JP2012519008A5 - - Google Patents
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- JP2012519008A5 JP2012519008A5 JP2011552364A JP2011552364A JP2012519008A5 JP 2012519008 A5 JP2012519008 A5 JP 2012519008A5 JP 2011552364 A JP2011552364 A JP 2011552364A JP 2011552364 A JP2011552364 A JP 2011552364A JP 2012519008 A5 JP2012519008 A5 JP 2012519008A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleic acid
- parvovirus
- polypeptide
- cds
- 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
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- 101000805768 Banna virus (strain Indonesia/JKT-6423/1980) mRNA (guanine-N(7))-methyltransferase Proteins 0.000 claims description 114
- 101000686790 Chaetoceros protobacilladnavirus 2 Replication-associated protein Proteins 0.000 claims description 114
- 101000864475 Chlamydia phage 1 Internal scaffolding protein VP3 Proteins 0.000 claims description 114
- 101000803553 Eumenes pomiformis Venom peptide 3 Proteins 0.000 claims description 114
- 101000583961 Halorubrum pleomorphic virus 1 Matrix protein Proteins 0.000 claims description 114
- 150000007523 nucleic acids Chemical class 0.000 claims description 66
- 239000002245 particle Substances 0.000 claims description 65
- 241000125945 Protoparvovirus Species 0.000 claims description 59
- 238000000034 method Methods 0.000 claims description 53
- 108020004707 nucleic acids Proteins 0.000 claims description 51
- 102000039446 nucleic acids Human genes 0.000 claims description 51
- 230000035772 mutation Effects 0.000 claims description 42
- 239000002773 nucleotide Substances 0.000 claims description 33
- 125000003729 nucleotide group Chemical group 0.000 claims description 33
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 28
- 229920001184 polypeptide Polymers 0.000 claims description 27
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 27
- 108090000623 proteins and genes Proteins 0.000 claims description 21
- 102000004169 proteins and genes Human genes 0.000 claims description 21
- 108091081024 Start codon Proteins 0.000 claims description 19
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 14
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 14
- 108700026244 Open Reading Frames Proteins 0.000 claims description 12
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 11
- 101710081079 Minor spike protein H Proteins 0.000 claims description 11
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 claims description 11
- 101710132601 Capsid protein Proteins 0.000 claims description 10
- 101710197658 Capsid protein VP1 Proteins 0.000 claims description 10
- 101710172711 Structural protein Proteins 0.000 claims description 10
- 101710108545 Viral protein 1 Proteins 0.000 claims description 10
- 239000003795 chemical substances by application Substances 0.000 claims description 10
- 230000037431 insertion Effects 0.000 claims description 10
- 238000003780 insertion Methods 0.000 claims description 10
- 241000700605 Viruses Species 0.000 claims description 8
- 108091026890 Coding region Proteins 0.000 claims description 7
- 230000000295 complement effect Effects 0.000 claims description 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 description 6
- 208000026935 allergic disease Diseases 0.000 claims description 6
- 210000000234 capsid Anatomy 0.000 claims description 6
- 239000002299 complementary DNA Substances 0.000 claims description 6
- 230000014621 translational initiation Effects 0.000 claims description 6
- 229960005486 vaccine Drugs 0.000 claims description 6
- 208000023275 Autoimmune disease Diseases 0.000 claims description 5
- 206010028980 Neoplasm Diseases 0.000 claims description 5
- 208000012902 Nervous system disease Diseases 0.000 claims description 5
- 208000025966 Neurological disease Diseases 0.000 claims description 5
- 238000004519 manufacturing process Methods 0.000 claims description 5
- 208000030159 metabolic disease Diseases 0.000 claims description 5
- 238000013518 transcription Methods 0.000 claims description 5
- 230000035897 transcription Effects 0.000 claims description 5
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 4
- 208000027866 inflammatory disease Diseases 0.000 claims description 4
- 230000002265 prevention Effects 0.000 claims description 4
- 238000011144 upstream manufacturing Methods 0.000 claims description 4
- 208000035473 Communicable disease Diseases 0.000 claims description 3
- 108020004414 DNA Proteins 0.000 claims description 3
- 241000702421 Dependoparvovirus Species 0.000 claims description 3
- 108091034057 RNA (poly(A)) Proteins 0.000 claims description 3
- 230000037430 deletion Effects 0.000 claims description 3
- 238000012217 deletion Methods 0.000 claims description 3
- 239000003446 ligand Substances 0.000 claims description 3
- 239000008177 pharmaceutical agent Substances 0.000 claims description 3
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 3
- 230000037432 silent mutation Effects 0.000 claims description 3
- 238000006467 substitution reaction Methods 0.000 claims description 3
- 238000013519 translation Methods 0.000 claims description 3
- 206010012335 Dependence Diseases 0.000 claims description 2
- 241000684283 Duck parvovirus Species 0.000 claims description 2
- 241001517118 Goose parvovirus Species 0.000 claims description 2
- 241000425549 Snake parvovirus Species 0.000 claims description 2
- 230000001684 chronic effect Effects 0.000 claims description 2
- 108020001507 fusion proteins Proteins 0.000 claims description 2
- 102000037865 fusion proteins Human genes 0.000 claims description 2
- 101710136297 Protein VP2 Proteins 0.000 claims 1
- 239000012634 fragment Substances 0.000 description 19
- 210000004027 cell Anatomy 0.000 description 15
- 101100524317 Adeno-associated virus 2 (isolate Srivastava/1982) Rep40 gene Proteins 0.000 description 8
- 101100524319 Adeno-associated virus 2 (isolate Srivastava/1982) Rep52 gene Proteins 0.000 description 8
- 101100524321 Adeno-associated virus 2 (isolate Srivastava/1982) Rep68 gene Proteins 0.000 description 8
- 101100524324 Adeno-associated virus 2 (isolate Srivastava/1982) Rep78 gene Proteins 0.000 description 8
- 210000003719 b-lymphocyte Anatomy 0.000 description 6
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 3
- 238000001415 gene therapy Methods 0.000 description 3
- 230000014616 translation Effects 0.000 description 3
- 208000024827 Alzheimer disease Diseases 0.000 description 2
- 201000001320 Atherosclerosis Diseases 0.000 description 2
- 241000283690 Bos taurus Species 0.000 description 2
- 108020004705 Codon Proteins 0.000 description 2
- 208000011231 Crohn disease Diseases 0.000 description 2
- 108020005202 Viral DNA Proteins 0.000 description 2
- 239000002671 adjuvant Substances 0.000 description 2
- 208000006673 asthma Diseases 0.000 description 2
- 208000037976 chronic inflammation Diseases 0.000 description 2
- 208000037893 chronic inflammatory disorder Diseases 0.000 description 2
- 201000010099 disease Diseases 0.000 description 2
- 208000016097 disease of metabolism Diseases 0.000 description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 2
- 208000015181 infectious disease Diseases 0.000 description 2
- 206010039073 rheumatoid arthritis Diseases 0.000 description 2
- 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
- 241000271566 Aves Species 0.000 description 1
- 241000282465 Canis Species 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 102000053602 DNA Human genes 0.000 description 1
- 206010020751 Hypersensitivity Diseases 0.000 description 1
- 241000701945 Parvoviridae Species 0.000 description 1
- 201000004681 Psoriasis Diseases 0.000 description 1
- 206010039085 Rhinitis allergic Diseases 0.000 description 1
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 1
- -1 VP3 Nucleic acid Chemical class 0.000 description 1
- 108091027569 Z-DNA Proteins 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 206010064930 age-related macular degeneration Diseases 0.000 description 1
- 201000010105 allergic rhinitis Diseases 0.000 description 1
- 230000007815 allergy Effects 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 230000001580 bacterial effect Effects 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 230000006058 immune tolerance Effects 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 208000002780 macular degeneration Diseases 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 241000894007 species Species 0.000 description 1
- 241000701447 unidentified baculovirus Species 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US15743609P | 2009-03-04 | 2009-03-04 | |
| US61/157,436 | 2009-03-04 | ||
| US30620510P | 2010-02-19 | 2010-02-19 | |
| US61/306,205 | 2010-02-19 | ||
| PCT/EP2010/001343 WO2010099960A2 (en) | 2009-03-04 | 2010-03-04 | Assembly activating protein (aap) and its use for the manufacture of parvovirus particles essential consisting of vp3 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015200962A Division JP6151328B2 (ja) | 2009-03-04 | 2015-10-09 | 集合活性化タンパク質(aap)およびvp3から本質的になるパルボウイルス粒子を製造するためのaapの使用 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2012519008A JP2012519008A (ja) | 2012-08-23 |
| JP2012519008A5 true JP2012519008A5 (enExample) | 2013-04-11 |
Family
ID=42313642
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011552364A Pending JP2012519008A (ja) | 2009-03-04 | 2010-03-04 | 集合活性化タンパク質(aap)およびvp3から本質的になるパルボウイルス粒子を製造するためのaapの使用 |
| JP2015200962A Expired - Fee Related JP6151328B2 (ja) | 2009-03-04 | 2015-10-09 | 集合活性化タンパク質(aap)およびvp3から本質的になるパルボウイルス粒子を製造するためのaapの使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015200962A Expired - Fee Related JP6151328B2 (ja) | 2009-03-04 | 2015-10-09 | 集合活性化タンパク質(aap)およびvp3から本質的になるパルボウイルス粒子を製造するためのaapの使用 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US9464119B2 (enExample) |
| EP (1) | EP2403867B1 (enExample) |
| JP (2) | JP2012519008A (enExample) |
| CN (1) | CN102341406B (enExample) |
| BR (1) | BRPI1013364A2 (enExample) |
| CA (1) | CA2754335C (enExample) |
| DK (1) | DK2403867T3 (enExample) |
| ES (1) | ES2742180T3 (enExample) |
| MX (2) | MX2011009193A (enExample) |
| PL (1) | PL2403867T3 (enExample) |
| PT (1) | PT2403867T (enExample) |
| WO (1) | WO2010099960A2 (enExample) |
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102341406B (zh) | 2009-03-04 | 2016-06-08 | 德国癌症研究中心 | 组装活化蛋白(aap)及其用于制备基本上由vp3组成的细小病毒颗粒的应用 |
| US20130052226A1 (en) * | 2010-02-12 | 2013-02-28 | The Government Of The Usa, As Represented By The Secretary, Department Of Health & Human Services | Compositions and methods for preventing or treating a human parvovirus infection |
| AR082925A1 (es) * | 2010-09-08 | 2013-01-16 | Medigene Ag | Proteinas estructurales mutadas por parvovirus con epitopo de celulas b de proteccion cruzada, producto y metodos relacionados |
| RU2487708C1 (ru) * | 2012-03-12 | 2013-07-20 | Государственное бюджетное образовательное учреждение высшего профессионального образования "Уральская государственная медицинская академия Министерства здравоохранения и социального развития Российской Федерации" (ГБОУ ВПО УГМА Минздравсоцразвития России) | Способ лечения парвовирусной инфекции в19 у детей раннего возраста |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| KR102288849B1 (ko) | 2014-03-17 | 2021-08-12 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016073693A2 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| CA2975583A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| JP7095994B2 (ja) | 2015-03-02 | 2022-07-05 | アドヴェラム バイオテクノロジーズ, インコーポレイテッド | ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| JP7166168B2 (ja) | 2015-07-30 | 2022-11-07 | マサチューセッツ アイ アンド イヤー インファーマリー | 祖先ウイルス配列およびその使用 |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| 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 |
| IL262784B2 (en) | 2016-05-18 | 2023-10-01 | Voyager Therapeutics Inc | modulatory polynucleotides |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| 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 |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| WO2018204786A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| EP3645021A4 (en) * | 2017-06-30 | 2021-04-21 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| AU2018311069A1 (en) | 2017-08-03 | 2020-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
| EP3688014A4 (en) * | 2017-09-29 | 2020-09-16 | Massachusetts Eye and Ear Infirmary | PRODUCTION OF ADENO-ASSOCIATED VIRUSES IN INSECT CELLS |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US20190202866A1 (en) * | 2017-12-22 | 2019-07-04 | William Marsh Rice University | Viral nanoparticles and methods of use thereof |
| AU2019220386A1 (en) | 2018-02-16 | 2020-08-20 | 2A Pharma Ab | Parvovirus structural protein for the treatment of autoimmune diseases |
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| US20210147487A1 (en) * | 2018-04-04 | 2021-05-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for engineered assembly activating proteins (eaaps) |
| WO2019195701A1 (en) | 2018-04-05 | 2019-10-10 | Massachusetts Eye And Ear Infirmary | Methods of making and using combinatorial barcoded nucleic acid libraries having defined variation |
| WO2019210267A2 (en) * | 2018-04-27 | 2019-10-31 | Spark Therapeutics, Inc. | Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2019418750A1 (en) * | 2019-01-02 | 2021-07-22 | Intima Bioscience, Inc. | Modified adeno-associated viral vectors for use in genetic engineering |
| PT3850089T (pt) | 2019-02-04 | 2024-01-24 | Freeline Therapeutics Ltd | Polinucleótidos |
| JP2022520647A (ja) | 2019-02-15 | 2022-03-31 | サンガモ セラピューティクス, インコーポレイテッド | 組み換えaav生産のための組成物および方法 |
| JP7562558B2 (ja) * | 2019-04-12 | 2024-10-07 | アセンド アドヴァンスト セラピーズ リミテッド | プラスミドシステム |
| CA3170169A1 (en) * | 2020-02-07 | 2021-08-12 | Vecprobio, Inc. | Recombinant adeno-associated viral vectors in plants |
| EP4172322A1 (en) * | 2020-06-25 | 2023-05-03 | Ferring Ventures SA | Improved adeno-associated virus gene therapy vectors |
| WO2023083964A1 (en) | 2021-11-11 | 2023-05-19 | 2A Pharma Ab | Parvovirus structural protein against beta- and gamma-hpv |
| EP4603505A1 (en) * | 2022-10-13 | 2025-08-20 | Kanglin Biotech (Hangzhou) Co., Ltd. | Method for modifying capsid protein coding gene of adeno-associated virus |
| CN117126270B (zh) * | 2023-10-25 | 2024-02-13 | 首都儿科研究所 | 一种2型人博卡病毒型别特异性抗体及其应用 |
| WO2025125222A1 (en) | 2023-12-11 | 2025-06-19 | Leukocare Ag | Compositions comprising viruses, viral vectors or virus-like particles |
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| US4168062A (en) | 1977-12-05 | 1979-09-18 | Mccarthy Gerald F | Automated goalie |
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| DE19905501B4 (de) | 1999-02-10 | 2005-05-19 | MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie | Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels |
| US20020052040A1 (en) * | 1999-06-30 | 2002-05-02 | Nicholas Hunt | Virus like particles, their preparation and their use preferably in pharmaceutical screening and functional genomics |
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| DE19933719A1 (de) | 1999-07-19 | 2001-01-25 | Medigene Ag | Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung |
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| WO2008145400A2 (en) | 2007-05-31 | 2008-12-04 | Medigene Ag | Mutated structural protein of a parvovirus |
| CN102341406B (zh) | 2009-03-04 | 2016-06-08 | 德国癌症研究中心 | 组装活化蛋白(aap)及其用于制备基本上由vp3组成的细小病毒颗粒的应用 |
-
2010
- 2010-03-04 CN CN201080010540.8A patent/CN102341406B/zh active Active
- 2010-03-04 DK DK10708716.5T patent/DK2403867T3/da active
- 2010-03-04 EP EP10708716.5A patent/EP2403867B1/en active Active
- 2010-03-04 CA CA2754335A patent/CA2754335C/en active Active
- 2010-03-04 WO PCT/EP2010/001343 patent/WO2010099960A2/en not_active Ceased
- 2010-03-04 US US13/203,442 patent/US9464119B2/en active Active
- 2010-03-04 BR BRPI1013364A patent/BRPI1013364A2/pt not_active Application Discontinuation
- 2010-03-04 JP JP2011552364A patent/JP2012519008A/ja active Pending
- 2010-03-04 PL PL10708716T patent/PL2403867T3/pl unknown
- 2010-03-04 PT PT10708716T patent/PT2403867T/pt unknown
- 2010-03-04 MX MX2011009193A patent/MX2011009193A/es active IP Right Grant
- 2010-03-04 MX MX2014014365A patent/MX345729B/es unknown
- 2010-03-04 ES ES10708716T patent/ES2742180T3/es active Active
-
2015
- 2015-10-09 JP JP2015200962A patent/JP6151328B2/ja not_active Expired - Fee Related
-
2016
- 2016-09-12 US US15/263,072 patent/US10344057B2/en active Active
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2019
- 2019-05-17 US US16/415,676 patent/US11267847B2/en active Active
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