BR112015012152A2 - Proteína de fusão terapêutica direcionada que compreende uma enzima lisossomal, ácido nucleico, composição farmacêutica, método para produzir a dita proteína e usos da proteína para tratar uma doença de armazenamento lisossomal e mucopolissacaridose tipo iiib - Google Patents
Proteína de fusão terapêutica direcionada que compreende uma enzima lisossomal, ácido nucleico, composição farmacêutica, método para produzir a dita proteína e usos da proteína para tratar uma doença de armazenamento lisossomal e mucopolissacaridose tipo iiibInfo
- Publication number
- BR112015012152A2 BR112015012152A2 BR112015012152A BR112015012152A BR112015012152A2 BR 112015012152 A2 BR112015012152 A2 BR 112015012152A2 BR 112015012152 A BR112015012152 A BR 112015012152A BR 112015012152 A BR112015012152 A BR 112015012152A BR 112015012152 A2 BR112015012152 A2 BR 112015012152A2
- Authority
- BR
- Brazil
- Prior art keywords
- protein
- therapeutic fusion
- mucopolysacchaidosis
- lysosol
- nucleic acid
- Prior art date
Links
- 108020001507 fusion proteins Proteins 0.000 title abstract 5
- 230000001225 therapeutic effect Effects 0.000 title abstract 5
- 108090000623 proteins and genes Proteins 0.000 title abstract 4
- 102000004169 proteins and genes Human genes 0.000 title abstract 4
- 108090000790 Enzymes Proteins 0.000 title abstract 3
- 102000004190 Enzymes Human genes 0.000 title abstract 3
- 201000010099 disease Diseases 0.000 title abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 3
- 210000003712 lysosome Anatomy 0.000 title abstract 2
- 230000001868 lysosomic effect Effects 0.000 title abstract 2
- 238000004519 manufacturing process Methods 0.000 title abstract 2
- 108020004707 nucleic acids Proteins 0.000 title abstract 2
- 102000039446 nucleic acids Human genes 0.000 title abstract 2
- 150000007523 nucleic acids Chemical class 0.000 title abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 102000037865 fusion proteins Human genes 0.000 abstract 3
- 230000002132 lysosomal effect Effects 0.000 abstract 3
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 abstract 2
- 101800001707 Spacer peptide Proteins 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 230000008685 targeting Effects 0.000 abstract 2
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 abstract 1
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 208000005340 mucopolysaccharidosis III Diseases 0.000 abstract 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 abstract 1
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 abstract 1
- 208000011580 syndromic disease Diseases 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- 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)
- C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Biophysics (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Toxicology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Abstract
PROTEÍNA DE FUSÃO TERAPÊUTICA DIRECIONADA QUE COMPREENDE UMA ENZIMA LISOSSOMAL, ÁCIDO NUCLEICO, COMPOSIÇÃO FARMACÊUTICA, MÉTODO PARA PRODUZIR A DITA PROTEÍNA E USOS DA PROTEÍNA PARA TRATAR UMA DOENÇA DE ARMAZENAMENTO LISOSSOMAL E MUCOPOLISSACARIDOSE TIPO IIIB. A presente invenção se refere em geral a proteínas de fusão terapêuticas úteis para tratar doenças de armazenamento lisossômico e métodos para tratar essas doenças. Proteínas de fusão terapêuticas exemplares compreendem uma enzima lisossômica, umafração de alvo lisossômico, por exemplo, um peptídeo IGF-II e um peptídeo espaçador. Também são fornecidas composições e métodos para tratar Mucopolissacaridose Tipo IIIB (Síndrome de Sanfilippo B) compreendendo uma proteína de fusão terapêutica direcionada compreendendo alfa-N-acetilglicosaminidase (Naglu), uma fração de alvo lisossômica, por exemplo, um peptídeo IGF-II e um peptídeo espaçador.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261730378P | 2012-11-27 | 2012-11-27 | |
| US61/730,378 | 2012-11-27 | ||
| US201361788968P | 2013-03-15 | 2013-03-15 | |
| US61/788,968 | 2013-03-15 | ||
| PCT/US2013/072287 WO2014085621A1 (en) | 2012-11-27 | 2013-11-27 | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| BR112015012152A2 true BR112015012152A2 (pt) | 2017-08-15 |
| BR112015012152B1 BR112015012152B1 (pt) | 2023-04-25 |
Family
ID=49753532
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112015012152-7A BR112015012152B1 (pt) | 2012-11-27 | 2013-11-27 | Proteína de fusão terapêutica direcionada que compreende uma enzima lisossomal, ácido nucleico, composição farmacêutica, método para produzir a dita proteína e usos da proteína para tratar uma doença de armazenamento lisossomal e mucopolissacaridose tipo iiib |
Country Status (27)
| Country | Link |
|---|---|
| US (8) | US9376480B2 (pt) |
| EP (2) | EP3115372B1 (pt) |
| JP (2) | JP6831176B2 (pt) |
| KR (4) | KR102385392B1 (pt) |
| CN (1) | CN104822701B (pt) |
| AR (1) | AR093626A1 (pt) |
| AU (1) | AU2013352184B2 (pt) |
| BR (1) | BR112015012152B1 (pt) |
| CA (1) | CA2892146A1 (pt) |
| CL (1) | CL2015001371A1 (pt) |
| CY (1) | CY1122555T1 (pt) |
| DK (2) | DK3115372T3 (pt) |
| ES (2) | ES2729997T3 (pt) |
| HK (1) | HK1216026A1 (pt) |
| HR (2) | HRP20181351T1 (pt) |
| HU (2) | HUE039334T2 (pt) |
| IL (3) | IL238824B (pt) |
| LT (1) | LT3115372T (pt) |
| MX (2) | MX367024B (pt) |
| PL (2) | PL2925776T3 (pt) |
| PT (2) | PT2925776T (pt) |
| RS (1) | RS58916B1 (pt) |
| RU (1) | RU2680581C2 (pt) |
| SI (1) | SI3115372T1 (pt) |
| TW (2) | TWI626250B (pt) |
| WO (1) | WO2014085621A1 (pt) |
| ZA (1) | ZA201503509B (pt) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| ES2830350T3 (es) | 2008-05-07 | 2021-06-03 | Biomarin Pharm Inc | Péptidos de dirección lisosómica y usos de los mismos |
| PL2925776T3 (pl) | 2012-11-27 | 2018-11-30 | Biomarin Pharmaceutical Inc. | Ukierunkowane terapeutyczne fuzyjne białka enzymu lizosomalnego i ich zastosowania |
| CA2912678C (en) * | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| WO2016025519A1 (en) * | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| US20180303877A1 (en) * | 2014-09-25 | 2018-10-25 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| MA40791A (fr) | 2014-10-24 | 2017-09-27 | Shire Human Genetic Therapies | Ciblage lysosomal d'enzymes et utilisations associées |
| TWI752907B (zh) * | 2015-05-08 | 2022-01-21 | 美商拜奧馬林製藥公司 | 用於治療cln2疾病之tpp1調配物及方法 |
| JP2018538008A (ja) | 2015-11-06 | 2018-12-27 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム酵素の取り込みを中和する抗体または他の因子の検出のための細胞ベースアッセイ |
| JP7109369B2 (ja) * | 2016-02-24 | 2022-07-29 | バイオマリン ファーマシューティカル インコーポレイテッド | ターゲティングされた治療用リソソーム酵素融合タンパク質、関連する製剤、およびその使用 |
| KR20230125339A (ko) | 2016-04-15 | 2023-08-29 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| CL2016003282A1 (es) | 2016-12-21 | 2017-08-18 | Univ Chile | Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington |
| BR112020005249A2 (pt) | 2017-09-22 | 2020-09-24 | The Trustees Of The University Of Pennsylvania | terapia de gene para tratar mucopolissacaridose tipo ii |
| JP2021500857A (ja) | 2017-10-02 | 2021-01-14 | デナリ セラピューティクス インコーポレイテッドＤｅｎａｌｉ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． | 酵素補充療法用酵素を含む融合タンパク質 |
| MA54792A (fr) * | 2018-03-09 | 2021-12-01 | Avrobio Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
| AU2019261982A1 (en) * | 2018-04-30 | 2020-10-15 | Amicus Therapeutics, Inc. | Gene therapy constructs and methods of use |
| CN112243444A (zh) | 2018-06-08 | 2021-01-19 | 豪夫迈·罗氏有限公司 | 具有减少的翻译后修饰的肽接头 |
| BR112021006829A2 (pt) | 2018-10-10 | 2021-07-20 | Amicus Therapeutics, Inc. | composições de polipeptídeos estabilizados com ligações dissulfeto e métodos de uso |
| US20220133906A1 (en) * | 2018-11-16 | 2022-05-05 | Asklepios Biopharmaceutical, Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal teargeting sequence |
| JP2022552254A (ja) * | 2019-10-10 | 2022-12-15 | アミカス セラピューティックス インコーポレイテッド | バリアントｉｇｆ２構築体 |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| CN117343197A (zh) * | 2021-10-15 | 2024-01-05 | 中山大学 | 重组融合抗体 |
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| US5206161A (en) | 1991-02-01 | 1993-04-27 | Genentech, Inc. | Human plasma carboxypeptidase B |
| AUPN674895A0 (en) | 1995-11-23 | 1995-12-14 | Women's And Children's Hospital | Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same |
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| DE10035433C2 (de) | 2000-07-20 | 2002-07-18 | Tuma Wolfgang | Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben |
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| DE602005020745D1 (de) | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
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| ES2830350T3 (es) * | 2008-05-07 | 2021-06-03 | Biomarin Pharm Inc | Péptidos de dirección lisosómica y usos de los mismos |
| US8697654B2 (en) * | 2008-12-18 | 2014-04-15 | E I Du Pont De Nemours And Company | Peptide linkers for effective multivalent peptide binding |
| US9029530B2 (en) | 2009-01-02 | 2015-05-12 | Biomarin Pharmaceutical Inc. | Detection of oligosaccharides |
| ES2569514T3 (es) * | 2009-06-17 | 2016-05-11 | Biomarin Pharmaceutical Inc. | Formulaciones para enzimas lisosómicas |
| MX2013000324A (es) * | 2010-06-25 | 2013-02-01 | Shire Human Genetic Therapies | Tratamiento del sindrome de sanfilippo tipo b. |
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| WO2012088461A2 (en) * | 2010-12-23 | 2012-06-28 | Biogen Idec Inc. | Linker peptides and polypeptides comprising same |
| BR112013022557A2 (pt) * | 2011-03-04 | 2017-08-01 | Shire Human Genetic Therapies | ligantes de peptídeo composições de polipeptídeo e métodos para uso dos mesmos |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| US9409958B2 (en) * | 2011-03-10 | 2016-08-09 | Novozymes, Inc. | Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same |
| PL2925776T3 (pl) | 2012-11-27 | 2018-11-30 | Biomarin Pharmaceutical Inc. | Ukierunkowane terapeutyczne fuzyjne białka enzymu lizosomalnego i ich zastosowania |
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2013
- 2013-11-27 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/ko active IP Right Grant
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 BR BR112015012152-7A patent/BR112015012152B1/pt active IP Right Grant
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 MX MX2015006644A patent/MX367024B/es active IP Right Grant
- 2013-11-27 TW TW102143329A patent/TWI626250B/zh active
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active IP Right Grant
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 PL PL16182420T patent/PL3115372T3/pl unknown
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 AR ARP130104367A patent/AR093626A1/es active IP Right Grant
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 RU RU2015125491A patent/RU2680581C2/ru active
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en active Application Filing
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 KR KR1020237012007A patent/KR20230054482A/ko not_active Application Discontinuation
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active IP Right Grant
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 EP EP13802834.5A patent/EP2925776B1/en active Active
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2015
- 2015-05-14 IL IL238824A patent/IL238824B/en active IP Right Grant
- 2015-05-19 ZA ZA2015/03509A patent/ZA201503509B/en unknown
- 2015-05-20 CL CL2015001371A patent/CL2015001371A1/es unknown
- 2015-05-26 MX MX2019009191A patent/MX2019009191A/es active IP Right Grant
- 2015-10-14 US US14/883,193 patent/US9834587B2/en active Active
- 2015-10-14 US US14/883,211 patent/US9771408B2/en active Active
- 2015-10-14 US US14/883,169 patent/US9845346B2/en active Active
- 2015-10-14 US US14/883,203 patent/US9834588B2/en active Active
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2016
- 2016-04-06 HK HK16103915.8A patent/HK1216026A1/zh unknown
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2017
- 2017-08-28 US US15/688,438 patent/US10301369B2/en active Active
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2018
- 2018-08-23 HR HRP20181351TT patent/HRP20181351T1/hr unknown
- 2018-11-13 IL IL262976A patent/IL262976B/en active IP Right Grant
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2019
- 2019-04-08 US US16/378,163 patent/US11254725B2/en active Active
- 2019-05-17 HR HRP20190918TT patent/HRP20190918T1/hr unknown
- 2019-06-06 CY CY20191100600T patent/CY1122555T1/el unknown
- 2019-07-24 JP JP2019136013A patent/JP6913719B2/ja active Active
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2020
- 2020-02-23 IL IL272854A patent/IL272854B/en unknown
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2022
- 2022-01-10 US US17/572,018 patent/US20220127326A1/en not_active Abandoned
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