JP2021019591A5 - - Google Patents
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- JP2021019591A5 JP2021019591A5 JP2020156320A JP2020156320A JP2021019591A5 JP 2021019591 A5 JP2021019591 A5 JP 2021019591A5 JP 2020156320 A JP2020156320 A JP 2020156320A JP 2020156320 A JP2020156320 A JP 2020156320A JP 2021019591 A5 JP2021019591 A5 JP 2021019591A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- acid sequence
- vector according
- aav2
- 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 44
- 239000013598 vector Substances 0.000 claims 26
- 108020004707 nucleic acids Proteins 0.000 claims 23
- 102000039446 nucleic acids Human genes 0.000 claims 23
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 21
- 102000003869 Frataxin Human genes 0.000 claims 17
- 108090000217 Frataxin Proteins 0.000 claims 17
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 11
- 210000000234 capsid Anatomy 0.000 claims 11
- 239000013608 rAAV vector Substances 0.000 claims 11
- 208000024412 Friedreich ataxia Diseases 0.000 claims 8
- 239000003814 drug Substances 0.000 claims 8
- 229940124597 therapeutic agent Drugs 0.000 claims 8
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 7
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 108700042658 GAP-43 Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 238000007917 intracranial administration Methods 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 102000008186 Collagen Human genes 0.000 claims 1
- 108010035532 Collagen Proteins 0.000 claims 1
- 241000701022 Cytomegalovirus Species 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 230000006978 adaptation Effects 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 229920001436 collagen Polymers 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 230000000087 stabilizing effect Effects 0.000 claims 1
- 238000004114 suspension culture Methods 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562251288P | 2015-11-05 | 2015-11-05 | |
| US62/251,288 | 2015-11-05 | ||
| US201662411980P | 2016-10-24 | 2016-10-24 | |
| US62/411,980 | 2016-10-24 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521395A Division JP6767483B2 (ja) | 2015-11-05 | 2016-11-01 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021019591A JP2021019591A (ja) | 2021-02-18 |
| JP2021019591A5 true JP2021019591A5 (https=) | 2021-04-08 |
Family
ID=57288476
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521395A Expired - Fee Related JP6767483B2 (ja) | 2015-11-05 | 2016-11-01 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
| JP2020156320A Pending JP2021019591A (ja) | 2015-11-05 | 2020-09-17 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521395A Expired - Fee Related JP6767483B2 (ja) | 2015-11-05 | 2016-11-01 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10548947B2 (https=) |
| EP (1) | EP3370778A1 (https=) |
| JP (2) | JP6767483B2 (https=) |
| KR (2) | KR20210005994A (https=) |
| CN (1) | CN108348621A (https=) |
| AU (2) | AU2016347887B2 (https=) |
| BR (1) | BR112018007453A2 (https=) |
| CA (1) | CA2947584A1 (https=) |
| CO (1) | CO2018004564A2 (https=) |
| HK (1) | HK1250924A1 (https=) |
| IL (1) | IL259141A (https=) |
| MX (1) | MX2018005084A (https=) |
| PE (1) | PE20181156A1 (https=) |
| PH (1) | PH12018500964A1 (https=) |
| RU (2) | RU2021102893A (https=) |
| SG (2) | SG10201912763QA (https=) |
| TW (2) | TWI743442B (https=) |
| WO (1) | WO2017077451A1 (https=) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016172659A1 (en) * | 2015-04-24 | 2016-10-27 | University Of Florida Research Foundation, Inc. | Aav vector for treatment of friedreich's ataxia |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2016190780A1 (en) | 2015-05-22 | 2016-12-01 | Dmitry Dmitrievich Genkin | Extracellular dna as a therapeutic target in neurodegeneration |
| RU2021102893A (ru) * | 2015-11-05 | 2021-03-03 | Бамбу Терапьютикс, Инк. | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| CN116059245A (zh) * | 2016-03-23 | 2023-05-05 | 加利福尼亚大学董事会 | 治疗线粒体障碍的方法 |
| US10935747B2 (en) | 2016-09-21 | 2021-03-02 | Commscope Technologies Llc | Adapter block assembly |
| CN110139933B (zh) * | 2016-11-09 | 2024-03-08 | 英特瑞克斯顿股份有限公司 | 共济蛋白表达构建体 |
| US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| WO2019076973A1 (en) * | 2017-10-17 | 2019-04-25 | Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol | VECTORS FOR THE TREATMENT OF FRIEDREICH ATAXIA |
| KR20200122320A (ko) | 2018-01-16 | 2020-10-27 | 씨엘에스 테라퓨틱스 리미티드 | 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| KR20210086601A (ko) * | 2018-08-02 | 2021-07-08 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 프리드라이히 운동실조를 치료하기 위한 그의 용도 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| KR20210049833A (ko) * | 2018-08-10 | 2021-05-06 | 로직바이오 테라퓨틱스, 인크. | Mma의 치료를 위한 비-붕괴적 유전자 요법 |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| SG11202105079QA (en) * | 2018-11-14 | 2021-06-29 | Flagship Pioneering Innovations V Inc | Fusosome compositions for cns delivery |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| AU2019385506A1 (en) * | 2018-11-21 | 2021-06-03 | Takeda Pharmaceutical Company Limited | Recombinant viral vectors and nucleic acids for producing the same |
| CA3124415A1 (en) * | 2018-12-21 | 2020-06-25 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| CN114222590A (zh) * | 2019-03-27 | 2022-03-22 | 西吉隆医疗股份有限公司 | 用于因子vii疗法的组合物、装置和方法 |
| BR112021019224A2 (pt) * | 2019-03-28 | 2021-11-30 | Intellia Therapeutics Inc | Polinucleotídeos, composições e métodos para expressão de polipeptídeo |
| CA3136117A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| WO2021003195A1 (en) * | 2019-06-30 | 2021-01-07 | John Fraser Wright | Recombinant aav vectors with altered immunogencity and methods of making the same |
| AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
| CA3147034A1 (en) * | 2019-07-12 | 2021-01-21 | Cls Therapeutics Limited | Treatment of diseases associated with protein misfolding by nervous system expression of an enzyme which has a deoxyribonuclease (dnase) activity |
| JP2022548270A (ja) * | 2019-09-13 | 2022-11-17 | ラセルタ セラピューティクス， インコーポレイテッド | フリードライヒ運動失調症の治療のための組成物及び方法 |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| EP4085144A4 (en) * | 2019-12-19 | 2024-03-27 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH AAXIA |
| CN113755524B (zh) * | 2020-06-02 | 2023-11-03 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途 |
| US11946065B2 (en) | 2020-07-29 | 2024-04-02 | The Board Of Regents Of The University Of Texas System | Transgene cassettes, AAV vectors, and AAV viral vectors for expression of human codon-optimized CSTB |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| JP2023548816A (ja) | 2020-11-03 | 2023-11-21 | ファイザー・インク | 陰イオン交換クロマトグラフィーによるａａｖベクターの精製方法 |
| JP2024503234A (ja) | 2020-12-23 | 2024-01-25 | ファイザー・インク | 親和性クロマトグラフィーによるａａｖベクターの精製方法 |
| US20240058477A1 (en) * | 2021-01-04 | 2024-02-22 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treatment of friedreich's ataxia |
| EP4274592A4 (en) * | 2021-01-11 | 2025-03-19 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH'S ATAXIA |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024011109A2 (en) * | 2022-07-06 | 2024-01-11 | Adverum Biotechnologies, Inc. | Compositions and methods for treatment of achromotopsia |
| CN116121350A (zh) * | 2022-12-02 | 2023-05-16 | 百码科技(深圳)有限公司 | 一种检测fxn致病基因突变的方法 |
| CN117089514A (zh) * | 2023-10-13 | 2023-11-21 | 思鹏生物科技(苏州)有限公司 | 提升hek293细胞系aav生产效率的细胞筛选驯化方法 |
| WO2026017965A1 (en) | 2024-07-19 | 2026-01-22 | The University Court Of The University Of Edinburgh | Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions |
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| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| AU707862B2 (en) | 1995-06-07 | 1999-07-22 | University Of North Carolina At Chapel Hill, The | Helper virus-free aav production |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| EP1082413B1 (en) | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| CA2349838C (en) | 1998-11-05 | 2011-06-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| 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 |
| AU2002240890A1 (en) | 2000-12-07 | 2002-06-18 | Universite De Nantes | Inducible highly productive raav packaging cell-lines |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| ES2473623T3 (es) * | 2007-08-20 | 2014-07-07 | Glaxo Group Limited | Procedimiento de producción |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| JP2013526840A (ja) * | 2009-12-21 | 2013-06-27 | ファーマシーネ，インコーポレイテッド | 組み換えブチリルコリンエステラーゼおよびその切断型 |
| CA2788902C (en) * | 2010-02-08 | 2019-09-03 | The Trustees Of The University Of Pennsylvania | Nucleic acid molecules encoding rantes, and compositions comprising and methods of using the same |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
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| JP6396988B2 (ja) | 2013-03-15 | 2018-09-26 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 二重グリカン結合性ａａｖベクターのための方法および組成物 |
| MX384795B (es) | 2013-03-15 | 2025-03-14 | Childrens Hospital Philadelphia | Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso. |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| HK1222673A1 (zh) | 2013-07-12 | 2017-07-07 | The Children's Hospital Of Philadelphia | Aav载体和用於抗aav(腺相关病毒)中和抗体的检测 |
| KR20240090694A (ko) | 2013-07-22 | 2024-06-21 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| RU2021102893A (ru) * | 2015-11-05 | 2021-03-03 | Бамбу Терапьютикс, Инк. | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
-
2016
- 2016-11-01 RU RU2021102893A patent/RU2021102893A/ru unknown
- 2016-11-01 EP EP16795164.9A patent/EP3370778A1/en not_active Withdrawn
- 2016-11-01 KR KR1020217000034A patent/KR20210005994A/ko not_active Withdrawn
- 2016-11-01 KR KR1020187015819A patent/KR102200642B1/ko not_active Expired - Fee Related
- 2016-11-01 BR BR112018007453A patent/BR112018007453A2/pt not_active IP Right Cessation
- 2016-11-01 PE PE2018000726A patent/PE20181156A1/es unknown
- 2016-11-01 HK HK18110376.3A patent/HK1250924A1/zh unknown
- 2016-11-01 RU RU2018116076A patent/RU2743792C2/ru active
- 2016-11-01 US US15/340,721 patent/US10548947B2/en active Active
- 2016-11-01 AU AU2016347887A patent/AU2016347887B2/en not_active Ceased
- 2016-11-01 MX MX2018005084A patent/MX2018005084A/es unknown
- 2016-11-01 JP JP2018521395A patent/JP6767483B2/ja not_active Expired - Fee Related
- 2016-11-01 SG SG10201912763QA patent/SG10201912763QA/en unknown
- 2016-11-01 CN CN201680064440.0A patent/CN108348621A/zh active Pending
- 2016-11-01 SG SG11201802408RA patent/SG11201802408RA/en unknown
- 2016-11-01 WO PCT/IB2016/056572 patent/WO2017077451A1/en not_active Ceased
- 2016-11-04 TW TW108104049A patent/TWI743442B/zh not_active IP Right Cessation
- 2016-11-04 TW TW105135911A patent/TWI654301B/zh not_active IP Right Cessation
- 2016-11-04 CA CA2947584A patent/CA2947584A1/en active Pending
-
2018
- 2018-04-27 CO CONC2018/0004564A patent/CO2018004564A2/es unknown
- 2018-05-03 PH PH12018500964A patent/PH12018500964A1/en unknown
- 2018-05-03 IL IL259141A patent/IL259141A/en unknown
-
2019
- 2019-12-16 US US16/716,033 patent/US20200384073A1/en not_active Abandoned
-
2020
- 2020-02-11 AU AU2020200976A patent/AU2020200976B2/en not_active Ceased
- 2020-09-17 JP JP2020156320A patent/JP2021019591A/ja active Pending
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