SI2984166T1 - Sestavki za zdravljenje MPSI - Google Patents
Sestavki za zdravljenje MPSIInfo
- Publication number
- SI2984166T1 SI2984166T1 SI201431605T SI201431605T SI2984166T1 SI 2984166 T1 SI2984166 T1 SI 2984166T1 SI 201431605 T SI201431605 T SI 201431605T SI 201431605 T SI201431605 T SI 201431605T SI 2984166 T1 SI2984166 T1 SI 2984166T1
- Authority
- SI
- Slovenia
- Prior art keywords
- compositions
- mpsi
- treating
- treating mpsi
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Dermatology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361788724P | 2013-03-15 | 2013-03-15 | |
| PCT/US2014/025509 WO2014151341A1 (en) | 2013-03-15 | 2014-03-13 | Compositions and methods for treating mpsi |
| EP14770186.6A EP2984166B1 (en) | 2013-03-15 | 2014-03-13 | Compositions for treating mpsi |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI2984166T1 true SI2984166T1 (sl) | 2020-09-30 |
Family
ID=51580934
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201431605T SI2984166T1 (sl) | 2013-03-15 | 2014-03-13 | Sestavki za zdravljenje MPSI |
Country Status (21)
| Country | Link |
|---|---|
| US (3) | US10137176B2 (enExample) |
| EP (2) | EP2984166B1 (enExample) |
| JP (4) | JP6591956B2 (enExample) |
| KR (2) | KR102346455B1 (enExample) |
| CN (1) | CN105026554B (enExample) |
| AU (2) | AU2014235096B2 (enExample) |
| BR (1) | BR112015021036A2 (enExample) |
| CA (1) | CA2901328C (enExample) |
| CY (1) | CY1123169T1 (enExample) |
| DK (1) | DK2984166T3 (enExample) |
| ES (2) | ES2805355T3 (enExample) |
| HR (1) | HRP20201063T1 (enExample) |
| HU (1) | HUE051373T2 (enExample) |
| LT (1) | LT2984166T (enExample) |
| MX (2) | MX386059B (enExample) |
| PL (1) | PL2984166T3 (enExample) |
| PT (1) | PT2984166T (enExample) |
| RU (1) | RU2708318C2 (enExample) |
| SG (2) | SG10201707319UA (enExample) |
| SI (1) | SI2984166T1 (enExample) |
| WO (1) | WO2014151341A1 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2984166T3 (pl) | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
| BR112016024379A2 (pt) * | 2014-04-25 | 2017-10-10 | Univ Pennsylvania | variantes ldlr e seu uso em composições para reduzir os níveis de colesterol |
| EP3400304B1 (en) * | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| WO2017106345A1 (en) * | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Composition for treatment of crigler-najjar syndrome |
| WO2017136533A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses |
| SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| CN108697774A (zh) * | 2016-02-22 | 2018-10-23 | 北卡罗来纳大学教堂山分校 | 治疗mps i-相关失明的aav-idua载体 |
| KR20190008237A (ko) | 2016-04-15 | 2019-01-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| JP7046828B2 (ja) | 2016-04-15 | 2022-04-04 | ザ・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 | 滲出型加齢性黄斑変性の治療のための組成物 |
| US11883470B2 (en) | 2016-07-25 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| WO2018035503A1 (en) | 2016-08-18 | 2018-02-22 | The Regents Of The University Of California | Crispr-cas genome engineering via a modular aav delivery system |
| WO2018126112A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating phenylketonuria |
| KR20200053468A (ko) | 2017-07-06 | 2020-05-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | I형 점액다당류증을 치료하기 위한 유전자 요법 |
| WO2019060662A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II |
| CN107699590A (zh) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | 一种制备重组人α‑L‑艾杜糖醛酸酶的方法 |
| JP7384797B2 (ja) | 2017-11-30 | 2023-11-21 | ザ・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 | ムコ多糖症ｉｉｉｂ型のための遺伝子療法 |
| PE20212076A1 (es) | 2018-02-01 | 2021-10-26 | Homology Medicines Inc | Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| WO2019204303A2 (en) | 2018-04-16 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating duchenne muscular dystrophy |
| MX2020011937A (es) | 2018-05-09 | 2021-01-29 | Biomarin Pharm Inc | Metodos de tratamiento de fenilcetonuria. |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| AU2019304569B2 (en) | 2018-07-17 | 2023-07-06 | Helixmith Co., Ltd | Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms |
| EP3917566A4 (en) | 2019-01-31 | 2022-10-26 | Oregon Health & Science University | METHODS OF USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS |
| 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 |
| BR112022022212A2 (pt) | 2020-05-12 | 2022-12-13 | Univ Pennsylvania | Composições para redução específica de drg de expressão de transgene |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| TW202229560A (zh) | 2020-10-09 | 2022-08-01 | 賓州大學委員會 | 治療法布瑞氏症之組成物及方法 |
| KR102590276B1 (ko) * | 2021-01-04 | 2023-10-16 | 부산대학교 산학협력단 | 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도 |
| US20240091380A1 (en) | 2021-02-01 | 2024-03-21 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4148078B1 (de) | 2021-09-08 | 2025-11-05 | Covestro Deutschland AG | Auf bernsteinsäure basierende polyesterpolyole |
| CN114181318B (zh) * | 2021-11-08 | 2023-08-01 | 四川大学 | 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20240408157A1 (en) | 2021-12-02 | 2024-12-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| EP4504797A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of The University of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
| EP4504258A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of the University of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
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| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase |
| CN1300323A (zh) | 1998-05-13 | 2001-06-20 | 加州大学洛杉矶分校港口研究和教育院 | 重组α－L－艾杜糖苷酸酶,其生产和纯化的方法以及治疗其缺乏导致的疾病的方法 |
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| CA2469785C (en) | 2001-12-17 | 2014-04-22 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
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| PL2984166T3 (pl) | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| WO2016055437A1 (en) | 2014-10-06 | 2016-04-14 | Arthrogen B.V. | Aav-based gene therapy |
| KR20250150167A (ko) | 2015-05-15 | 2025-10-17 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 치료적 전달 |
| CN107922932A (zh) | 2015-08-06 | 2018-04-17 | 中央研究院 | 用于酶置换疗法的改造酶 |
| CN116650668A (zh) | 2015-10-23 | 2023-08-29 | 衣阿华大学研究基金会 | 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| WO2017100704A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| KR20180127319A (ko) | 2016-01-15 | 2018-11-28 | 상가모 테라퓨틱스, 인코포레이티드 | 신경 질환의 치료를 위한 방법 및 조성물 |
| SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| KR20200053468A (ko) | 2017-07-06 | 2020-05-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | I형 점액다당류증을 치료하기 위한 유전자 요법 |
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2014
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- 2014-03-13 DK DK14770186.6T patent/DK2984166T3/da active
- 2014-03-13 MX MX2015012739A patent/MX386059B/es unknown
- 2014-03-13 CN CN201480010987.3A patent/CN105026554B/zh active Active
- 2014-03-13 US US14/769,596 patent/US10137176B2/en active Active
- 2014-03-13 HR HRP20201063TT patent/HRP20201063T1/hr unknown
- 2014-03-13 ES ES14770186T patent/ES2805355T3/es active Active
- 2014-03-13 EP EP14770186.6A patent/EP2984166B1/en active Active
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- 2014-03-13 CA CA2901328A patent/CA2901328C/en active Active
- 2014-03-13 RU RU2015144234A patent/RU2708318C2/ru active
- 2014-03-13 KR KR1020157022504A patent/KR102346455B1/ko active Active
- 2014-03-13 HU HUE14770186A patent/HUE051373T2/hu unknown
- 2014-03-13 WO PCT/US2014/025509 patent/WO2014151341A1/en not_active Ceased
- 2014-03-13 AU AU2014235096A patent/AU2014235096B2/en active Active
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- 2014-03-13 JP JP2016501864A patent/JP6591956B2/ja active Active
- 2014-03-13 KR KR1020217043023A patent/KR102581652B1/ko active Active
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2015
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2018
- 2018-10-22 US US16/166,346 patent/US10792343B2/en active Active
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2019
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- 2020-09-10 US US17/017,150 patent/US20200397872A1/en not_active Abandoned
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2022
- 2022-10-27 JP JP2022172314A patent/JP7553527B2/ja active Active
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