |
| US62/680,087 | 2018-06-04 | ||
| US62/680,092 | 2018-06-04 | ||
| PCT/US2019/018016 WO2019161059A1 (en) | 2018-02-14 | 2019-02-14 | Non-viral dna vectors and uses thereof for antibody and fusion protein production |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024141968A Division JP2024167281A (ja) | 2018-02-14 | 2024-08-23 | 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021513355A JP2021513355A (ja) | 2021-05-27 |
| JP2021513355A5 JP2021513355A5 (https=) | 2022-02-22 |
| JPWO2019161059A5 true JPWO2019161059A5 (https=) | 2022-02-22 |
Family
ID=67619630
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020543344A Pending JP2021513355A (ja) | 2018-02-14 | 2019-02-14 | 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用 |
| JP2024141968A Pending JP2024167281A (ja) | 2018-02-14 | 2024-08-23 | 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024141968A Pending JP2024167281A (ja) | 2018-02-14 | 2024-08-23 | 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20220042035A1 (https=) |
| EP (1) | EP3752191A4 (https=) |
| JP (2) | JP2021513355A (https=) |
| KR (1) | KR20200120649A (https=) |
| CN (1) | CN111818942A (https=) |
| AU (1) | AU2019221642A1 (https=) |
| BR (1) | BR112020016288A2 (https=) |
| CA (1) | CA3091250A1 (https=) |
| IL (1) | IL276469A (https=) |
| MA (1) | MA51842A (https=) |
| MX (1) | MX2020008470A (https=) |
| PH (1) | PH12020551039A1 (https=) |
| SG (1) | SG11202006431WA (https=) |
| WO (1) | WO2019161059A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW202545984A (zh) | 2017-08-09 | 2025-12-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| SG11202005271TA (en) * | 2018-01-19 | 2020-07-29 | Generation Bio Co | Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors |
| WO2020033863A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| JP7700101B2 (ja) * | 2019-09-06 | 2025-06-30 | ジェネレーション バイオ カンパニー | 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法 |
| WO2021169167A1 (en) * | 2020-02-29 | 2021-09-02 | Nanjing GenScript Biotech Co., Ltd. | Method for treating coronavirus infections |
| DE102020111571A1 (de) * | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| AU2021244559A1 (en) * | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| WO2022153957A1 (ja) * | 2021-01-12 | 2022-07-21 | Ｊｃｒファーマ株式会社 | リガンドと生理活性を有する蛋白質の融合蛋白質をコードする遺伝子が組み込まれた核酸分子 |
| US20240092885A1 (en) * | 2021-01-26 | 2024-03-21 | Kriya Therapeutics, Inc. | Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same |
| CN117881786A (zh) * | 2021-04-27 | 2024-04-12 | 世代生物公司 | 表达治疗性抗体的非病毒dna载体及其用途 |
| WO2022236016A1 (en) * | 2021-05-07 | 2022-11-10 | Generation Bio Co. | Lyophilized non-viral dna vector compositions and uses thereof |
| WO2023091708A1 (en) * | 2021-11-18 | 2023-05-25 | The Brigham And Women's Hospital, Inc. | Induced proteinopathy models |
| CN118186005A (zh) * | 2022-12-12 | 2024-06-14 | 苏州荷光科汇生物科技有限公司 | 一种适于阿达木单抗的表达盒及其载体与应用 |
| US11993783B1 (en) * | 2023-03-27 | 2024-05-28 | Genecraft Inc. | Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof |
| CN116549632B (zh) * | 2023-03-28 | 2024-09-10 | 中国人民解放军军事科学院军事医学研究院 | 抗Lingo-1抗体Opicinumab的新用途及针对新用途的药物 |
| WO2024234869A1 (en) * | 2023-05-15 | 2024-11-21 | Immunecare Biopharmaceuticals (Shanghai) Co., Ltd. | Recombinant fusion protein for treatment of pulmonary hypertension |
| WO2025092951A1 (en) * | 2023-11-03 | 2025-05-08 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant adeno-associated viral vectors for treating fabry disease |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ522840A (en) * | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| KR100968664B1 (ko) * | 2001-08-27 | 2010-07-06 | 제넨테크, 인크. | 항체 발현 및 조립을 위한 시스템 |
| GB0903207D0 (en) * | 2009-02-25 | 2009-04-08 | Ucb Pharma Sa | Method for expressing multimeric proteins |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| US9447433B2 (en) * | 2013-03-15 | 2016-09-20 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
| TWI687225B (zh) * | 2014-02-06 | 2020-03-11 | 美商健臻公司 | 用於治療及預防黃斑部病變的組成物及方法 |
| WO2015191508A1 (en) * | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2017152149A1 (en) * | 2016-03-03 | 2017-09-08 | University Of Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
| US11549125B2 (en) * | 2017-08-09 | 2023-01-10 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof |
| AU2018330208B2 (en) * | 2017-09-08 | 2025-04-17 | Generation Bio Co. | Lipid nanoparticle formulations of non-viral, capsid-free DNA vectors |
| JP2020532981A (ja) * | 2017-09-08 | 2020-11-19 | ジェネレーション バイオ カンパニー | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
| BR112020007405A2 (pt) * | 2017-10-18 | 2020-12-08 | Regenxbio Inc. | Produtos terapêuticos de anticorpo pós-translacionalmente modificados totalmente humanos |
| KR20200093635A (ko) * | 2017-12-06 | 2020-08-05 | 제너레이션 바이오 컴퍼니 | 변형된 폐쇄된 말단 dna (cedna)를 사용한 유전자 편집 |
| SG11202005271TA (en) * | 2018-01-19 | 2020-07-29 | Generation Bio Co | Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors |
| US20210054405A1 (en) * | 2018-03-02 | 2021-02-25 | Generation Bio Co. | Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes |
-
2019
- 2019-02-14 MA MA051842A patent/MA51842A/fr unknown
- 2019-02-14 WO PCT/US2019/018016 patent/WO2019161059A1/en not_active Ceased
- 2019-02-14 SG SG11202006431WA patent/SG11202006431WA/en unknown
- 2019-02-14 JP JP2020543344A patent/JP2021513355A/ja active Pending
- 2019-02-14 CA CA3091250A patent/CA3091250A1/en active Pending
- 2019-02-14 EP EP19753655.0A patent/EP3752191A4/en active Pending
- 2019-02-14 CN CN201980013028.XA patent/CN111818942A/zh active Pending
- 2019-02-14 KR KR1020207024274A patent/KR20200120649A/ko not_active Ceased
- 2019-02-14 US US16/968,990 patent/US20220042035A1/en not_active Abandoned
- 2019-02-14 BR BR112020016288-4A patent/BR112020016288A2/pt unknown
- 2019-02-14 AU AU2019221642A patent/AU2019221642A1/en not_active Abandoned
- 2019-02-14 MX MX2020008470A patent/MX2020008470A/es unknown
-
2020
- 2020-07-03 PH PH12020551039A patent/PH12020551039A1/en unknown
- 2020-08-03 IL IL276469A patent/IL276469A/en unknown
-
2024
- 2024-08-23 JP JP2024141968A patent/JP2024167281A/ja active Pending
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