JP2021019591A5 - - Google Patents
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- JP2021019591A5 JP2021019591A5 JP2020156320A JP2020156320A JP2021019591A5 JP 2021019591 A5 JP2021019591 A5 JP 2021019591A5 JP 2020156320 A JP2020156320 A JP 2020156320A JP 2020156320 A JP2020156320 A JP 2020156320A JP 2021019591 A5 JP2021019591 A5 JP 2021019591A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- acid sequence
- vector according
- aav2
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 150000007523 nucleic acids Chemical class 0.000 claims 44
- 239000013598 vector Substances 0.000 claims 26
- 108020004707 nucleic acids Proteins 0.000 claims 23
- 102000039446 nucleic acids Human genes 0.000 claims 23
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 21
- 102000003869 Frataxin Human genes 0.000 claims 17
- 108090000217 Frataxin Proteins 0.000 claims 17
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 11
- 210000000234 capsid Anatomy 0.000 claims 11
- 239000013608 rAAV vector Substances 0.000 claims 11
- 208000024412 Friedreich ataxia Diseases 0.000 claims 8
- 239000003814 drug Substances 0.000 claims 8
- 229940124597 therapeutic agent Drugs 0.000 claims 8
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 7
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 108700042658 GAP-43 Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 238000007917 intracranial administration Methods 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 102000008186 Collagen Human genes 0.000 claims 1
- 108010035532 Collagen Proteins 0.000 claims 1
- 241000701022 Cytomegalovirus Species 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 230000006978 adaptation Effects 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 229920001436 collagen Polymers 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 230000000087 stabilizing effect Effects 0.000 claims 1
- 238000004114 suspension culture Methods 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562251288P | 2015-11-05 | 2015-11-05 | |
| US62/251,288 | 2015-11-05 | ||
| US201662411980P | 2016-10-24 | 2016-10-24 | |
| US62/411,980 | 2016-10-24 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521395A Division JP6767483B2 (ja) | 2015-11-05 | 2016-11-01 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021019591A JP2021019591A (ja) | 2021-02-18 |
| JP2021019591A5 true JP2021019591A5 (enExample) | 2021-04-08 |
Family
ID=57288476
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521395A Expired - Fee Related JP6767483B2 (ja) | 2015-11-05 | 2016-11-01 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
| JP2020156320A Pending JP2021019591A (ja) | 2015-11-05 | 2020-09-17 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521395A Expired - Fee Related JP6767483B2 (ja) | 2015-11-05 | 2016-11-01 | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10548947B2 (enExample) |
| EP (1) | EP3370778A1 (enExample) |
| JP (2) | JP6767483B2 (enExample) |
| KR (2) | KR102200642B1 (enExample) |
| CN (1) | CN108348621A (enExample) |
| AU (2) | AU2016347887B2 (enExample) |
| BR (1) | BR112018007453A2 (enExample) |
| CA (1) | CA2947584A1 (enExample) |
| CO (1) | CO2018004564A2 (enExample) |
| HK (1) | HK1250924A1 (enExample) |
| IL (1) | IL259141A (enExample) |
| MX (1) | MX2018005084A (enExample) |
| PE (1) | PE20181156A1 (enExample) |
| PH (1) | PH12018500964A1 (enExample) |
| RU (2) | RU2021102893A (enExample) |
| SG (2) | SG11201802408RA (enExample) |
| TW (2) | TWI654301B (enExample) |
| WO (1) | WO2017077451A1 (enExample) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3286321A4 (en) * | 2015-04-24 | 2018-10-17 | University of Florida Research Foundation, Inc. | Aav vector for treatment of friedreich's ataxia |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| JP6745873B2 (ja) | 2015-05-22 | 2020-08-26 | ドミトリエヴィッチ ゲンキン，ドミトリー | 神経変性における治療標的としての細胞外ｄｎａ |
| CN108348621A (zh) * | 2015-11-05 | 2018-07-31 | 竹治疗股份有限公司 | 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体 |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| AU2017237912B2 (en) | 2016-03-23 | 2022-03-10 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| WO2018057615A1 (en) | 2016-09-21 | 2018-03-29 | Commscope Technologies Llc | Adapter block assembly |
| CA3042706A1 (en) * | 2016-11-09 | 2018-05-17 | Intrexon Corporation | Frataxin expression constructs |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| US11802294B2 (en) * | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| PL3697914T3 (pl) * | 2017-10-17 | 2023-10-16 | Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol | Wektory do leczenia ataksji friedreicha |
| JP2021510539A (ja) | 2018-01-16 | 2021-04-30 | シーエルエス セラピューティクス リミテッドＣＬＳ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｌｉｍｉｔｅｄ | デオキシリボヌクレアーゼ（ＤＮａｓｅ）活性を有する酵素の肝臓発現による疾患の処置 |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| JP2021533200A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
| KR20210086601A (ko) * | 2018-08-02 | 2021-07-08 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 프리드라이히 운동실조를 치료하기 위한 그의 용도 |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| CA3109114A1 (en) * | 2018-08-10 | 2020-02-13 | Logicbio Therapeutics, Inc. | Non-disruptive gene therapy for the treatment of methaylmalonic aciduria |
| SG11202103151RA (en) * | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3880179A2 (en) * | 2018-11-14 | 2021-09-22 | Flagship Pioneering Innovations V, Inc. | Fusosome compositions for cns delivery |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| EA202191418A1 (ru) * | 2018-11-21 | 2021-08-05 | Страйдбайо, Инк. | Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения |
| KR20210117271A (ko) * | 2018-12-21 | 2021-09-28 | 노쓰웨스턴유니버시티 | 근막 손상 예방 및 치료를 위한 아넥신의 사용근각 손상 예방 및 치료를 위한 아넥신의 사용 |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| AU2020248101A1 (en) * | 2019-03-27 | 2021-09-16 | Sigilon Therapeutics, Inc. | Compositions, devices and methods for factor VII therapy |
| BR112021019224A2 (pt) * | 2019-03-28 | 2021-11-30 | Intellia Therapeutics Inc | Polinucleotídeos, composições e métodos para expressão de polipeptídeo |
| WO2020210713A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| EP3989994A4 (en) * | 2019-06-30 | 2023-08-09 | John Fraser Wright | RECOMBINANT AAV VECTORS WITH ALTERED IMMUNOGENICITY AND METHODS FOR THEIR PRODUCTION |
| EP3999120A4 (en) * | 2019-07-04 | 2024-02-21 | Children's Medical Research Institute | Methods and aav vectors for in vivo transduction |
| US20220241394A1 (en) * | 2019-07-12 | 2022-08-04 | Cls Therapeutics Limited | TREATMENT OF DISEASES ASSOCIATED WITH PROTEIN MISFOLDING BY NERVOUS SYSTEM EXPRESSION OF AN ENZYME WHICH HAS A DEOXYRIBONUCLEASE (DNase) ACTIVITY |
| CA3151073A1 (en) * | 2019-09-13 | 2021-03-18 | Darin Falk | Compositions and methods for treatment of friedreich's ataxia |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| AU2020405149A1 (en) * | 2019-12-19 | 2022-07-07 | The Trustees Of The University Of Pennsylvania | Compositions for treating Friedreich's ataxia |
| CN113755524B (zh) * | 2020-06-02 | 2023-11-03 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途 |
| US11946065B2 (en) | 2020-07-29 | 2024-04-02 | The Board Of Regents Of The University Of Texas System | Transgene cassettes, AAV vectors, and AAV viral vectors for expression of human codon-optimized CSTB |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| IL302608A (en) | 2020-11-03 | 2023-07-01 | Pfizer | Methods for purification of aav vectors by anion exchange chromatography |
| IL303959A (en) | 2020-12-23 | 2023-08-01 | Pfizer | Methods for purification of aav vectors by affinity chromatography |
| CA3207161A1 (en) * | 2021-01-04 | 2022-07-07 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treatment of friedreich's ataxia |
| US20240050521A1 (en) * | 2021-01-11 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Compositions for treating friedreich's ataxia |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| CA3257440A1 (en) * | 2022-07-06 | 2024-01-11 | Adverum Biotechnologies Inc | COMPOSITIONS AND METHODS FOR THE TREATMENT OF ACHROMOTOPSY |
| CN117089514A (zh) * | 2023-10-13 | 2023-11-21 | 思鹏生物科技(苏州)有限公司 | 提升hek293细胞系aav生产效率的细胞筛选驯化方法 |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| AU707862B2 (en) | 1995-06-07 | 1999-07-22 | University Of North Carolina At Chapel Hill, The | Helper virus-free aav production |
| ATE465267T1 (de) | 1996-09-06 | 2010-05-15 | Univ Pennsylvania | Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen |
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| CA2399576A1 (en) | 2000-12-07 | 2002-06-13 | Universite De Nantes | Inducible highly productive raav packaging cell-lines |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| CA2591544A1 (en) | 2004-12-15 | 2006-06-22 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| US9163249B2 (en) * | 2007-08-20 | 2015-10-20 | Glaxo Group Limited | Production methods |
| ES2634118T3 (es) | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
| WO2011084145A2 (en) * | 2009-12-21 | 2011-07-14 | Pharmathene, Inc. | Recombinant butyrylcholinesterases and truncates thereof |
| WO2011097640A1 (en) * | 2010-02-08 | 2011-08-11 | The Trustees Of The University Of Pennsylvania | Nucleic acid molecules encoding rantes, and compositions comprising and methods of using the same |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| EP2718439B1 (en) * | 2011-06-09 | 2017-08-09 | CuRNA, Inc. | Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn |
| JP6165752B2 (ja) | 2011-10-28 | 2017-07-19 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | アデノ随伴ウイルスの産生のための細胞株 |
| US9066966B2 (en) | 2013-02-01 | 2015-06-30 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia |
| EP2970943A4 (en) | 2013-03-15 | 2017-04-19 | The Children's Hospital of Philadelphia | Vectors comprising stuffer/filler polynucleotide sequences and methods of use |
| CA2904396A1 (en) | 2013-03-15 | 2014-09-18 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav vectors |
| ES2897508T3 (es) | 2013-05-31 | 2022-03-01 | Univ California | Variantes de virus adenoasociados y métodos de uso de las mismas |
| RU2705249C2 (ru) | 2013-07-12 | 2019-11-06 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Вектор aav и анализ на нейтрализующие антитела против aav (аденоассоциированного вируса) |
| HUE047996T2 (hu) | 2013-07-22 | 2020-05-28 | Childrens Hospital Philadelphia | AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
| JP6552511B2 (ja) | 2013-10-11 | 2019-07-31 | マサチューセッツ アイ アンド イヤー インファーマリー | 祖先ウイルス配列を予測する方法およびその使用 |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| CN108348621A (zh) * | 2015-11-05 | 2018-07-31 | 竹治疗股份有限公司 | 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体 |
-
2016
- 2016-11-01 CN CN201680064440.0A patent/CN108348621A/zh active Pending
- 2016-11-01 WO PCT/IB2016/056572 patent/WO2017077451A1/en not_active Ceased
- 2016-11-01 KR KR1020187015819A patent/KR102200642B1/ko not_active Expired - Fee Related
- 2016-11-01 EP EP16795164.9A patent/EP3370778A1/en not_active Withdrawn
- 2016-11-01 SG SG11201802408RA patent/SG11201802408RA/en unknown
- 2016-11-01 PE PE2018000726A patent/PE20181156A1/es unknown
- 2016-11-01 BR BR112018007453A patent/BR112018007453A2/pt not_active IP Right Cessation
- 2016-11-01 JP JP2018521395A patent/JP6767483B2/ja not_active Expired - Fee Related
- 2016-11-01 RU RU2021102893A patent/RU2021102893A/ru unknown
- 2016-11-01 US US15/340,721 patent/US10548947B2/en active Active
- 2016-11-01 AU AU2016347887A patent/AU2016347887B2/en not_active Ceased
- 2016-11-01 SG SG10201912763QA patent/SG10201912763QA/en unknown
- 2016-11-01 RU RU2018116076A patent/RU2743792C2/ru active
- 2016-11-01 KR KR1020217000034A patent/KR20210005994A/ko not_active Withdrawn
- 2016-11-01 HK HK18110376.3A patent/HK1250924A1/zh unknown
- 2016-11-01 MX MX2018005084A patent/MX2018005084A/es unknown
- 2016-11-04 TW TW105135911A patent/TWI654301B/zh not_active IP Right Cessation
- 2016-11-04 CA CA2947584A patent/CA2947584A1/en active Pending
- 2016-11-04 TW TW108104049A patent/TWI743442B/zh not_active IP Right Cessation
-
2018
- 2018-04-27 CO CONC2018/0004564A patent/CO2018004564A2/es unknown
- 2018-05-03 PH PH12018500964A patent/PH12018500964A1/en unknown
- 2018-05-03 IL IL259141A patent/IL259141A/en unknown
-
2019
- 2019-12-16 US US16/716,033 patent/US20200384073A1/en not_active Abandoned
-
2020
- 2020-02-11 AU AU2020200976A patent/AU2020200976B2/en not_active Ceased
- 2020-09-17 JP JP2020156320A patent/JP2021019591A/ja active Pending
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