JP2019513399A5 - - Google Patents
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- Publication number
- JP2019513399A5 JP2019513399A5 JP2018554055A JP2018554055A JP2019513399A5 JP 2019513399 A5 JP2019513399 A5 JP 2019513399A5 JP 2018554055 A JP2018554055 A JP 2018554055A JP 2018554055 A JP2018554055 A JP 2018554055A JP 2019513399 A5 JP2019513399 A5 JP 2019513399A5
- Authority
- JP
- Japan
- Prior art keywords
- aav vector
- recombinant aav
- composition
- seq
- muscle
- 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.)
- Granted
Links
- 239000013607 AAV vector Substances 0.000 claims 25
- 239000000203 mixture Substances 0.000 claims 18
- 210000003205 muscle Anatomy 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 108091033319 polynucleotide Proteins 0.000 claims 6
- 239000002157 polynucleotide Substances 0.000 claims 6
- 102000040430 polynucleotide Human genes 0.000 claims 6
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims 5
- 201000006938 muscular dystrophy Diseases 0.000 claims 5
- 208000034067 Beta-sarcoglycan-related limb-girdle muscular dystrophy R4 Diseases 0.000 claims 3
- 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
- 108010085238 Actins Proteins 0.000 claims 2
- 206010016654 Fibrosis Diseases 0.000 claims 2
- 108090000362 Lymphotoxin-beta Proteins 0.000 claims 2
- 102000005604 Myosin Heavy Chains Human genes 0.000 claims 2
- 108010084498 Myosin Heavy Chains Proteins 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 108091027981 Response element Proteins 0.000 claims 2
- 208000027418 Wounds and injury Diseases 0.000 claims 2
- 230000008602 contraction Effects 0.000 claims 2
- 230000006378 damage Effects 0.000 claims 2
- 230000004761 fibrosis Effects 0.000 claims 2
- 208000014674 injury Diseases 0.000 claims 2
- 238000010255 intramuscular injection Methods 0.000 claims 2
- 238000010253 intravenous injection Methods 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 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
- 241000300529 Adeno-associated virus 13 Species 0.000 claims 1
- 102000019370 Beta-sarcoglycan Human genes 0.000 claims 1
- 102000004420 Creatine Kinase Human genes 0.000 claims 1
- 108010042126 Creatine kinase Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims 1
- 206010021143 Hypoxia Diseases 0.000 claims 1
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims 1
- 108010083379 Sarcoglycans Proteins 0.000 claims 1
- 108010065729 Troponin I Proteins 0.000 claims 1
- 230000005856 abnormality Effects 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 201000009553 autosomal recessive limb-girdle muscular dystrophy type 2E Diseases 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 239000003862 glucocorticoid Substances 0.000 claims 1
- 230000007954 hypoxia Effects 0.000 claims 1
- 239000007943 implant Substances 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 239000007927 intramuscular injection Substances 0.000 claims 1
- 108091047189 miR-29c stem-loop Proteins 0.000 claims 1
- 108091054490 miR-29c-2 stem-loop Proteins 0.000 claims 1
- 210000000663 muscle cell Anatomy 0.000 claims 1
- 238000007911 parenteral administration Methods 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 208000022561 qualitative or quantitative defects of beta-sarcoglycan Diseases 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 210000002027 skeletal muscle Anatomy 0.000 claims 1
- 150000003431 steroids Chemical class 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022061771A JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323333P | 2016-04-15 | 2016-04-15 | |
| US62/323,333 | 2016-04-15 | ||
| US201662433548P | 2016-12-13 | 2016-12-13 | |
| US62/433,548 | 2016-12-13 | ||
| PCT/US2017/027583 WO2017180976A1 (en) | 2016-04-15 | 2017-04-14 | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022061771A Division JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019513399A JP2019513399A (ja) | 2019-05-30 |
| JP2019513399A5 true JP2019513399A5 (enExample) | 2020-05-14 |
| JP7079733B2 JP7079733B2 (ja) | 2022-06-02 |
Family
ID=60042261
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018554055A Active JP7079733B2 (ja) | 2016-04-15 | 2017-04-14 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
| JP2022061771A Active JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022061771A Active JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Country Status (28)
| Country | Link |
|---|---|
| US (3) | US11358993B2 (enExample) |
| EP (2) | EP4407035A3 (enExample) |
| JP (2) | JP7079733B2 (enExample) |
| KR (1) | KR102502091B1 (enExample) |
| CN (2) | CN117384964A (enExample) |
| AU (2) | AU2017250756B2 (enExample) |
| BR (1) | BR112018071167A2 (enExample) |
| CA (1) | CA3021057A1 (enExample) |
| CO (1) | CO2018012082A2 (enExample) |
| DK (1) | DK3442600T5 (enExample) |
| ES (1) | ES2979174T3 (enExample) |
| FI (1) | FI3442600T3 (enExample) |
| HR (1) | HRP20240580T1 (enExample) |
| HU (1) | HUE066442T2 (enExample) |
| IL (1) | IL262262B2 (enExample) |
| LT (1) | LT3442600T (enExample) |
| MA (1) | MA44681A (enExample) |
| MX (2) | MX391208B (enExample) |
| MY (1) | MY195439A (enExample) |
| PL (1) | PL3442600T3 (enExample) |
| PT (1) | PT3442600T (enExample) |
| RS (1) | RS65490B1 (enExample) |
| SA (1) | SA518400245B1 (enExample) |
| SG (1) | SG11201809029RA (enExample) |
| SI (1) | SI3442600T1 (enExample) |
| SM (1) | SMT202400169T1 (enExample) |
| WO (1) | WO2017180976A1 (enExample) |
| ZA (1) | ZA201806863B (enExample) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| EP3020403A1 (en) | 2014-11-14 | 2016-05-18 | Universitat de Valéncia | Compounds for the treatment of myotonic dystrophy |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| EP3377042A4 (en) | 2015-11-16 | 2019-05-29 | The Research Institute at Nationwide Children's Hospital | MATERIALS AND METHOD FOR THE TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES |
| WO2017095967A2 (en) | 2015-11-30 | 2017-06-08 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| DK3442602T3 (da) * | 2016-04-15 | 2025-05-19 | Res Inst Nationwide Childrens Hospital | Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi |
| EP4407035A3 (en) * | 2016-04-15 | 2024-10-30 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| MA47800A (fr) | 2017-03-17 | 2020-01-22 | Univ Newcastle | Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire |
| IL321310A (en) | 2017-10-20 | 2025-08-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for NT-3 gene therapy |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| BR112020025995A2 (pt) | 2018-06-18 | 2021-03-23 | Research Institute At Nationwide Children's Hospital | administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular |
| WO2020006458A1 (en) * | 2018-06-29 | 2020-01-02 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| EP3598978B1 (en) | 2018-07-26 | 2024-05-29 | EXOFIX S.r.l. | Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles |
| US20220119489A1 (en) * | 2019-01-25 | 2022-04-21 | Children's Medical Center Corporation | Compositions and methods for treating muscular dystrophy and related disorders |
| WO2020160542A1 (en) * | 2019-02-02 | 2020-08-06 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation |
| CA3131390A1 (en) | 2019-02-26 | 2020-09-03 | Research Institute Of Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| EP4488287A3 (en) * | 2019-08-21 | 2025-03-05 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| KR20220083708A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | Aav 전달 카세트 |
| JP7847084B2 (ja) * | 2019-12-16 | 2026-04-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法 |
| CN115996758A (zh) * | 2020-06-19 | 2023-04-21 | 吉尼松公司 | 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统 |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| KR20250044484A (ko) | 2022-06-24 | 2025-03-31 | 튠 쎄라퓨틱스, 인코포레이티드 | 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법 |
| AU2024406119A1 (en) | 2023-12-18 | 2026-04-02 | F. Hoffmann-La Roche Ag | Skeletal muscle-specific promoters |
| CN118045206B (zh) * | 2024-04-12 | 2024-07-05 | 四川至善唯新生物科技有限公司 | 一种治疗脊髓型肌肉萎缩的药物组合物及其用途 |
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| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
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| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
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| US5672694A (en) | 1995-10-24 | 1997-09-30 | University Of Iowa Research Foundation | β-sarcoglycan nucleic acid sequence, and nucleic acid probes |
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| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
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| US7883858B2 (en) | 2005-01-27 | 2011-02-08 | Institute For Systems Biology | Methods for identifying and monitoring drug side effects |
| US20070099251A1 (en) | 2005-10-17 | 2007-05-03 | Institute For Systems Biology | Tissue-and serum-derived glycoproteins and methods of their use |
| JP5244103B2 (ja) | 2006-08-09 | 2013-07-24 | ホームステッド クリニカル コーポレイション | 器官特異的蛋白質およびその使用方法 |
| US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
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| EP2203173B1 (en) | 2007-10-26 | 2015-12-23 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| WO2009137006A2 (en) | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
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| KR102057540B1 (ko) | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| WO2013151663A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| EP2892617B1 (en) * | 2012-09-06 | 2018-06-13 | The University of Chicago | Antisense polynucleotides to induce exon skipping and methods of treating dystrophies |
| US9624282B2 (en) | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| SG11201605906UA (en) | 2014-01-21 | 2016-08-30 | Univ Bruxelles | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| EP4407035A3 (en) * | 2016-04-15 | 2024-10-30 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy |
| BR112022025586A2 (pt) * | 2020-06-15 | 2023-03-07 | Res Inst Nationwide Childrens Hospital | Liberação de vetor de vírus adenoassociado para distrofias musculares |
-
2017
- 2017-04-14 EP EP24163078.9A patent/EP4407035A3/en active Pending
- 2017-04-14 FI FIEP17783207.8T patent/FI3442600T3/fi active
- 2017-04-14 SM SM20240169T patent/SMT202400169T1/it unknown
- 2017-04-14 SG SG11201809029RA patent/SG11201809029RA/en unknown
- 2017-04-14 HU HUE17783207A patent/HUE066442T2/hu unknown
- 2017-04-14 MY MYPI2018001743A patent/MY195439A/en unknown
- 2017-04-14 HR HRP20240580TT patent/HRP20240580T1/hr unknown
- 2017-04-14 JP JP2018554055A patent/JP7079733B2/ja active Active
- 2017-04-14 SI SI201731512T patent/SI3442600T1/sl unknown
- 2017-04-14 CA CA3021057A patent/CA3021057A1/en active Pending
- 2017-04-14 US US16/093,027 patent/US11358993B2/en active Active
- 2017-04-14 RS RS20240504A patent/RS65490B1/sr unknown
- 2017-04-14 EP EP17783207.8A patent/EP3442600B1/en active Active
- 2017-04-14 MA MA044681A patent/MA44681A/fr unknown
- 2017-04-14 LT LTEPPCT/US2017/027583T patent/LT3442600T/lt unknown
- 2017-04-14 MX MX2018012605A patent/MX391208B/es unknown
- 2017-04-14 PT PT177832078T patent/PT3442600T/pt unknown
- 2017-04-14 DK DK17783207.8T patent/DK3442600T5/da active
- 2017-04-14 BR BR112018071167-5A patent/BR112018071167A2/pt active Search and Examination
- 2017-04-14 AU AU2017250756A patent/AU2017250756B2/en active Active
- 2017-04-14 WO PCT/US2017/027583 patent/WO2017180976A1/en not_active Ceased
- 2017-04-14 IL IL262262A patent/IL262262B2/en unknown
- 2017-04-14 ES ES17783207T patent/ES2979174T3/es active Active
- 2017-04-14 CN CN202210977746.2A patent/CN117384964A/zh active Pending
- 2017-04-14 PL PL17783207.8T patent/PL3442600T3/pl unknown
- 2017-04-14 CN CN201780023705.7A patent/CN109121395B/zh active Active
- 2017-06-20 KR KR1020187032688A patent/KR102502091B1/ko active Active
-
2018
- 2018-10-15 ZA ZA2018/06863A patent/ZA201806863B/en unknown
- 2018-10-15 MX MX2022003857A patent/MX2022003857A/es unknown
- 2018-10-15 SA SA518400245A patent/SA518400245B1/ar unknown
- 2018-11-08 CO CONC2018/0012082A patent/CO2018012082A2/es unknown
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2021
- 2021-05-13 AU AU2021203044A patent/AU2021203044B2/en active Active
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2022
- 2022-01-20 US US17/580,228 patent/US20220251156A1/en not_active Abandoned
- 2022-04-01 JP JP2022061771A patent/JP7390426B2/ja active Active
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2023
- 2023-10-06 US US18/482,526 patent/US20240182533A1/en active Pending