JP2020513811A5 - - Google Patents
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- JP2020513811A5 JP2020513811A5 JP2019550767A JP2019550767A JP2020513811A5 JP 2020513811 A5 JP2020513811 A5 JP 2020513811A5 JP 2019550767 A JP2019550767 A JP 2019550767A JP 2019550767 A JP2019550767 A JP 2019550767A JP 2020513811 A5 JP2020513811 A5 JP 2020513811A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- muscular dystrophy
- item
- recombinant
- seq
- 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 description 32
- 239000002773 nucleotide Substances 0.000 claims description 27
- 125000003729 nucleotide group Chemical group 0.000 claims description 27
- 201000006938 muscular dystrophy Diseases 0.000 claims description 25
- 210000003205 muscle Anatomy 0.000 claims description 16
- 239000013598 vector Substances 0.000 claims description 9
- 239000003814 drug Substances 0.000 claims description 8
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 6
- 238000001802 infusion Methods 0.000 claims description 5
- 238000002347 injection Methods 0.000 claims description 5
- 239000007924 injection Substances 0.000 claims description 5
- 238000002054 transplantation Methods 0.000 claims description 5
- 206010016654 Fibrosis Diseases 0.000 claims description 4
- 230000004761 fibrosis Effects 0.000 claims description 4
- 238000010255 intramuscular injection Methods 0.000 claims description 4
- 238000010253 intravenous injection Methods 0.000 claims description 4
- 238000007911 parenteral administration Methods 0.000 claims description 3
- 239000003937 drug carrier Substances 0.000 claims description 2
- 239000007927 intramuscular injection Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 239000013607 AAV vector Substances 0.000 description 33
- 238000000034 method Methods 0.000 description 14
- 239000003623 enhancer Substances 0.000 description 8
- 108090000623 proteins and genes Proteins 0.000 description 6
- 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 description 5
- 239000003795 chemical substances by application Substances 0.000 description 5
- 101001044938 Dictyostelium discoideum Diacylglycerol kinase A Proteins 0.000 description 4
- 229940079593 drug Drugs 0.000 description 3
- 102000004169 proteins and genes Human genes 0.000 description 3
- 230000001105 regulatory effect Effects 0.000 description 3
- 108010085238 Actins Proteins 0.000 description 2
- 108010059343 MM Form Creatine Kinase Proteins 0.000 description 2
- 102000005604 Myosin Heavy Chains Human genes 0.000 description 2
- 108010084498 Myosin Heavy Chains Proteins 0.000 description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 108010051583 Ventricular Myosins Proteins 0.000 description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 1
- 241000702423 Adeno-associated virus - 2 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
- 241000300529 Adeno-associated virus 13 Species 0.000 description 1
- 102000004420 Creatine Kinase Human genes 0.000 description 1
- 108010042126 Creatine kinase Proteins 0.000 description 1
- 102100024108 Dystrophin Human genes 0.000 description 1
- 206010021143 Hypoxia Diseases 0.000 description 1
- 108091000080 Phosphotransferase Proteins 0.000 description 1
- 108091027981 Response element Proteins 0.000 description 1
- 102000011856 Utrophin Human genes 0.000 description 1
- 108010075653 Utrophin Proteins 0.000 description 1
- 230000000747 cardiac effect Effects 0.000 description 1
- CVSVTCORWBXHQV-UHFFFAOYSA-N creatine Chemical compound NC(=[NH2+])N(C)CC([O-])=O CVSVTCORWBXHQV-UHFFFAOYSA-N 0.000 description 1
- 239000003862 glucocorticoid Substances 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 210000000663 muscle cell Anatomy 0.000 description 1
- 102000020233 phosphotransferase Human genes 0.000 description 1
- 238000012385 systemic delivery Methods 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023019888A JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2024229759A JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762473148P | 2017-03-17 | 2017-03-17 | |
| US62/473,148 | 2017-03-17 | ||
| PCT/US2018/022881 WO2018170408A1 (en) | 2017-03-17 | 2018-03-16 | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023019888A Division JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2024229759A Division JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020513811A JP2020513811A (ja) | 2020-05-21 |
| JP2020513811A5 true JP2020513811A5 (https=) | 2021-04-22 |
| JP7676111B2 JP7676111B2 (ja) | 2025-05-14 |
Family
ID=63523321
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019550767A Active JP7676111B2 (ja) | 2017-03-17 | 2018-03-16 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2023019888A Pending JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2024229759A Pending JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023019888A Pending JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2024229759A Pending JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Country Status (33)
| Country | Link |
|---|---|
| US (2) | US20200199621A1 (https=) |
| EP (2) | EP3596222B1 (https=) |
| JP (3) | JP7676111B2 (https=) |
| KR (3) | KR102887103B1 (https=) |
| CN (2) | CN110997923B (https=) |
| AR (1) | AR111292A1 (https=) |
| AU (3) | AU2018233732B2 (https=) |
| BR (1) | BR112019019248A2 (https=) |
| CA (1) | CA3056638A1 (https=) |
| CO (1) | CO2019011250A2 (https=) |
| CY (1) | CY1126080T1 (https=) |
| DK (1) | DK3596222T5 (https=) |
| EA (1) | EA201992201A1 (https=) |
| ES (1) | ES2948233T3 (https=) |
| FI (1) | FI3596222T3 (https=) |
| HR (1) | HRP20230522T1 (https=) |
| HU (1) | HUE062476T2 (https=) |
| IL (3) | IL269391B2 (https=) |
| LT (1) | LT3596222T (https=) |
| MA (1) | MA52112B1 (https=) |
| MD (1) | MD3596222T2 (https=) |
| MX (3) | MX2019011046A (https=) |
| MY (1) | MY203547A (https=) |
| PL (1) | PL3596222T3 (https=) |
| PT (1) | PT3596222T (https=) |
| RS (1) | RS64299B1 (https=) |
| SA (1) | SA519410146B1 (https=) |
| SG (1) | SG11201908575SA (https=) |
| SI (1) | SI3596222T1 (https=) |
| SM (1) | SMT202300201T1 (https=) |
| TW (2) | TW202525835A (https=) |
| WO (1) | WO2018170408A1 (https=) |
| ZA (1) | ZA201906251B (https=) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| EP3596112A2 (en) | 2017-03-17 | 2020-01-22 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| JP7676111B2 (ja) * | 2017-03-17 | 2025-05-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| US11926653B2 (en) | 2017-10-20 | 2024-03-12 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| MY208145A (en) | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| WO2020132647A1 (en) * | 2018-12-21 | 2020-06-25 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| JP2022523038A (ja) * | 2019-02-02 | 2022-04-21 | ユニバーシティ オブ ピッツバーグ －オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | 遺伝子置換及び抗炎症を介したデュシェンヌ型筋ジストロフィーに対する一段階遺伝子療法 |
| AU2020229340A1 (en) | 2019-02-26 | 2021-09-16 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy |
| AU2020284255A1 (en) * | 2019-05-30 | 2022-01-06 | Solid Biosciences Inc. | Recombinant herpesvirales vector |
| RS65421B1 (sr) | 2019-08-21 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije |
| WO2021042004A1 (en) * | 2019-08-29 | 2021-03-04 | Siemens Healthcare Diagnostics Inc. | Reagents and methods for detecting aav shedding |
| TWI908746B (zh) * | 2019-11-28 | 2025-12-21 | 美商銳進科斯生物股份有限公司 | 微小肌縮蛋白基因療法之構築體及其用途 |
| WO2021126880A1 (en) * | 2019-12-16 | 2021-06-24 | Research Institute At Nationwide Children's Hospital | Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc) |
| IL297753A (en) | 2020-04-29 | 2022-12-01 | Bristol Myers Squibb Co | Minimized dystrophins with spectrin fusion domains and uses thereof |
| KR20230042468A (ko) * | 2020-08-05 | 2023-03-28 | 스페이스크래프트 세븐, 엘엘씨 | Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법 |
| WO2022029543A1 (en) * | 2020-08-06 | 2022-02-10 | Intas Pharmaceuticals Ltd. | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
| JP7642809B2 (ja) * | 2020-11-12 | 2025-03-10 | プレシジョン バイオサイエンシズ，インク． | ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ |
| EP4086276A1 (en) | 2021-05-03 | 2022-11-09 | Université d'Aix-Marseille | Composition for treating dysferlinopathy |
| EP4108263A3 (en) * | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| US20250041450A1 (en) * | 2021-08-05 | 2025-02-06 | Insmed Incorporated | Adeno-associated virus particles and methods of use thereof |
| US20250019721A1 (en) * | 2021-08-11 | 2025-01-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| CN114316070B (zh) * | 2021-12-29 | 2022-11-15 | 上海勉亦生物科技有限公司 | 用于治疗肌营养不良症的转基因表达盒 |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| CN118460618A (zh) * | 2022-04-19 | 2024-08-09 | 康霖生物科技（杭州）有限公司 | 一种用于遗传性凝血因子缺乏病治疗的核酸构建体 |
| WO2023248251A1 (en) * | 2022-06-24 | 2023-12-28 | Indian Institute Of Technology Kanpur | An optimized aav vector for gene therapy of muscular dystrophy |
| CN115819546B (zh) * | 2022-08-19 | 2025-12-05 | 成都金唯科生物科技有限公司 | 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用 |
| WO2026080823A1 (en) | 2024-10-10 | 2026-04-16 | Research Institute At Nationwide Children's Hospital | Methods for diagnosing and treating muscular dystrophy diseases |
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| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
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| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| US20080044393A1 (en) * | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| ES2639852T3 (es) * | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| EP2421971B1 (en) * | 2009-04-24 | 2016-07-06 | BioMarin Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| EP2736539B1 (en) | 2011-07-25 | 2017-08-23 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
| WO2013102904A1 (en) | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
| MX367100B (es) | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| MX388221B (es) * | 2013-04-20 | 2025-03-19 | Res Institute At Nationwide Children´S Hospital | ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2. |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| PL3097197T3 (pl) | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
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| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| ES2977730T3 (es) * | 2016-04-15 | 2024-08-29 | Res Inst Nationwide Childrens Hospital | Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular |
| JP7676111B2 (ja) * | 2017-03-17 | 2025-05-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
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2018
- 2018-03-16 JP JP2019550767A patent/JP7676111B2/ja active Active
- 2018-03-16 PT PT187683958T patent/PT3596222T/pt unknown
- 2018-03-16 IL IL269391A patent/IL269391B2/en unknown
- 2018-03-16 MA MA52112A patent/MA52112B1/fr unknown
- 2018-03-16 ES ES18768395T patent/ES2948233T3/es active Active
- 2018-03-16 WO PCT/US2018/022881 patent/WO2018170408A1/en not_active Ceased
- 2018-03-16 US US16/494,614 patent/US20200199621A1/en not_active Abandoned
- 2018-03-16 IL IL311713A patent/IL311713B2/en unknown
- 2018-03-16 EP EP18768395.8A patent/EP3596222B1/en active Active
- 2018-03-16 HU HUE18768395A patent/HUE062476T2/hu unknown
- 2018-03-16 KR KR1020247022488A patent/KR102887103B1/ko active Active
- 2018-03-16 FI FIEP18768395.8T patent/FI3596222T3/fi active
- 2018-03-16 KR KR1020257037800A patent/KR20250163423A/ko active Pending
- 2018-03-16 RS RS20230434A patent/RS64299B1/sr unknown
- 2018-03-16 CN CN201880018893.9A patent/CN110997923B/zh active Active
- 2018-03-16 LT LTEPPCT/US2018/022881T patent/LT3596222T/lt unknown
- 2018-03-16 CA CA3056638A patent/CA3056638A1/en active Pending
- 2018-03-16 PL PL18768395.8T patent/PL3596222T3/pl unknown
- 2018-03-16 BR BR112019019248A patent/BR112019019248A2/pt unknown
- 2018-03-16 SG SG11201908575S patent/SG11201908575SA/en unknown
- 2018-03-16 CN CN202311798281.5A patent/CN118497275A/zh active Pending
- 2018-03-16 AU AU2018233732A patent/AU2018233732B2/en active Active
- 2018-03-16 EP EP23167357.5A patent/EP4245852A3/en active Pending
- 2018-03-16 EA EA201992201A patent/EA201992201A1/ru unknown
- 2018-03-16 MX MX2019011046A patent/MX2019011046A/es unknown
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