JP2022060228A5 - - Google Patents
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- Publication number
- JP2022060228A5 JP2022060228A5 JP2022007759A JP2022007759A JP2022060228A5 JP 2022060228 A5 JP2022060228 A5 JP 2022060228A5 JP 2022007759 A JP2022007759 A JP 2022007759A JP 2022007759 A JP2022007759 A JP 2022007759A JP 2022060228 A5 JP2022060228 A5 JP 2022060228A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- vector
- itr
- raav
- 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
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 26
- 150000007523 nucleic acids Chemical class 0.000 claims 25
- 108020004707 nucleic acids Proteins 0.000 claims 23
- 102000039446 nucleic acids Human genes 0.000 claims 23
- 108700019146 Transgenes Proteins 0.000 claims 17
- 239000013608 rAAV vector Substances 0.000 claims 16
- 210000004027 cell Anatomy 0.000 claims 14
- 239000013598 vector Substances 0.000 claims 12
- 241000702421 Dependoparvovirus Species 0.000 claims 10
- 238000002347 injection Methods 0.000 claims 9
- 239000007924 injection Substances 0.000 claims 9
- 108090000623 proteins and genes Proteins 0.000 claims 8
- 108090000565 Capsid Proteins Proteins 0.000 claims 6
- 102100023321 Ceruloplasmin Human genes 0.000 claims 6
- 239000002773 nucleotide Substances 0.000 claims 6
- 125000003729 nucleotide group Chemical group 0.000 claims 6
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 5
- 230000002401 inhibitory effect Effects 0.000 claims 5
- 239000000203 mixture Substances 0.000 claims 5
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108010006025 bovine growth hormone Proteins 0.000 claims 3
- 239000003623 enhancer Substances 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 241000701447 unidentified baculovirus Species 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- 241000238631 Hexapoda Species 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 230000002093 peripheral effect Effects 0.000 claims 2
- 230000001124 posttranscriptional effect Effects 0.000 claims 2
- 230000001105 regulatory effect Effects 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 239000001963 growth medium Substances 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 230000005764 inhibitory process Effects 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024001041A JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762567303P | 2017-10-03 | 2017-10-03 | |
| US201762567305P | 2017-10-03 | 2017-10-03 | |
| US62/567,305 | 2017-10-03 | ||
| US62/567,303 | 2017-10-03 | ||
| JP2020540681A JP2020537542A (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
| PCT/US2018/054223 WO2019070891A1 (en) | 2017-10-03 | 2018-10-03 | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540681A Division JP2020537542A (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024001041A Division JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022060228A JP2022060228A (ja) | 2022-04-14 |
| JP2022060228A5 true JP2022060228A5 (enExample) | 2022-04-26 |
Family
ID=65995011
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540681A Pending JP2020537542A (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
| JP2022007759A Pending JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A Pending JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A Pending JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540681A Pending JP2020537542A (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023192753A Pending JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A Pending JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11807849B2 (enExample) |
| EP (1) | EP3692151A4 (enExample) |
| JP (4) | JP2020537542A (enExample) |
| KR (2) | KR20200078513A (enExample) |
| CN (2) | CN111465691A (enExample) |
| AU (3) | AU2018346102B2 (enExample) |
| BR (1) | BR112020006671A2 (enExample) |
| CA (1) | CA3078501A1 (enExample) |
| IL (2) | IL273768A (enExample) |
| MX (2) | MX2020004005A (enExample) |
| WO (1) | WO2019070891A1 (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7361037B2 (ja) | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| US11802294B2 (en) | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20200078513A (ko) | 2017-10-03 | 2020-07-01 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| CA3128875A1 (en) | 2019-02-04 | 2020-08-13 | Freeline Therapeutics Limited | Polynucleotides |
| SG11202108504YA (en) | 2019-02-22 | 2021-09-29 | Univ Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| BR112021019880A2 (pt) | 2019-04-10 | 2022-02-15 | Prevail Therapeutics Inc | Terapias gênicas para distúrbios lisossomais |
| 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 |
| EP4013870A1 (en) * | 2019-08-13 | 2022-06-22 | Alnylam Pharmaceuticals, Inc. | Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof |
| CN116157527A (zh) | 2020-08-10 | 2023-05-23 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
| US20230346979A1 (en) | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| WO2022081985A1 (en) | 2020-10-15 | 2022-04-21 | Prevail Therapeutics, Inc. | Assay for measuring potency of gene therapy drug product |
| CN116710567A (zh) | 2020-10-15 | 2023-09-05 | 普利维尔治疗公司 | 重组腺相关病毒组合物及其产生方法 |
| JPWO2022097727A1 (enExample) * | 2020-11-06 | 2022-05-12 | ||
| EP4359525A1 (en) * | 2021-06-21 | 2024-05-01 | uniQure biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| CA3223506A1 (en) * | 2021-06-28 | 2023-01-05 | Rachel M. Bailey | Methods and compositions for tau reduction gene therapy |
| CN113846122B (zh) * | 2021-08-16 | 2023-08-25 | 中国医学科学院医学生物学研究所 | 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用 |
| EP4493703A2 (en) * | 2022-03-18 | 2025-01-22 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating tmem43 related cardiomyopathy with a viral vector |
| WO2025077806A1 (en) * | 2023-10-11 | 2025-04-17 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of microtubule associated protein tau (mapt) |
| WO2025160429A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
Family Cites Families (74)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5879680A (en) | 1987-12-23 | 1999-03-09 | The United States Of America As Represented By The Department Of Health And Human Services | Cloned DNA for synthesizing unique glucocerebrosidase |
| US6521225B1 (en) * | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| CA2341325A1 (en) | 1998-09-09 | 2000-03-16 | Myelos Corporation | Method of stimulating prosaposin receptor activity |
| EP1121429A2 (en) | 1998-10-16 | 2001-08-08 | Introgene B.V. | Gene therapy of alzheimer's disease by delivery of an encoded apolipoprotein e |
| US6491907B1 (en) | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
| US6696272B1 (en) | 1999-06-02 | 2004-02-24 | Hsc Research & Development Limited Partnership | Products and methods for gaucher disease therapy |
| WO2001077136A1 (en) | 2000-04-06 | 2001-10-18 | Kos Pharmaceuticals, Inc. | Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia |
| GB0009887D0 (en) * | 2000-04-20 | 2000-06-07 | Btg Int Ltd | Cytotoxic agents |
| JP2004514407A (ja) * | 2000-04-28 | 2004-05-20 | 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 | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| DE60115070T2 (de) | 2000-09-18 | 2006-07-27 | Genzyme Corp., Cambridge | Expressionsvektoren mit hybriden ubiquitin-promotoren |
| US7232670B2 (en) | 2001-09-28 | 2007-06-19 | St. Jude Children's Research Hospital | Targeting proteins to cells expressing mannose receptors via expression in insect cells |
| ATE317916T1 (de) * | 2001-11-13 | 2006-03-15 | Univ Pennsylvania | Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode |
| US20030133924A1 (en) | 2001-12-21 | 2003-07-17 | Novazyme Pharmaceuticals, Inc. | Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease |
| US20060292117A1 (en) | 2002-04-17 | 2006-12-28 | Loiler Scott A | Improved rAAv vectors |
| PL1620133T3 (pl) | 2003-05-01 | 2016-05-31 | Genzyme Corp | Terapia genowa dla zaburzeń neurometabolicznych |
| WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
| DE602005015994D1 (de) | 2004-09-29 | 2009-09-24 | Childrens Memorial Hospital | siRNA-VERMITTELTES GEN-SILENCING VON ALPHA-SYNUKLEIN |
| AU2006254796B2 (en) | 2005-06-08 | 2012-08-16 | Amicus Therapeutics, Inc. | Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes |
| GB0605337D0 (en) * | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
| EP2687223B2 (en) | 2006-05-30 | 2024-11-20 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| DK2687223T4 (da) | 2006-05-30 | 2025-02-17 | Mayo Found Medical Education & Res | Påvisning og behandling af demens |
| CA2654292C (en) | 2006-06-07 | 2022-01-11 | Genzyme Corporation | Gene therapy for motor neuron disorders |
| EP2578692B1 (en) | 2007-04-05 | 2016-06-08 | The J. David Gladstone Institutes | Agents that reduce neuronal overexcitation |
| US8962273B2 (en) | 2007-05-11 | 2015-02-24 | Genzyme Corporation | Methods of producing a secreted protein |
| PL2154969T3 (pl) | 2007-05-16 | 2016-04-29 | Brigham & Womens Hospital Inc | Leczenie synukleinopatii |
| WO2009079399A2 (en) * | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| EP3009143B1 (en) | 2008-01-16 | 2018-10-03 | Neurodyn Life Sciences Inc. | Progranulin for use in treating parkinson's disease or alzheimer's disease |
| US9265843B2 (en) | 2008-03-27 | 2016-02-23 | The Ohio State University | Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor |
| HUE072183T2 (hu) | 2009-05-02 | 2025-10-28 | Genzyme Corp | Génterápia neurodegeneratív betegségekre |
| WO2010129791A1 (en) * | 2009-05-06 | 2010-11-11 | University Of Medicine And Dentistry Of New Jersey | Rna targeting in alpha-synucleinopathies |
| DK2826860T3 (en) * | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| WO2012027558A2 (en) * | 2010-08-25 | 2012-03-01 | The Trustees Of Columbia University In The City Of New York | OPTIMIZED miRNA CONSTRUCTS |
| WO2012027713A2 (en) * | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| EP2634253B1 (en) * | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| JP6312436B2 (ja) | 2010-11-16 | 2018-04-18 | ニューロダイン ライフ サイエンシズ インコーポレイテッドＮｅｕｒｏｄｙｎ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｉｎｃ． | ネプリライシンの発現および活性を増大させるための方法および医薬組成物 |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| EP2707369B1 (en) | 2011-05-09 | 2016-07-27 | Eip Pharma, LLC | Compositions and methods for treating alzheimer's disease |
| AU2013243954A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of cosmetic proteins and peptides |
| CN112574964A (zh) | 2012-05-18 | 2021-03-30 | 衣阿华大学研究基金会 | 用于治疗淀粉状蛋白沉积的方法和组合物 |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| MA38144A1 (fr) | 2012-11-05 | 2018-08-31 | Genzyme Corp | Compositions et procédés pour le traitement de protéinopathies |
| EP2983707B1 (en) * | 2013-04-08 | 2019-06-12 | University of Iowa Research Foundation | Chimeric adeno-associated virus/ bocavirus parvovirus vector |
| EP3622821A1 (en) | 2013-05-15 | 2020-03-18 | Regents of the University of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| US9347107B2 (en) | 2013-05-23 | 2016-05-24 | Norman Z Lai | Vector containing multiple nucleotide sequences for the expression of enzymes |
| EP3019175B1 (en) | 2013-07-11 | 2019-12-04 | The Trustees of Columbia University in the City of New York | Micrornas that silence tau expression |
| US9574184B2 (en) | 2013-09-25 | 2017-02-21 | Children's Hospital Medical Center | Lysosomal protein targeting sequence and therapeutic applications of same |
| CN105745326A (zh) | 2013-10-24 | 2016-07-06 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| GB201403260D0 (en) * | 2014-02-25 | 2014-04-09 | Univ Manchester | Treatment of retinal degeneration using gene therapy |
| US10603364B2 (en) | 2014-08-11 | 2020-03-31 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| SG11201703148TA (en) * | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| US10900053B2 (en) | 2014-11-21 | 2021-01-26 | University Of Florida Research Foundation, Incorporated | Genome-modified recombinant adeno-associated virus vectors |
| ES2820713T3 (es) * | 2015-03-25 | 2021-04-22 | Univ Degli Studi Di Trento | Terapia mediada por ARN interferente para enfermedades neurodegenerativas |
| US20170035860A1 (en) | 2015-04-02 | 2017-02-09 | Alexander C. Flynn | Compositions and methods for treatment of neurogenerative diseases |
| WO2016172155A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| EP3091087A1 (en) * | 2015-05-08 | 2016-11-09 | Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol | Method for in vitro diagnosis of synucleinopathies using alpha-synuclein gene transcripts |
| SMT202200022T1 (it) | 2015-05-15 | 2022-03-21 | Regenxbio Inc | Virus adeno-associato per veicolazione terapeutica al sistema nervoso centrale |
| MX2018004755A (es) | 2015-10-29 | 2018-12-19 | Voyager Therapeutics Inc | Entrega de polinucleotidos dirigidos al sistema nervioso central. |
| KR20210005994A (ko) | 2015-11-05 | 2021-01-15 | 뱀부 테라퓨틱스 인코포레이티드 | 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 |
| WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| SI3411484T1 (sl) | 2016-02-05 | 2023-12-29 | Emory University | Injiciranje enojnega ali samokomplementarnega adeno-povezanega virusa 9 v cerebrospinalno tekočino |
| US11066456B2 (en) | 2016-02-25 | 2021-07-20 | Washington University | Compositions comprising TREM2 and methods of use thereof |
| CA3016314A1 (en) | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖを送達するための組成物および方法 |
| US11802294B2 (en) | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20200078513A (ko) | 2017-10-03 | 2020-07-01 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| JP7361037B2 (ja) | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| US20210261981A1 (en) | 2017-10-23 | 2021-08-26 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| MX2021006253A (es) | 2018-11-28 | 2021-09-21 | Prevail Therapeutics Inc | Terapias génicas para enfermedad neurodegenerativa. |
| BR112021020157A2 (pt) | 2019-04-10 | 2021-12-14 | Prevail Therapeutics Inc | Terapias gênicas para transtornos lisossomais |
| BR112021019880A2 (pt) | 2019-04-10 | 2022-02-15 | Prevail Therapeutics Inc | Terapias gênicas para distúrbios lisossomais |
| 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 |
| US20220211871A1 (en) | 2019-04-10 | 2022-07-07 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
-
2018
- 2018-10-03 KR KR1020207012192A patent/KR20200078513A/ko not_active Ceased
- 2018-10-03 WO PCT/US2018/054223 patent/WO2019070891A1/en not_active Ceased
- 2018-10-03 CN CN201880075728.7A patent/CN111465691A/zh active Pending
- 2018-10-03 KR KR1020227000760A patent/KR20220010062A/ko not_active Ceased
- 2018-10-03 US US16/753,018 patent/US11807849B2/en active Active
- 2018-10-03 CA CA3078501A patent/CA3078501A1/en active Pending
- 2018-10-03 EP EP18864256.5A patent/EP3692151A4/en active Pending
- 2018-10-03 AU AU2018346102A patent/AU2018346102B2/en active Active
- 2018-10-03 CN CN202011187145.9A patent/CN112501208A/zh active Pending
- 2018-10-03 JP JP2020540681A patent/JP2020537542A/ja active Pending
- 2018-10-03 BR BR112020006671-0A patent/BR112020006671A2/pt unknown
- 2018-10-03 MX MX2020004005A patent/MX2020004005A/es unknown
-
2020
- 2020-04-02 US US16/838,993 patent/US12049626B2/en active Active
- 2020-04-02 IL IL273768A patent/IL273768A/en unknown
- 2020-07-13 MX MX2020011748A patent/MX2020011748A/es unknown
- 2020-10-29 AU AU2020260476A patent/AU2020260476B2/en active Active
- 2020-12-22 IL IL279669A patent/IL279669A/en unknown
-
2022
- 2022-01-21 JP JP2022007759A patent/JP2022060228A/ja active Pending
-
2023
- 2023-08-11 AU AU2023214366A patent/AU2023214366B2/en active Active
- 2023-11-13 JP JP2023192753A patent/JP2024014935A/ja active Pending
-
2024
- 2024-01-09 JP JP2024001041A patent/JP2024037189A/ja active Pending
- 2024-06-14 US US18/743,624 patent/US20250011783A1/en active Pending
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