JP2019502378A5 - - Google Patents
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- JP2019502378A5 JP2019502378A5 JP2018531202A JP2018531202A JP2019502378A5 JP 2019502378 A5 JP2019502378 A5 JP 2019502378A5 JP 2018531202 A JP2018531202 A JP 2018531202A JP 2018531202 A JP2018531202 A JP 2018531202A JP 2019502378 A5 JP2019502378 A5 JP 2019502378A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- seq
- viral vector
- sequence
- aav
- 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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- 239000013603 viral vector Substances 0.000 claims 20
- 230000014509 gene expression Effects 0.000 claims 18
- 239000000203 mixture Substances 0.000 claims 16
- 239000013598 vector Substances 0.000 claims 14
- 102100022881 Rab proteins geranylgeranyltransferase component A 1 Human genes 0.000 claims 11
- 101710088575 Rab escort protein 1 Proteins 0.000 claims 10
- 101710108890 Rab proteins geranylgeranyltransferase component A 1 Proteins 0.000 claims 10
- 102100029142 Cyclic nucleotide-gated cation channel alpha-3 Human genes 0.000 claims 8
- 101000771071 Homo sapiens Cyclic nucleotide-gated cation channel alpha-3 Proteins 0.000 claims 8
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 8
- 150000007523 nucleic acids Chemical group 0.000 claims 8
- 241000700605 Viruses Species 0.000 claims 7
- 210000000234 capsid Anatomy 0.000 claims 6
- 108020004705 Codon Proteins 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 4
- 239000002299 complementary DNA Substances 0.000 claims 4
- 239000013612 plasmid Substances 0.000 claims 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 3
- 238000004519 manufacturing process Methods 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical group O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108091006146 Channels Proteins 0.000 claims 2
- 208000006992 Color Vision Defects Diseases 0.000 claims 2
- 241000282412 Homo Species 0.000 claims 2
- 101000620777 Homo sapiens Rab proteins geranylgeranyltransferase component A 1 Proteins 0.000 claims 2
- 102100038300 Metabotropic glutamate receptor 6 Human genes 0.000 claims 2
- 102000004330 Rhodopsin Human genes 0.000 claims 2
- 108090000820 Rhodopsin Proteins 0.000 claims 2
- 208000003571 choroideremia Diseases 0.000 claims 2
- 201000007254 color blindness Diseases 0.000 claims 2
- 125000004122 cyclic group Chemical group 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 230000001939 inductive effect Effects 0.000 claims 2
- 108010038450 metabotropic glutamate receptor 6 Proteins 0.000 claims 2
- 238000004806 packaging method and process Methods 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 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 1
- 239000013607 AAV vector Substances 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 102000000806 Basic-Leucine Zipper Transcription Factors Human genes 0.000 claims 1
- 108010001572 Basic-Leucine Zipper Transcription Factors Proteins 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 208000033810 Choroidal dystrophy Diseases 0.000 claims 1
- 108010003730 Cone Opsins Proteins 0.000 claims 1
- UPEZCKBFRMILAV-JNEQICEOSA-N Ecdysone Natural products O=C1[C@H]2[C@@](C)([C@@H]3C([C@@]4(O)[C@@](C)([C@H]([C@H]([C@@H](O)CCC(O)(C)C)C)CC4)CC3)=C1)C[C@H](O)[C@H](O)C2 UPEZCKBFRMILAV-JNEQICEOSA-N 0.000 claims 1
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 claims 1
- 108091004242 G-Protein-Coupled Receptor Kinase 1 Proteins 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims 1
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 102100029533 Photoreceptor-specific nuclear receptor Human genes 0.000 claims 1
- 101710164507 Photoreceptor-specific nuclear receptor Proteins 0.000 claims 1
- 108090000799 Rhodopsin kinases Proteins 0.000 claims 1
- 239000004098 Tetracycline Substances 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 102000040945 Transcription factor Human genes 0.000 claims 1
- UPEZCKBFRMILAV-UHFFFAOYSA-N alpha-Ecdysone Natural products C1C(O)C(O)CC2(C)C(CCC3(C(C(C(O)CCC(C)(C)O)C)CCC33O)C)C3=CC(=O)C21 UPEZCKBFRMILAV-UHFFFAOYSA-N 0.000 claims 1
- 210000003161 choroid Anatomy 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- UPEZCKBFRMILAV-JMZLNJERSA-N ecdysone Chemical compound C1[C@@H](O)[C@@H](O)C[C@]2(C)[C@@H](CC[C@@]3([C@@H]([C@@H]([C@H](O)CCC(C)(C)O)C)CC[C@]33O)C)C3=CC(=O)[C@@H]21 UPEZCKBFRMILAV-JMZLNJERSA-N 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 229940011871 estrogen Drugs 0.000 claims 1
- 239000000262 estrogen Substances 0.000 claims 1
- 239000000833 heterodimer Substances 0.000 claims 1
- 102000049762 human CHM Human genes 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 230000002458 infectious effect Effects 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 claims 1
- 229960002930 sirolimus Drugs 0.000 claims 1
- 229930101283 tetracycline Natural products 0.000 claims 1
- 229960002180 tetracycline Drugs 0.000 claims 1
- 235000019364 tetracycline Nutrition 0.000 claims 1
- 150000003522 tetracyclines Chemical class 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022063749A JP2022088656A (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266789P | 2015-12-14 | 2015-12-14 | |
| US62/266,789 | 2015-12-14 | ||
| PCT/US2016/066402 WO2017106202A2 (en) | 2015-12-14 | 2016-12-13 | Gene therapy for ocular disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022063749A Division JP2022088656A (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019502378A JP2019502378A (ja) | 2019-01-31 |
| JP2019502378A5 true JP2019502378A5 (enExample) | 2020-01-30 |
| JP7057281B2 JP7057281B2 (ja) | 2022-04-19 |
Family
ID=58455636
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531202A Active JP7057281B2 (ja) | 2015-12-14 | 2016-12-13 | 眼疾患のための遺伝子療法 |
| JP2022063749A Pending JP2022088656A (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022063749A Pending JP2022088656A (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11090392B2 (enExample) |
| EP (2) | EP3389724B1 (enExample) |
| JP (2) | JP7057281B2 (enExample) |
| KR (1) | KR20180099719A (enExample) |
| CN (1) | CN109069668B (enExample) |
| AU (1) | AU2016370487C1 (enExample) |
| BR (1) | BR112018011838A2 (enExample) |
| CA (1) | CA3008264A1 (enExample) |
| ES (1) | ES2826384T3 (enExample) |
| MX (2) | MX2018007230A (enExample) |
| RU (1) | RU2762747C2 (enExample) |
| WO (1) | WO2017106202A2 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3389724B1 (en) * | 2015-12-14 | 2020-07-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| EP3377637B1 (en) * | 2016-04-08 | 2020-03-18 | Krystal Biotech, LLC | Compositions for use in methods for the treatment of wounds, disorders, and diseases of the skin |
| SG10202100632WA (en) * | 2016-07-26 | 2021-03-30 | Univ Cornell | Gene therapy for the treatment of aldehyde dehydrogenase deficiency |
| EP3638316A4 (en) * | 2017-06-14 | 2021-03-24 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR EYE DISEASES |
| CN112041434A (zh) | 2018-04-27 | 2020-12-04 | 克里斯托生物技术股份有限公司 | 用于美容性应用的编码一种或多种美容蛋白的重组核酸 |
| US20220143217A1 (en) * | 2019-03-04 | 2022-05-12 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the akt pathway |
| CN110889859A (zh) * | 2019-11-11 | 2020-03-17 | 珠海上工医信科技有限公司 | 一种用于眼底图像血管分割的u型网络 |
| IL297635A (en) * | 2020-04-27 | 2022-12-01 | Univ Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| CN116806158A (zh) * | 2020-09-02 | 2023-09-26 | 4D分子治疗有限公司 | 密码子优化的rep1基因及其用途 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| KR20230160829A (ko) * | 2021-02-26 | 2023-11-24 | 로직바이오 테라퓨틱스, 인크. | 재조합 aav 벡터의 제조 및 사용 |
| JP2024513826A (ja) | 2021-04-02 | 2024-03-27 | クリスタル バイオテック インコーポレイテッド | がん治療のためのウイルスベクター |
| US20240335562A1 (en) * | 2021-08-04 | 2024-10-10 | Alpine Biotherapeutics Corporation | Sustainable ocular cell-mediated intraocular delivery of cellular therapeutics for treatment of ocular diseases or disorders |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| EP1409677A2 (en) | 2001-01-19 | 2004-04-21 | Incyte Genomics, Inc. | Receptors and membrane-associated proteins |
| EP1381276A4 (en) | 2001-04-13 | 2005-02-02 | Univ Pennsylvania | METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| WO2010005533A2 (en) | 2008-06-30 | 2010-01-14 | The Johns Hopkins University | Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa |
| WO2010099383A2 (en) | 2009-02-26 | 2010-09-02 | The Johns Hopkins University | Compositions and methods for ex vivo hepatic nucleic acid delivery |
| RU2444977C2 (ru) | 2009-08-03 | 2012-03-20 | Федеральное государственное учреждение "МОСКОВСКИЙ НАУЧНО-ИССЛЕДОВАТЕЛЬСКИЙ ИНСТИТУТ ГЛАЗНЫХ БОЛЕЗНЕЙ ИМЕНИ ГЕЛЬМГОЛЬЦА ФЕДЕРАЛЬНОГО АГЕНТСТВА ПО ВЫСОКОТЕХНОЛОГИЧНОЙ МЕДИЦИНСКОЙ ПОМОЩИ" | Способ диагностики ахроматопсии |
| US20120172419A1 (en) * | 2009-09-15 | 2012-07-05 | Medical College Of Wisconsin Research Foundation Inc. | Reagents and methods for modulating cone photoreceptor activity |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| SG10201502270TA (en) | 2010-03-29 | 2015-05-28 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2661494B1 (en) * | 2011-01-07 | 2019-06-12 | Applied Genetic Technologies Corporation | Promoters, expression cassettes and vectors for use treating achromatopsia and other diseases |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| WO2012158757A1 (en) | 2011-05-16 | 2012-11-22 | The Trustees Of The University Of Pennsylvania | Proviral plasmids for production of recombinant adeno-associated virus |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US9375491B2 (en) | 2011-10-28 | 2016-06-28 | University Of Florida Research Foundation, Inc. | Chimeric promoter for cone photoreceptor targeted gene therapy |
| WO2013086515A1 (en) | 2011-12-09 | 2013-06-13 | The Johns Hopkins University | Compositions and methods for the prevention or treatment of diabetic complications |
| DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
| CA2878171C (en) | 2012-07-11 | 2021-04-27 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| EP2883981A4 (en) | 2012-08-08 | 2016-05-11 | Nihon Parkerizing | METAL SURFACE TREATMENT LIQUID, SURFACE TREATMENT METHOD FOR A METAL BASE, AND METAL BASE OBTAINED BY THE SURFACE TREATMENT PROCESS FOR METAL BASIS |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US10494645B2 (en) | 2013-04-18 | 2019-12-03 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| EP3517135B1 (en) | 2013-05-16 | 2025-11-19 | Beacon Therapeutics (USA), Inc. | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| KR102288849B1 (ko) | 2014-03-17 | 2021-08-12 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| JP6635942B2 (ja) | 2014-04-15 | 2020-01-29 | アプライド ジェネティック テクノロジーズ コーポレイション | 網膜色素変性症ｇｔｐアーゼレギュレーター（ｒｐｇｒ）をコードするコドン最適化核酸 |
| US20160206704A1 (en) | 2015-01-16 | 2016-07-21 | Isis Innovation Limited | Method |
| EP3389724B1 (en) * | 2015-12-14 | 2020-07-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
-
2016
- 2016-12-13 EP EP16852859.4A patent/EP3389724B1/en active Active
- 2016-12-13 ES ES16852859T patent/ES2826384T3/es active Active
- 2016-12-13 AU AU2016370487A patent/AU2016370487C1/en active Active
- 2016-12-13 WO PCT/US2016/066402 patent/WO2017106202A2/en not_active Ceased
- 2016-12-13 RU RU2018125468A patent/RU2762747C2/ru active
- 2016-12-13 CN CN201680081801.2A patent/CN109069668B/zh active Active
- 2016-12-13 CA CA3008264A patent/CA3008264A1/en active Pending
- 2016-12-13 JP JP2018531202A patent/JP7057281B2/ja active Active
- 2016-12-13 KR KR1020187019811A patent/KR20180099719A/ko not_active Ceased
- 2016-12-13 BR BR112018011838-9A patent/BR112018011838A2/pt active Search and Examination
- 2016-12-13 US US16/061,530 patent/US11090392B2/en active Active
- 2016-12-13 MX MX2018007230A patent/MX2018007230A/es unknown
- 2016-12-13 EP EP20183330.8A patent/EP3795180B1/en active Active
-
2018
- 2018-06-13 MX MX2022010959A patent/MX2022010959A/es unknown
-
2021
- 2021-07-07 US US17/369,525 patent/US12403204B2/en active Active
-
2022
- 2022-04-07 JP JP2022063749A patent/JP2022088656A/ja active Pending
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