JP2018510648A5 - - Google Patents
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- Publication number
- JP2018510648A5 JP2018510648A5 JP2017553005A JP2017553005A JP2018510648A5 JP 2018510648 A5 JP2018510648 A5 JP 2018510648A5 JP 2017553005 A JP2017553005 A JP 2017553005A JP 2017553005 A JP2017553005 A JP 2017553005A JP 2018510648 A5 JP2018510648 A5 JP 2018510648A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- optionally
- aav2
- raav
- oversized
- 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
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 45
- 239000002245 particle Substances 0.000 claims 42
- 210000000234 capsid Anatomy 0.000 claims 25
- 108700019146 Transgenes Proteins 0.000 claims 24
- 230000008488 polyadenylation Effects 0.000 claims 17
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 12
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 12
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 12
- 101000772176 Mus musculus Transthyretin Proteins 0.000 claims 11
- 238000000034 method Methods 0.000 claims 11
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 claims 9
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims 9
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 8
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 8
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 8
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 8
- 241000283690 Bos taurus Species 0.000 claims 8
- 108091092195 Intron Proteins 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 8
- 150000007523 nucleic acids Chemical class 0.000 claims 8
- 102000039446 nucleic acids Human genes 0.000 claims 8
- 241000283707 Capra Species 0.000 claims 7
- 239000000203 mixture Substances 0.000 claims 7
- 101150044789 Cap gene Proteins 0.000 claims 6
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 6
- 108010006025 bovine growth hormone Proteins 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 6
- 208000035475 disorder Diseases 0.000 claims 6
- 101150066583 rep gene Proteins 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 108010069091 Dystrophin Proteins 0.000 claims 5
- 102000001039 Dystrophin Human genes 0.000 claims 5
- 108010054218 Factor VIII Proteins 0.000 claims 5
- 102000001690 Factor VIII Human genes 0.000 claims 5
- 229960000301 factor viii Drugs 0.000 claims 5
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 4
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 4
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 4
- -1 dysferrin Proteins 0.000 claims 4
- 239000013646 rAAV2 vector Substances 0.000 claims 4
- 230000001225 therapeutic effect Effects 0.000 claims 4
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 3
- 241000288906 Primates Species 0.000 claims 3
- 101100409194 Rattus norvegicus Ppargc1b gene Proteins 0.000 claims 3
- 102000057593 human F8 Human genes 0.000 claims 3
- 239000000725 suspension Substances 0.000 claims 3
- 238000003146 transient transfection Methods 0.000 claims 3
- 241000701161 unidentified adenovirus Species 0.000 claims 3
- 241000701447 unidentified baculovirus Species 0.000 claims 3
- 238000012217 deletion Methods 0.000 claims 2
- 230000037430 deletion Effects 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 238000000746 purification Methods 0.000 claims 2
- 239000013608 rAAV vector Substances 0.000 claims 2
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 102000004168 Dysferlin Human genes 0.000 claims 1
- 108090000620 Dysferlin Proteins 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 208000022583 Qualitative or quantitative defects of dysferlin Diseases 0.000 claims 1
- 238000011210 chromatographic step Methods 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021075526A JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2023097370A JP2023116678A (ja) | 2015-04-08 | 2023-06-14 | オーバーサイズアデノ随伴ベクターの産生 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562144862P | 2015-04-08 | 2015-04-08 | |
| US62/144,862 | 2015-04-08 | ||
| US201562220067P | 2015-09-17 | 2015-09-17 | |
| US62/220,067 | 2015-09-17 | ||
| PCT/US2016/026486 WO2016164609A2 (en) | 2015-04-08 | 2016-04-07 | Production of oversized adeno-associated vectors |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021075526A Division JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018510648A JP2018510648A (ja) | 2018-04-19 |
| JP2018510648A5 true JP2018510648A5 (enExample) | 2019-05-09 |
| JP6878299B2 JP6878299B2 (ja) | 2021-05-26 |
Family
ID=56943906
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017553005A Active JP6878299B2 (ja) | 2015-04-08 | 2016-04-07 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2021075526A Withdrawn JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2023097370A Pending JP2023116678A (ja) | 2015-04-08 | 2023-06-14 | オーバーサイズアデノ随伴ベクターの産生 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021075526A Withdrawn JP2021118724A (ja) | 2015-04-08 | 2021-04-28 | オーバーサイズアデノ随伴ベクターの産生 |
| JP2023097370A Pending JP2023116678A (ja) | 2015-04-08 | 2023-06-14 | オーバーサイズアデノ随伴ベクターの産生 |
Country Status (27)
| Country | Link |
|---|---|
| US (1) | US10815497B2 (enExample) |
| EP (2) | EP3280799B1 (enExample) |
| JP (3) | JP6878299B2 (enExample) |
| KR (2) | KR102848324B1 (enExample) |
| CN (1) | CN107864657A (enExample) |
| AU (3) | AU2016245806A1 (enExample) |
| BR (1) | BR112017021505A2 (enExample) |
| CA (1) | CA2982123A1 (enExample) |
| CL (1) | CL2017002537A1 (enExample) |
| CO (1) | CO2017011344A2 (enExample) |
| CR (1) | CR20170505A (enExample) |
| DK (1) | DK3280799T3 (enExample) |
| EA (1) | EA201792236A1 (enExample) |
| EC (1) | ECSP17074016A (enExample) |
| ES (1) | ES3008382T3 (enExample) |
| GT (1) | GT201700214A (enExample) |
| HU (1) | HUE069779T2 (enExample) |
| IL (3) | IL283291B (enExample) |
| MX (1) | MX2017012935A (enExample) |
| PE (1) | PE20171800A1 (enExample) |
| PH (1) | PH12017501836A1 (enExample) |
| PL (1) | PL3280799T3 (enExample) |
| SG (3) | SG10201912912QA (enExample) |
| TN (1) | TN2017000431A1 (enExample) |
| TW (1) | TWI707951B (enExample) |
| UY (1) | UY36611A (enExample) |
| WO (1) | WO2016164609A2 (enExample) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2956477T4 (da) | 2013-02-15 | 2024-04-15 | Bioverativ Therapeutics Inc | Optimeret faktor viii-gen |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| RU2020108189A (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| BR112018006074A2 (pt) | 2015-09-24 | 2018-10-09 | Biomarin Pharm Inc | vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas |
| KR20180118659A (ko) | 2016-02-01 | 2018-10-31 | 바이오버라티브 테라퓨틱스 인크. | 최적화된 viii 인자 유전자 |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics, Inc. | composições e métodos de tratamento da doença de huntington |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| WO2018154096A1 (en) | 2017-02-24 | 2018-08-30 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin | Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| KR102636351B1 (ko) | 2017-05-19 | 2024-02-15 | 엔코디드 테라퓨틱스, 인크. | 고활성 조절 요소 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| WO2019028306A2 (en) | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES |
| TWI904068B (zh) | 2017-08-09 | 2025-11-11 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| SG11202004926WA (en) | 2017-12-01 | 2020-06-29 | Encoded Therapeutics Inc | Engineered dna binding proteins |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| US20210338838A1 (en) * | 2018-07-20 | 2021-11-04 | University Of Utah Research Foundation | Gene therapy for macular degeneration |
| WO2020033863A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP4461814A3 (en) * | 2018-10-12 | 2025-02-26 | Genzyme Corporation | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
| WO2021042004A1 (en) * | 2019-08-29 | 2021-03-04 | Siemens Healthcare Diagnostics Inc. | Reagents and methods for detecting aav shedding |
| TW202126284A (zh) | 2019-09-30 | 2021-07-16 | 美商百歐維拉提夫治療公司 | 慢病毒載體配製物 |
| CN111218446B (zh) * | 2019-12-25 | 2023-03-28 | 劲帆生物医药科技（武汉）有限公司 | 一种肝脏特异性启动子及其应用 |
| IL296509A (en) * | 2020-03-16 | 2022-11-01 | Univ North Carolina Chapel Hill | Preparations and methods for selective detection of tumor-derived viral DNA |
| WO2021262963A1 (en) | 2020-06-24 | 2021-12-30 | Bioverativ Therapeutics Inc. | Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein |
| JP2023536148A (ja) | 2020-07-30 | 2023-08-23 | シェイプ セラピューティクス インク． | Ｒａａｖウイルス粒子の誘導産生のための安定な細胞株 |
| BR112023002395A2 (pt) | 2020-08-23 | 2023-03-21 | Bioverativ Therapeutics Inc | Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna) |
| CA3229668A1 (en) | 2021-08-23 | 2023-03-02 | Bioverativ Therapeutics Inc. | Baculovirus expression system |
| AR126845A1 (es) | 2021-08-23 | 2023-11-22 | Bioverativ Therapeutics Inc | Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida |
| KR20240049332A (ko) | 2021-08-23 | 2024-04-16 | 바이오버라티브 테라퓨틱스 인크. | 최적화된 인자 viii 유전자 |
| EP4408874A1 (en) | 2021-09-30 | 2024-08-07 | Bioverativ Therapeutics Inc. | Nucleic acids encoding factor viii polypeptides with reduced immunogenicity |
| JP2024537896A (ja) | 2021-10-18 | 2024-10-16 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | アデノウイルス随伴ウイルスポリヌクレオチドを含む真核細胞 |
| TW202509211A (zh) * | 2023-04-26 | 2025-03-01 | 法商賽諾菲公司 | 宿主細胞系以及鑑定和使用該等宿主細胞系之方法 |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| WO2001027303A1 (en) * | 1999-10-12 | 2001-04-19 | The University Of North Carolina At Chapel Hill | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| 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 |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| US20040092008A1 (en) * | 2002-11-12 | 2004-05-13 | Snyder Richard O. | Recombinant AAV vectors for gene therapy of hemophilia A |
| US7510872B2 (en) | 2003-02-26 | 2009-03-31 | Nationwide Children's Hospital | Recombinant adeno-associated virus production |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| HUE040636T2 (hu) | 2009-06-16 | 2019-03-28 | Genzyme Corp | Javított eljárások rekombináns AAV-vektorok tisztítására |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| ES2724800T3 (es) | 2011-02-17 | 2019-09-16 | Univ Pennsylvania | Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9 |
| DK2911687T3 (da) | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| ES2813698T3 (es) | 2013-09-12 | 2021-03-24 | Biomarin Pharm Inc | Vectores de AAV que comprenden un gen que codifica el factor VIII |
| WO2015168666A2 (en) | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| NZ772772A (en) | 2015-02-10 | 2024-12-20 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
-
2016
- 2016-04-06 TW TW105110751A patent/TWI707951B/zh active
- 2016-04-07 IL IL283291A patent/IL283291B/en unknown
- 2016-04-07 CR CR20170505A patent/CR20170505A/es unknown
- 2016-04-07 BR BR112017021505A patent/BR112017021505A2/pt not_active Application Discontinuation
- 2016-04-07 KR KR1020177032062A patent/KR102848324B1/ko active Active
- 2016-04-07 CN CN201680033119.6A patent/CN107864657A/zh active Pending
- 2016-04-07 SG SG10201912912QA patent/SG10201912912QA/en unknown
- 2016-04-07 US US15/565,020 patent/US10815497B2/en active Active
- 2016-04-07 CA CA2982123A patent/CA2982123A1/en active Pending
- 2016-04-07 JP JP2017553005A patent/JP6878299B2/ja active Active
- 2016-04-07 PL PL16767050.4T patent/PL3280799T3/pl unknown
- 2016-04-07 KR KR1020257027291A patent/KR20250126875A/ko active Pending
- 2016-04-07 MX MX2017012935A patent/MX2017012935A/es unknown
- 2016-04-07 EP EP16767050.4A patent/EP3280799B1/en active Active
- 2016-04-07 ES ES16767050T patent/ES3008382T3/es active Active
- 2016-04-07 WO PCT/US2016/026486 patent/WO2016164609A2/en not_active Ceased
- 2016-04-07 TN TNP/2017/000431A patent/TN2017000431A1/en unknown
- 2016-04-07 AU AU2016245806A patent/AU2016245806A1/en not_active Abandoned
- 2016-04-07 SG SG11201708203UA patent/SG11201708203UA/en unknown
- 2016-04-07 SG SG10201909381W patent/SG10201909381WA/en unknown
- 2016-04-07 DK DK16767050.4T patent/DK3280799T3/da active
- 2016-04-07 HU HUE16767050A patent/HUE069779T2/hu unknown
- 2016-04-07 IL IL294965A patent/IL294965A/en unknown
- 2016-04-07 EP EP24207442.5A patent/EP4512429A3/en active Pending
- 2016-04-07 EA EA201792236A patent/EA201792236A1/ru unknown
- 2016-04-07 PE PE2017001997A patent/PE20171800A1/es unknown
- 2016-04-08 UY UY0001036611A patent/UY36611A/es not_active Application Discontinuation
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2017
- 2017-10-06 CL CL2017002537A patent/CL2017002537A1/es unknown
- 2017-10-06 GT GT201700214A patent/GT201700214A/es unknown
- 2017-10-06 PH PH12017501836A patent/PH12017501836A1/en unknown
- 2017-10-08 IL IL254933A patent/IL254933B/en active IP Right Grant
- 2017-11-03 CO CONC2017/0011344A patent/CO2017011344A2/es unknown
- 2017-11-08 EC ECIEPI201774016A patent/ECSP17074016A/es unknown
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2021
- 2021-04-28 JP JP2021075526A patent/JP2021118724A/ja not_active Withdrawn
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2022
- 2022-06-07 AU AU2022203942A patent/AU2022203942B2/en active Active
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2023
- 2023-06-14 JP JP2023097370A patent/JP2023116678A/ja active Pending
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2024
- 2024-03-12 AU AU2024201619A patent/AU2024201619A1/en active Pending