AR093626A1 - Proteinas de fusion de enzima lisosomal y usos de las mismas - Google Patents
Proteinas de fusion de enzima lisosomal y usos de las mismasInfo
- Publication number
- AR093626A1 AR093626A1 ARP130104367A ARP130104367A AR093626A1 AR 093626 A1 AR093626 A1 AR 093626A1 AR P130104367 A ARP130104367 A AR P130104367A AR P130104367 A ARP130104367 A AR P130104367A AR 093626 A1 AR093626 A1 AR 093626A1
- Authority
- AR
- Argentina
- Prior art keywords
- seq
- peptide
- lysosomal
- igf
- therapeutic fusion
- Prior art date
Links
- 102000037865 fusion proteins Human genes 0.000 title abstract 5
- 108020001507 fusion proteins Proteins 0.000 title abstract 5
- 102000004190 Enzymes Human genes 0.000 title abstract 4
- 108090000790 Enzymes Proteins 0.000 title abstract 4
- 230000002132 lysosomal effect Effects 0.000 abstract 5
- 101800001707 Spacer peptide Proteins 0.000 abstract 4
- 230000001225 therapeutic effect Effects 0.000 abstract 4
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 abstract 3
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 2
- 108040004574 (S)-3-O-geranylgeranylglyceryl phosphate synthase activity proteins Proteins 0.000 abstract 1
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 abstract 1
- 150000001413 amino acids Chemical class 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 102000057877 human IGF2 Human genes 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
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 208000011580 syndromic disease Diseases 0.000 abstract 1
Classifications
-
- 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
-
- 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
- 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)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Biophysics (AREA)
- Toxicology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261730378P | 2012-11-27 | 2012-11-27 | |
| US201361788968P | 2013-03-15 | 2013-03-15 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR093626A1 true AR093626A1 (es) | 2015-06-17 |
Family
ID=49753532
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP130104367A AR093626A1 (es) | 2012-11-27 | 2013-11-27 | Proteinas de fusion de enzima lisosomal y usos de las mismas |
Country Status (26)
| Country | Link |
|---|---|
| US (9) | US9376480B2 (enExample) |
| EP (2) | EP2925776B1 (enExample) |
| JP (2) | JP6831176B2 (enExample) |
| KR (4) | KR20230054482A (enExample) |
| CN (1) | CN104822701B (enExample) |
| AR (1) | AR093626A1 (enExample) |
| AU (1) | AU2013352184B2 (enExample) |
| BR (1) | BR112015012152B1 (enExample) |
| CA (1) | CA2892146A1 (enExample) |
| CL (1) | CL2015001371A1 (enExample) |
| CY (1) | CY1122555T1 (enExample) |
| DK (2) | DK3115372T3 (enExample) |
| ES (2) | ES2679374T3 (enExample) |
| HR (2) | HRP20181351T1 (enExample) |
| HU (2) | HUE043679T2 (enExample) |
| IL (3) | IL238824B (enExample) |
| LT (1) | LT3115372T (enExample) |
| MX (2) | MX377150B (enExample) |
| PL (2) | PL3115372T3 (enExample) |
| PT (2) | PT2925776T (enExample) |
| RS (1) | RS58916B1 (enExample) |
| RU (1) | RU2680581C2 (enExample) |
| SI (1) | SI3115372T1 (enExample) |
| TW (2) | TWI711632B (enExample) |
| WO (1) | WO2014085621A1 (enExample) |
| ZA (1) | ZA201503509B (enExample) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2629853T3 (es) | 2008-05-07 | 2017-08-16 | Biomarin Pharmaceutical Inc. | Péptidos de dirección lisosómica y usos de los mismos |
| LT3115372T (lt) | 2012-11-27 | 2019-06-25 | Biomarin Pharmaceutical Inc. | Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas |
| KR20160010526A (ko) * | 2013-05-15 | 2016-01-27 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| MX2017001898A (es) * | 2014-08-11 | 2017-04-11 | Shire Human Genetic Therapies | Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales. |
| US20180303877A1 (en) * | 2014-09-25 | 2018-10-25 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| EP3220958A1 (en) * | 2014-10-24 | 2017-09-27 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
| 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 |
| MY189159A (en) | 2015-07-06 | 2022-01-29 | Regeneron Pharma | Multispecific antigen-binding molecules and uses thereof |
| MX2018005258A (es) | 2015-11-06 | 2019-09-04 | Biomarin Pharm Inc | Ensayos basados en células para la detección de anticuerpos u otros factores que neutralizan la absorción de enzimas lisosómicas. |
| IL261246B2 (en) * | 2016-02-24 | 2023-03-01 | Biomarin Pharm Inc | Therapeutic fusion proteins targeting lysosomal enzymes, their formulations and their uses |
| AU2017250298B2 (en) | 2016-04-15 | 2024-10-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| 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 |
| ES3065816T3 (en) | 2017-06-07 | 2026-05-08 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| JP7449223B2 (ja) | 2017-09-22 | 2024-03-13 | 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ｉｉ型を治療するための遺伝子療法 |
| MX2020002918A (es) | 2017-10-02 | 2020-07-22 | Denali Therapeutics Inc | Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas. |
| CN112040985A (zh) | 2018-02-07 | 2020-12-04 | 瑞泽恩制药公司 | 用于递送治疗性蛋白质的方法和组合物 |
| CA3092961A1 (en) * | 2018-03-09 | 2019-09-12 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| JP7749322B2 (ja) | 2018-04-30 | 2025-10-06 | アミカス セラピューティックス インコーポレイテッド | 遺伝子治療構築物及び使用方法 |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| 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 |
| EA202190999A1 (ru) | 2018-10-10 | 2021-10-15 | Амикус Терапьютикс, Инк. | Композиции на основе полипептидов, стабилизированных с помощью дисульфидной связи, и способы применения |
| EP3880830A4 (en) * | 2018-11-16 | 2022-08-24 | Asklepios Biopharmaceutical, Inc. | VECTORS WITH A NUCLEIC ACID ENCODING LYSOSOMAL ENZYMES CONDENSED TO A LYSOSOMAL TARGETING SEQUENCE |
| AU2020361703A1 (en) * | 2019-10-10 | 2022-04-28 | Amicus Therapeutics, Inc. | Variant IGF2 constructs |
| US12358963B2 (en) | 2019-12-24 | 2025-07-15 | Juvena Therapeutics, Inc. | Regenerative polypeptides and uses thereof |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| CA3223707A1 (en) | 2021-06-21 | 2022-12-29 | Hanadie Yousef | Regenerative polypeptides and uses thereof |
| US20240279360A1 (en) * | 2021-06-23 | 2024-08-22 | Lycia Therapeutics, Inc. | Bifunctional compounds containing igf-2 polypeptides |
| CN117343197A (zh) * | 2021-10-15 | 2024-01-05 | 中山大学 | 重组融合抗体 |
| WO2024159071A1 (en) * | 2023-01-27 | 2024-08-02 | Regeneron Pharmaceuticals, Inc. | Modified rhabdovirus glycoproteins and uses thereof |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| DE10035433C2 (de) | 2000-07-20 | 2002-07-18 | Tuma Wolfgang | Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben |
| WO2003102583A1 (en) | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| EP1974752B1 (en) * | 2001-04-30 | 2012-09-26 | BioMarin Pharmaceutical Inc. | Subcellular targeting of therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| IL161352A0 (en) | 2001-10-16 | 2004-09-27 | Symbiontics Inc | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| US7485314B2 (en) | 2002-05-06 | 2009-02-03 | Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center | Induction of antigen specific immunologic tolerance |
| JP4629047B2 (ja) | 2003-06-12 | 2011-02-09 | イーライ リリー アンド カンパニー | Ｇｌｐ−１アナログ複合タンパク質 |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| CA2553955C (en) * | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| JP2008531060A (ja) | 2005-03-04 | 2008-08-14 | バイオジェン・アイデック・エムエイ・インコーポレイテッド | 相補性決定残基の合理的改変を介して免疫グロブリン可変領域をヒト化する方法 |
| ES2629853T3 (es) | 2008-05-07 | 2017-08-16 | Biomarin Pharmaceutical 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 |
| EP2475376B1 (en) * | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| NZ702801A (en) * | 2010-06-25 | 2016-08-26 | Shire Human Genetic Therapies | Treatment of sanfilippo syndrome type b |
| EP2585385B1 (en) | 2010-06-25 | 2019-12-18 | Amcor Rigid Plastics USA, LLC | Oxygen scavenging system for a container |
| EP2655624B1 (en) * | 2010-12-23 | 2017-11-29 | Biogen MA Inc. | Linker peptides and polypeptides comprising same |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| CA3080181A1 (en) * | 2011-03-04 | 2012-09-13 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| WO2012122477A1 (en) * | 2011-03-10 | 2012-09-13 | Novozymes A/S | Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same |
| LT3115372T (lt) | 2012-11-27 | 2019-06-25 | Biomarin Pharmaceutical Inc. | Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas |
-
2013
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 RU RU2015125491A patent/RU2680581C2/ru active
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 PL PL16182420T patent/PL3115372T3/pl unknown
- 2013-11-27 KR KR1020237012007A patent/KR20230054482A/ko not_active Ceased
- 2013-11-27 MX MX2019009191A patent/MX377150B/es active IP Right Grant
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 BR BR112015012152-7A patent/BR112015012152B1/pt active IP Right Grant
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/ko active Active
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 EP EP13802834.5A patent/EP2925776B1/en active Active
- 2013-11-27 AR ARP130104367A patent/AR093626A1/es active IP Right Grant
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 TW TW102143329A patent/TWI626250B/zh active
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en not_active Ceased
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active Active
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 MX MX2015006644A patent/MX367024B/es active IP Right Grant
- 2013-11-27 HR HRP20181351TT patent/HRP20181351T1/hr unknown
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active Active
-
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
- 2015-10-14 US US14/883,203 patent/US9834588B2/en active Active
- 2015-10-14 US US14/883,169 patent/US9845346B2/en active Active
- 2015-10-14 US US14/883,193 patent/US9834587B2/en active Active
- 2015-10-14 US US14/883,211 patent/US9771408B2/en active Active
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2017
- 2017-08-28 US US15/688,438 patent/US10301369B2/en active Active
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2018
- 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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2025
- 2025-01-17 US US19/030,525 patent/US20250388644A1/en active Pending
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