HUE044182T2 - Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezelésére - Google Patents
Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezeléséreInfo
- Publication number
- HUE044182T2 HUE044182T2 HUE12152550A HUE044182T2 HU E044182 T2 HUE044182 T2 HU E044182T2 HU E12152550 A HUE12152550 A HU E12152550A HU E044182 T2 HUE044182 T2 HU E044182T2
- Authority
- HU
- Hungary
- Prior art keywords
- gene therapy
- spinal cord
- lateral sclerosis
- amyotrophic lateral
- cord disorders
- Prior art date
Links
- 208000029033 Spinal Cord disease Diseases 0.000 title 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
Classifications
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/22—Hormones
- A61K38/30—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
- 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
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
-
- 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
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- 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
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Zoology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US81141906P | 2006-06-07 | 2006-06-07 |
Publications (1)
Publication Number | Publication Date |
---|---|
HUE044182T2 true HUE044182T2 (hu) | 2019-10-28 |
Family
ID=38832369
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE12152550 HUE044182T2 (hu) | 2006-06-07 | 2007-06-07 | Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezelésére |
HUE07795835A HUE031156T2 (hu) | 2006-06-07 | 2007-06-07 | Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE07795835A HUE031156T2 (hu) | 2006-06-07 | 2007-06-07 | Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen |
Country Status (19)
Country | Link |
---|---|
US (4) | US9034836B2 (hu) |
EP (3) | EP3540054A3 (hu) |
JP (6) | JP2009539847A (hu) |
CN (1) | CN101460621B (hu) |
AR (2) | AR061282A1 (hu) |
BR (1) | BRPI0711965A2 (hu) |
CA (2) | CA3139726A1 (hu) |
CY (1) | CY1118555T1 (hu) |
ES (2) | ES2725552T3 (hu) |
HU (2) | HUE044182T2 (hu) |
IL (2) | IL195647A (hu) |
LT (2) | LT2489733T (hu) |
MX (4) | MX2008015530A (hu) |
PL (2) | PL2489733T3 (hu) |
PT (2) | PT2029742T (hu) |
RU (1) | RU2008152821A (hu) |
SI (2) | SI2029742T1 (hu) |
TR (1) | TR201906106T4 (hu) |
WO (1) | WO2007146046A2 (hu) |
Families Citing this family (35)
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EP3540054A3 (en) | 2006-06-07 | 2019-10-09 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
US20100324127A1 (en) * | 2008-01-16 | 2010-12-23 | Kay Denis G | Treating neurodegenerative diseases with progranulin |
US9573972B2 (en) | 2008-10-31 | 2017-02-21 | Neurodyn, Inc. | Nurotoxic sterol glycosides |
JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
US20150352185A1 (en) * | 2010-11-16 | 2015-12-10 | Denis G. Kay | Method for increasing neprilysin expression and activity |
CN103083667A (zh) * | 2011-11-01 | 2013-05-08 | 北京清美联创干细胞科技有限公司 | 嗅鞘细胞增殖试剂的连续给药方案 |
EP2850093B1 (en) | 2012-01-27 | 2019-01-09 | The Board of Trustees of the Leland Stanford Junior University | Therapeutic il-13 polypeptides |
US9663564B2 (en) | 2013-03-15 | 2017-05-30 | The Regents Of The University Of California | Vectors and methods to treat ischemia |
US20140065110A1 (en) | 2012-08-31 | 2014-03-06 | The Regents Of The University Of California | Genetically modified msc and therapeutic methods |
IN2015DN04172A (hu) | 2012-10-24 | 2015-10-16 | Daiichi Sankyo Companyltd | |
JP6232547B2 (ja) * | 2012-11-28 | 2017-11-22 | 国立大学法人名古屋大学 | 聴覚障害又は小脳性運動失調症の予防・治療剤 |
WO2014178863A1 (en) * | 2013-05-01 | 2014-11-06 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
ES2859605T3 (es) | 2013-07-26 | 2021-10-04 | Univ Iowa Res Found | Procedimientos y composiciones para tratar enfermedades del cerebro |
KR101548989B1 (ko) | 2013-07-30 | 2015-09-01 | 광주과학기술원 | SirT1을 이용한 관절 질환의 예방 또는 치료용 물질의 스크리닝 방법 |
US9119832B2 (en) | 2014-02-05 | 2015-09-01 | The Regents Of The University Of California | Methods of treating mild brain injury |
DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
IL283475B1 (en) * | 2015-05-28 | 2024-03-01 | Univ Cornell | Administering esterase C.1 using adenovirus as a treatment for vascular edema |
CL2015003024A1 (es) | 2015-10-09 | 2016-05-13 | Univ Chile | Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica. |
WO2017087486A1 (en) * | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
AU2017227803B2 (en) * | 2016-03-02 | 2024-05-02 | Defne AMADO | Therapy for frontotemporal dementia |
SG11201907714UA (en) | 2017-02-28 | 2019-09-27 | Univ Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
US11802294B2 (en) | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
CN112481269A (zh) | 2017-10-03 | 2021-03-12 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
AU2018347796A1 (en) * | 2017-10-10 | 2020-05-07 | Medicenna Therapeutics, Inc. | IL-4-fusion formulations for treatment of central nervous system (CNS) tumors |
RU2731514C2 (ru) * | 2018-12-21 | 2020-09-03 | Селл энд Джин Терапи Лтд | Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора |
JP2022523913A (ja) * | 2019-02-01 | 2022-04-27 | アブロバイオ,インコーポレーテッド | 神経認知障害を処置するための組成物及び方法 |
CN113710281A (zh) * | 2019-02-22 | 2021-11-26 | 宾夕法尼亚州大学信托人 | 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒 |
CN114174324A (zh) | 2019-04-10 | 2022-03-11 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
BR112021020421A2 (pt) * | 2019-04-12 | 2021-12-21 | Encoded Therapeutics Inc | Composições e métodos para administração de componentes terapêuticos |
WO2021068242A1 (en) * | 2019-10-12 | 2021-04-15 | Shenzhen University | An agent that enables sirt7 gene expression and the use thereof |
CA3165846A1 (en) | 2019-12-23 | 2021-07-01 | Denali Therapeutics Inc. | Progranulin variants |
CN115323002A (zh) * | 2022-07-28 | 2022-11-11 | 深圳先进技术研究院 | 一种基因递送***在从脑部逆行递送基因到脊髓神经元中的应用 |
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JP2002322068A (ja) * | 2001-02-26 | 2002-11-08 | Japan Science & Technology Corp | 血管再生促進剤 |
EP1402043A1 (en) * | 2001-07-03 | 2004-03-31 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
WO2003055983A2 (en) | 2001-12-21 | 2003-07-10 | The Salk Institute For Biological Studies | Targeted retrograde gene delivery to motor neurons |
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AU2003215297A1 (en) * | 2002-02-15 | 2003-09-09 | Cornell Research Foundation, Inc. | Enhancing neurotrophin-induced neurogenesis by endogenous neural progenitor cells by concurrent overexpression of brain derived neurotrophic factor and an inhibitor of a pro-gliogenic bone morphogenetic protein |
US20060246036A1 (en) * | 2002-09-06 | 2006-11-02 | The General Hospital Corporation | Delivery of therapeutics to the brain and spinal cord |
JPWO2006035741A1 (ja) | 2004-09-29 | 2008-05-15 | 伸弥 山中 | Es細胞特異的発現遺伝子及びその利用 |
US7951928B2 (en) | 2004-11-18 | 2011-05-31 | Tokyo Medical And Dental University | Gene encoding a protein and preventive/remedy for neurodegenerative diseases such as polyglutamine diseases by utilizing the same |
CN104306986A (zh) | 2005-05-02 | 2015-01-28 | 建新公司 | 神经代谢疾病的基因治疗 |
CN105770909A (zh) * | 2005-05-02 | 2016-07-20 | 建新公司 | 脊髓失调症的基因治疗 |
EP3540054A3 (en) | 2006-06-07 | 2019-10-09 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
PL2773354T3 (pl) | 2011-11-01 | 2019-12-31 | Resverlogix Corp. | Doustna formulacja o natychmiastowym uwalnianiu dla podstawowych chinazolinów |
US20150182637A1 (en) | 2012-06-21 | 2015-07-02 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
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2007
- 2007-06-07 EP EP19156264.4A patent/EP3540054A3/en active Pending
- 2007-06-07 AR ARP070102460A patent/AR061282A1/es not_active Application Discontinuation
- 2007-06-07 PL PL12152550T patent/PL2489733T3/pl unknown
- 2007-06-07 CN CN200780020891.5A patent/CN101460621B/zh active Active
- 2007-06-07 ES ES12152550T patent/ES2725552T3/es active Active
- 2007-06-07 PL PL07795835T patent/PL2029742T3/pl unknown
- 2007-06-07 MX MX2008015530A patent/MX2008015530A/es active IP Right Grant
- 2007-06-07 MX MX2015001403A patent/MX364444B/es unknown
- 2007-06-07 EP EP07795835.3A patent/EP2029742B1/en active Active
- 2007-06-07 PT PT77958353T patent/PT2029742T/pt unknown
- 2007-06-07 TR TR2019/06106T patent/TR201906106T4/tr unknown
- 2007-06-07 JP JP2009514369A patent/JP2009539847A/ja not_active Withdrawn
- 2007-06-07 WO PCT/US2007/013391 patent/WO2007146046A2/en active Application Filing
- 2007-06-07 EP EP12152550.5A patent/EP2489733B1/en active Active
- 2007-06-07 SI SI200731840A patent/SI2029742T1/sl unknown
- 2007-06-07 BR BRPI0711965-8A patent/BRPI0711965A2/pt not_active Application Discontinuation
- 2007-06-07 RU RU2008152821/10A patent/RU2008152821A/ru not_active Application Discontinuation
- 2007-06-07 CA CA3139726A patent/CA3139726A1/en active Pending
- 2007-06-07 SI SI200732107T patent/SI2489733T1/sl unknown
- 2007-06-07 HU HUE12152550 patent/HUE044182T2/hu unknown
- 2007-06-07 ES ES07795835.3T patent/ES2596885T3/es active Active
- 2007-06-07 LT LTEP12152550.5T patent/LT2489733T/lt unknown
- 2007-06-07 HU HUE07795835A patent/HUE031156T2/hu unknown
- 2007-06-07 LT LTEP07795835.3T patent/LT2029742T/lt unknown
- 2007-06-07 PT PT12152550T patent/PT2489733T/pt unknown
- 2007-06-07 CA CA2654292A patent/CA2654292C/en active Active
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2008
- 2008-12-02 IL IL195647A patent/IL195647A/en active IP Right Grant
- 2008-12-04 MX MX2019004843A patent/MX2019004843A/es unknown
- 2008-12-04 US US12/328,267 patent/US9034836B2/en active Active
- 2008-12-04 MX MX2023001965A patent/MX2023001965A/es unknown
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2013
- 2013-08-22 JP JP2013172437A patent/JP2014012697A/ja active Pending
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2015
- 2015-01-23 JP JP2015011696A patent/JP2015120719A/ja not_active Withdrawn
- 2015-04-21 US US14/692,469 patent/US20160067312A1/en not_active Abandoned
- 2015-10-06 IL IL241890A patent/IL241890A0/en unknown
-
2016
- 2016-10-19 CY CY20161101055T patent/CY1118555T1/el unknown
- 2016-11-10 JP JP2016219801A patent/JP6320489B2/ja active Active
-
2017
- 2017-07-27 AR ARP170102115A patent/AR109171A2/es unknown
-
2018
- 2018-04-03 JP JP2018071383A patent/JP6606215B2/ja active Active
-
2019
- 2019-08-20 US US16/546,070 patent/US11554161B2/en active Active
- 2019-10-17 JP JP2019190202A patent/JP2020079234A/ja active Pending
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2022
- 2022-12-14 US US18/066,149 patent/US20230364200A1/en active Pending
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