IL215804A - Gene vector, a pharmacological drug for use in gene therapy to prevent and reduce the expression of a nucleotide sequence in a hematopoietic or non-sorted but non-sorted stem cell and a method for in vitro - Google Patents
Gene vector, a pharmacological drug for use in gene therapy to prevent and reduce the expression of a nucleotide sequence in a hematopoietic or non-sorted but non-sorted stem cell and a method for in vitroInfo
- Publication number
- IL215804A IL215804A IL215804A IL21580411A IL215804A IL 215804 A IL215804 A IL 215804A IL 215804 A IL215804 A IL 215804A IL 21580411 A IL21580411 A IL 21580411A IL 215804 A IL215804 A IL 215804A
- Authority
- IL
- Israel
- Prior art keywords
- cell
- hematopoietic
- hematopoietic stem
- hematopoietic progenitor
- stem cell
- Prior art date
Links
- 210000003958 hematopoietic stem cell Anatomy 0.000 title 4
- 210000004027 cell Anatomy 0.000 title 2
- 230000004069 differentiation Effects 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 238000000338 in vitro Methods 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000002773 nucleotide Substances 0.000 title 1
- 125000003729 nucleotide group Chemical group 0.000 title 1
- 239000008194 pharmaceutical composition Substances 0.000 title 1
- 108090000623 proteins and genes Proteins 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/04—Immunostimulants
-
- 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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/555—Interferons [IFN]
- C07K14/56—IFN-alpha
-
- 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
-
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US17412409P | 2009-04-30 | 2009-04-30 | |
| PCT/IB2010/001166 WO2010125471A2 (en) | 2009-04-30 | 2010-04-30 | Gene vector |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| IL215804A0 IL215804A0 (en) | 2012-01-31 |
| IL215804A true IL215804A (en) | 2016-10-31 |
Family
ID=42790671
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL215804A IL215804A (en) | 2009-04-30 | 2011-10-23 | Gene vector, a pharmacological drug for use in gene therapy to prevent and reduce the expression of a nucleotide sequence in a hematopoietic or non-sorted but non-sorted stem cell and a method for in vitro |
Country Status (23)
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2011011508A (es) | 2009-04-30 | 2012-02-13 | San Raffaele Centro Fond | Vector genico. |
| SI2772265T1 (en) | 2010-05-14 | 2018-06-29 | Oregon Health & Science University | HCMV AND RHCMV RECOMBINANT VECTORS AND THEIR APPLICATIONS |
| WO2012006577A2 (en) * | 2010-07-08 | 2012-01-12 | Duke University | Direct reprogramming of cells to cardiac myocyte fate |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| WO2013152230A1 (en) * | 2012-04-04 | 2013-10-10 | The Trustees Of Columbia University In The City Of New York | Smooth muscle specific inhibition for anti-restenotic therapy |
| GB2537000C (en) | 2012-05-25 | 2019-10-09 | Univ California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| CA2878037C (en) * | 2012-07-11 | 2021-08-31 | Sangamo Biosciences, Inc. | Methods and compositions for delivery of biologics |
| CA2891347C (en) | 2012-12-06 | 2018-02-27 | Sigma-Aldrich Co. Llc | Crispr-based genome modification and regulation |
| PL2956477T5 (pl) | 2013-02-15 | 2024-05-27 | Bioverativ Therapeutics Inc. | Zoptymalizowany gen czynnika viii |
| AU2014235794A1 (en) | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| EP3060670B1 (en) | 2013-10-24 | 2019-07-10 | Ospedale San Raffaele S.r.l. | Method |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| EP3318630B1 (en) * | 2015-04-22 | 2020-11-04 | iPS Academia Japan, Inc. | Method for sorting tissue cells |
| WO2016201507A1 (en) * | 2015-06-15 | 2016-12-22 | Murdoch Childrens Research Institute | Method of measuring chimerism |
| US10849920B2 (en) | 2015-10-05 | 2020-12-01 | Modernatx, Inc. | Methods for therapeutic administration of messenger ribonucleic acid drugs |
| US10188750B1 (en) | 2015-10-23 | 2019-01-29 | University Of South Florida | Self-replicating cell selective gene delivery compositions, methods, and uses thereof |
| RS63548B1 (sr) | 2016-02-01 | 2022-09-30 | Bioverativ Therapeutics Inc | Optimizovani geni faktora viii |
| GB201612214D0 (en) | 2016-07-14 | 2016-08-31 | Univ Oxford Innovation Ltd | Method |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| CA3040303A1 (en) * | 2016-10-18 | 2018-04-26 | Oregon Health & Science University | Cytomegalovirus vectors eliciting t cells restricted by major histocompatibility complex e molecules |
| CN106566838B (zh) * | 2016-11-14 | 2019-11-01 | 上海伯豪生物技术有限公司 | 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用 |
| WO2018193119A1 (en) * | 2017-04-21 | 2018-10-25 | Ospedale San Raffaele S.R.L. | Gene therapy |
| IT201700086403A1 (it) * | 2017-07-27 | 2019-01-27 | Univ Del Piemonte Orientale | Endothelial-specific promoter sequences and uses thereof |
| KR20250040746A (ko) | 2017-08-09 | 2025-03-24 | 바이오버라티브 테라퓨틱스 인크. | 핵산 분자 및 이의 용도 |
| CN111918674A (zh) | 2018-02-01 | 2020-11-10 | 比奥维拉迪维治疗股份有限公司 | 表达因子viii的慢病毒载体的用途 |
| JP7602454B2 (ja) | 2018-08-09 | 2024-12-18 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 |
| SG11202105880TA (en) | 2018-12-06 | 2021-07-29 | Bioverativ Therapeutics Inc | Use of lentiviral vectors expressing factor ix |
| US12403164B2 (en) | 2019-09-30 | 2025-09-02 | Bioverativ Therapeutics Inc. | Lentiviral vector formulations |
| CA3161175A1 (en) * | 2019-11-12 | 2021-05-20 | The Regents Of The University Of California | Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease |
| BR112022026127A2 (pt) | 2020-06-24 | 2023-01-17 | Bioverativ Therapeutics Inc | Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína |
| AU2021330936A1 (en) | 2020-08-23 | 2023-05-04 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| JP2024534123A (ja) | 2021-08-23 | 2024-09-18 | バイオベラティブ セラピューティクス インコーポレイテッド | バキュロウイルス発現システム |
| TW202323274A (zh) | 2021-08-23 | 2023-06-16 | 美商百歐維拉提夫治療公司 | 優化因子viii基因 |
| CN115725656A (zh) * | 2021-09-01 | 2023-03-03 | 联邦生物科技(珠海横琴)有限公司 | 一种以细胞质rna病毒为载体递送干扰rna的方法 |
| AU2022355088A1 (en) | 2021-09-30 | 2024-05-16 | Bioverativ Therapeutics Inc. | Nucleic acids encoding factor viii polypeptides with reduced immunogenicity |
| EP4561638A2 (en) * | 2022-07-25 | 2025-06-04 | ModernaTX, Inc. | Engineered polynucleotides for cell selective expression |
| WO2025122739A1 (en) * | 2023-12-05 | 2025-06-12 | University Of Pittsburgh-Of The Commonwealth System Of Higher Education | Compositions and methods for treating a heart disease |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5723315A (en) | 1996-08-23 | 1998-03-03 | Genetics Institute, Inc. | Secreted proteins and polynucleotides encoding them |
| US6118001A (en) | 1996-08-07 | 2000-09-12 | Darwin Discovery, Ltd. | Hydroxamic and carboxylic acid derivatives having MMP and TNF inhibitory activity |
| US6126939A (en) | 1996-09-03 | 2000-10-03 | Yeda Research And Development Co. Ltd. | Anti-inflammatory dipeptide and pharmaceutical composition thereof |
| NZ532060A (en) * | 2001-10-02 | 2005-11-25 | Inst Clayton De La Rech | Methods and compositions relating to restricted expression lentiviral vectors and their applications |
| EP2290071B1 (en) | 2004-05-28 | 2014-12-31 | Asuragen, Inc. | Methods and compositions involving microRNA |
| US20060134784A1 (en) * | 2004-11-30 | 2006-06-22 | Basch Ross S | Methods and compositions for the growth and maintenance of stem cells |
| SI2002003T1 (sl) | 2005-05-27 | 2016-05-31 | Ospedale San Raffaele S.R.L. | Genski vektor, ki vsebuje mi-RNA |
| CA2610702A1 (en) * | 2005-06-03 | 2006-12-07 | Michael Zenon Michael | Targeting cells with altered microrna expression |
| US20090180989A1 (en) * | 2005-10-05 | 2009-07-16 | Synageva Biopharma Corp. | Compositions and methods for delivering nucleotide sequences to vertebrates |
| EP2479286B1 (en) | 2006-01-05 | 2013-11-13 | The Ohio State University Research Foundation | MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers |
| US20080095750A1 (en) | 2006-05-10 | 2008-04-24 | Pennington Biomedical Research Center, Louisiana State University System | Use of adipose-derived stem cells for treatment of leukodystrophies |
| WO2008073921A2 (en) | 2006-12-08 | 2008-06-19 | Asuragen, Inc. | Mir-126 regulated genes and pathways as targets for therapeutic intervention |
| ES2635726T3 (es) | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| EP2364362B1 (en) | 2008-11-12 | 2015-10-21 | Ospedale San Raffaele S.r.l. | Gene vector for inducing transgene-specific immune tolerance |
| MX2011011508A (es) * | 2009-04-30 | 2012-02-13 | San Raffaele Centro Fond | Vector genico. |
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2010
- 2010-04-30 MX MX2011011508A patent/MX2011011508A/es active IP Right Grant
- 2010-04-30 PT PT107233058T patent/PT2424571T/pt unknown
- 2010-04-30 EP EP20167404.1A patent/EP3733214A1/en active Pending
- 2010-04-30 JP JP2012507847A patent/JP6144911B2/ja active Active
- 2010-04-30 SI SI201032001T patent/SI2424571T1/sl unknown
- 2010-04-30 PL PL10723305T patent/PL2424571T3/pl unknown
- 2010-04-30 EA EA201171335A patent/EA023938B1/ru not_active IP Right Cessation
- 2010-04-30 HR HRP20200647TT patent/HRP20200647T1/hr unknown
- 2010-04-30 SG SG2011080009A patent/SG175839A1/en unknown
- 2010-04-30 AU AU2010243276A patent/AU2010243276B2/en active Active
- 2010-04-30 LT LTEP10723305.8T patent/LT2424571T/lt unknown
- 2010-04-30 SG SG10201610827RA patent/SG10201610827RA/en unknown
- 2010-04-30 KR KR1020117028569A patent/KR101793615B1/ko active Active
- 2010-04-30 DK DK10723305.8T patent/DK2424571T3/da active
- 2010-04-30 HU HUE10723305A patent/HUE049845T2/hu unknown
- 2010-04-30 CN CN201080030337.7A patent/CN102596255B/zh active Active
- 2010-04-30 ES ES10723305T patent/ES2786039T3/es active Active
- 2010-04-30 US US13/266,381 patent/US10287579B2/en active Active
- 2010-04-30 CA CA2759438A patent/CA2759438C/en active Active
- 2010-04-30 WO PCT/IB2010/001166 patent/WO2010125471A2/en not_active Ceased
- 2010-04-30 BR BRPI1010873A patent/BRPI1010873B1/pt active IP Right Grant
- 2010-04-30 SM SM20200224T patent/SMT202000224T1/it unknown
- 2010-04-30 EP EP10723305.8A patent/EP2424571B1/en active Active
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2011
- 2011-10-23 IL IL215804A patent/IL215804A/en active IP Right Grant
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2015
- 2015-09-07 JP JP2015175986A patent/JP6370278B2/ja active Active
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2017
- 2017-08-31 US US15/693,112 patent/US9951328B2/en active Active
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2019
- 2019-04-15 US US16/384,571 patent/US11407996B2/en active Active
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2020
- 2020-05-04 CY CY20201100405T patent/CY1123218T1/el unknown
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2022
- 2022-06-30 US US17/855,135 patent/US20220333103A1/en active Pending
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Legal Events
| Date | Code | Title | Description |
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| FF | Patent granted | ||
| KB | Patent renewed | ||
| KB | Patent renewed |