DK2678435T3 - Aav-vektorer til anvendelse til genterapi af choroideremi - Google Patents
Aav-vektorer til anvendelse til genterapi af choroideremi Download PDFInfo
- Publication number
- DK2678435T3 DK2678435T3 DK12706308.9T DK12706308T DK2678435T3 DK 2678435 T3 DK2678435 T3 DK 2678435T3 DK 12706308 T DK12706308 T DK 12706308T DK 2678435 T3 DK2678435 T3 DK 2678435T3
- Authority
- DK
- Denmark
- Prior art keywords
- aav
- vector
- rep1
- sequence
- seq
- Prior art date
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Claims (10)
1. Adenoassocieret virus-serotype 2 (AAV2)-vektorpartikel til anvendelse til behandling eller forebyggelse af choroideremi, hvor genomet i partiklen er et AAV2-genom, der omfatter en polynukleotidsekvens, der koder for REP1 som vist i SEQ ID NO: 3 eller en variant deraf, der har mindst 97 % identitet over hele længden af SEQ ID NO: 3, hvor polynukleotidsekvensen, der koder for REP1, er operabelt koblet til en promotorsekvens.
2. AAV2-vektorpartikel til anvendelse ifølge krav 1, hvor genomet er som vist i SEQ ID NO: 1 og/eller mangler én eller flere fra gruppen, der består af én inverteret terminal gentagelses (ITR)-sekvens, replikations (rep)-gensekvensen og capsid (cap)-gensekvensen.
3. AAV2-vektorpartikel til anvendelse ifølge krav 1 eller 2, hvor REP1 har sekvensen ifølge SEQ ID NO: 3.
4. AAV2-vektorpartikel til anvendelse ifølge krav 1 eller 2, hvor polynukleotidsekvensen, der koder for REP1 som vist i SEQ ID NO: 3, har mindst 90 % identitet med SEQ ID NO: 2 over hele dens sekvens.
5. AAV2-vektorpartikel til anvendelse ifølge et hvilket som helst af kravene 1 til 4, hvor promotoren er konstitutivt aktiv.
6. AAV2-vektorpartikel til anvendelse ifølge et hvilket som helst af kravene 1 til 4, hvor ekspression fra promotoren er retinaceliespecifik.
7. AAV2-vektorpartikel til anvendelse ifølge et hvilket som helst af kravene 1 til 6, der omfatter en eller flere yderligere regulatoriske sekvenser.
8. AAV2-vektorpartikel til anvendelse ifølge krav 7, hvor genomet omfatter en sekvens, der har mindst 70 % identitet med SEQ ID NO: 5 over hele dets sekvens.
9. AAV-vektorpartikel til anvendelse ifølge et hvilket som helst af ovennævnte krav, hvor vektorpartiklen administreres ved hjælp af direkte retinal, subretinal eller intravitreal inj ektion.
10. AAV2-vektorpartikel til anvendelse ifølge et hvilket som helst af ovennævnte krav, hvor vektorpartiklen administreres direkte i det subretinale rum.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1103062.4A GB201103062D0 (en) | 2011-02-22 | 2011-02-22 | Method |
| PCT/GB2012/050376 WO2012114090A1 (en) | 2011-02-22 | 2012-02-21 | Aav -vectors for use in gene therapy of choroideremia |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2678435T3 true DK2678435T3 (da) | 2018-08-27 |
Family
ID=43881504
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12706308.9T DK2678435T3 (da) | 2011-02-22 | 2012-02-21 | Aav-vektorer til anvendelse til genterapi af choroideremi |
Country Status (18)
| Country | Link |
|---|---|
| US (4) | US9834788B2 (da) |
| EP (2) | EP2678435B2 (da) |
| JP (4) | JP2014512171A (da) |
| KR (3) | KR102299430B1 (da) |
| CN (2) | CN107881198A (da) |
| AU (2) | AU2012220404B2 (da) |
| BR (1) | BR112013021318B1 (da) |
| CA (1) | CA2827975C (da) |
| DK (1) | DK2678435T3 (da) |
| ES (1) | ES2676550T3 (da) |
| GB (1) | GB201103062D0 (da) |
| HK (2) | HK1258917A1 (da) |
| IL (1) | IL228031B (da) |
| MX (1) | MX356525B (da) |
| PL (1) | PL2678435T3 (da) |
| PT (1) | PT2678435T (da) |
| SG (1) | SG192911A1 (da) |
| WO (1) | WO2012114090A1 (da) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| WO2015020522A1 (en) | 2013-08-05 | 2015-02-12 | Koninklijke Nederlandse Akademie Van Wetenschappen | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
| TN2016000220A1 (en) | 2013-12-06 | 2017-10-06 | Ct Hospitalier Universitaire Montpellier | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject. |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CA2966620A1 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| US20180043034A1 (en) * | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| CN109069668B (zh) * | 2015-12-14 | 2023-04-18 | 宾夕法尼亚州大学信托人 | 用于眼病的基因疗法 |
| US20190017096A1 (en) | 2016-03-10 | 2019-01-17 | Nightstarx Limited | Prenylation assay |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| KR102392236B1 (ko) | 2016-05-18 | 2022-05-03 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| WO2018085844A1 (en) * | 2016-11-07 | 2018-05-11 | Spark Therapeutics, Inc. | Rab escort protein potency assay |
| AU2018207259B2 (en) * | 2017-01-10 | 2023-11-09 | Children's Medical Research Institute | Polynucleotides and vectors for the expression of transgenes |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| CN110913866A (zh) | 2017-05-05 | 2020-03-24 | 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 |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| US11827898B2 (en) | 2017-06-14 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | NETWORK EQUIPMENT TRACK GUIDE SYSTEM |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖを送達するための組成物および方法 |
| AU2018346102B2 (en) * | 2017-10-03 | 2023-05-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| JP7502991B2 (ja) | 2017-10-16 | 2024-06-19 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症（ａｌｓ）の治療 |
| SG11202003462SA (en) | 2017-10-17 | 2020-05-28 | Nightstarx Ltd | Prenylation assay |
| MX2020005952A (es) * | 2017-12-06 | 2022-01-03 | Ovid Therapeutics Inc | Uso de mir101 o mir128 en el tratamiento de trastornos convulsivos. |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| EP3755795A4 (en) | 2018-02-19 | 2022-07-20 | Homology Medicines, Inc. | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF |
| MX2020010191A (es) | 2018-04-05 | 2021-02-26 | Nightstarx Ltd | Composiciones de virus adenoasociado (aav), métodos de fabricación y métodos de uso. |
| JP2021522775A (ja) * | 2018-05-07 | 2021-09-02 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルＴｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ | 合理的なポリプロイド性アデノ随伴ウイルスベクターならびにその製造法および使用法 |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| MX2023001998A (es) | 2020-08-17 | 2023-05-04 | Massachusetts Inst Technology | Enfoques de terapia con el gen shank3. |
| WO2022109247A1 (en) | 2020-11-20 | 2022-05-27 | Biogen Ma Inc. | Viral vector potency assay |
| WO2023164545A1 (en) | 2022-02-23 | 2023-08-31 | Massachusetts Institute Of Technology | Methods for upregulating shank3 expression |
| WO2024033837A1 (en) | 2022-08-11 | 2024-02-15 | Institute Of Molecular And Clinical Ophthalmology Basel (Iob) | Human cone photoreceptor optogenetic constructs |
| WO2024033834A1 (en) | 2022-08-11 | 2024-02-15 | Institute Of Molecular And Clinical Opthalmology Basel (Iob) | Promoters for specific expression of genes in cone photoreceptors |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0856585A1 (en) | 1997-01-29 | 1998-08-05 | Introgene B.V. | A conditional replication and expression system |
| US20020045264A1 (en) * | 2000-03-14 | 2002-04-18 | During Matthew J. | Production of chimeric capsid vectors |
| WO2002082904A2 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
| US8278284B2 (en) * | 2005-02-23 | 2012-10-02 | Masanori Miyazaki | Therapeutic agents for diseases associated with apoptotic degeneration in ocular tissue cells that use SIV-PEDF vectors |
| CN101213203A (zh) * | 2005-04-29 | 2008-07-02 | 教堂山北卡罗莱纳州大学 | 在转录后水平调节核酸表达的方法和组合物 |
| US20090022731A1 (en) * | 2006-08-25 | 2009-01-22 | Wyeth | Arthritis-associated B cell gene expression |
| US20100278843A1 (en) * | 2007-08-16 | 2010-11-04 | St. Vincent's Hospital Sydney Limited | Agents and methods for modulating macrophage inhibitory cytokine (mic-1) activity |
| CA2713338C (en) * | 2008-01-29 | 2021-10-26 | Applied Genetic Technologies Corporation | Recombinant virus production using mammalian cells in suspension |
| US8242093B2 (en) * | 2008-02-07 | 2012-08-14 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein |
| HUE057606T2 (hu) * | 2009-05-02 | 2022-05-28 | Genzyme Corp | Génterápia neurodegeneratív rendellenességekre |
| WO2011088081A1 (en) | 2010-01-12 | 2011-07-21 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
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2011
- 2011-02-22 GB GBGB1103062.4A patent/GB201103062D0/en not_active Ceased
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2012
- 2012-02-21 WO PCT/GB2012/050376 patent/WO2012114090A1/en active Application Filing
- 2012-02-21 KR KR1020207021515A patent/KR102299430B1/ko active IP Right Grant
- 2012-02-21 US US14/000,836 patent/US9834788B2/en active Active
- 2012-02-21 PL PL12706308T patent/PL2678435T3/pl unknown
- 2012-02-21 PT PT127063089T patent/PT2678435T/pt unknown
- 2012-02-21 EP EP12706308.9A patent/EP2678435B2/en active Active
- 2012-02-21 MX MX2013009604A patent/MX356525B/es active IP Right Grant
- 2012-02-21 JP JP2013554938A patent/JP2014512171A/ja active Pending
- 2012-02-21 ES ES12706308.9T patent/ES2676550T3/es active Active
- 2012-02-21 DK DK12706308.9T patent/DK2678435T3/da active
- 2012-02-21 SG SG2013063797A patent/SG192911A1/en unknown
- 2012-02-21 CN CN201710784145.9A patent/CN107881198A/zh active Pending
- 2012-02-21 KR KR1020137024782A patent/KR102139203B1/ko active IP Right Grant
- 2012-02-21 CA CA2827975A patent/CA2827975C/en active Active
- 2012-02-21 CN CN201280019547.5A patent/CN103562396A/zh active Pending
- 2012-02-21 BR BR112013021318-3A patent/BR112013021318B1/pt active IP Right Grant
- 2012-02-21 EP EP18172129.1A patent/EP3428283A1/en active Pending
- 2012-02-21 KR KR1020217028082A patent/KR20210112407A/ko not_active Application Discontinuation
- 2012-02-21 AU AU2012220404A patent/AU2012220404B2/en active Active
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2013
- 2013-08-19 IL IL228031A patent/IL228031B/en active IP Right Grant
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2014
- 2014-05-23 HK HK19101394.9A patent/HK1258917A1/zh unknown
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2017
- 2017-06-14 JP JP2017117286A patent/JP2017221192A/ja active Pending
- 2017-07-20 AU AU2017206254A patent/AU2017206254A1/en not_active Abandoned
- 2017-10-31 US US15/799,852 patent/US20180135078A1/en not_active Abandoned
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2018
- 2018-07-13 HK HK18109118.8A patent/HK1249548A1/zh unknown
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2019
- 2019-11-05 JP JP2019201092A patent/JP2020054345A/ja active Pending
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2020
- 2020-01-24 US US16/751,699 patent/US20210032656A1/en not_active Abandoned
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2022
- 2022-10-26 US US17/974,466 patent/US20230332176A1/en active Pending
- 2022-12-02 JP JP2022193831A patent/JP2023036618A/ja active Pending
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