CN115836129A - 用于治疗tdp-43蛋白病的组合物和方法 - Google Patents
用于治疗tdp-43蛋白病的组合物和方法 Download PDFInfo
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- CN115836129A CN115836129A CN202180045995.1A CN202180045995A CN115836129A CN 115836129 A CN115836129 A CN 115836129A CN 202180045995 A CN202180045995 A CN 202180045995A CN 115836129 A CN115836129 A CN 115836129A
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- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
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- C07—ORGANIC CHEMISTRY
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- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
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- 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
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- 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
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- 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
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- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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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
- C12N15/62—DNA sequences coding for fusion proteins
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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
- C12N15/63—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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/35—Fusion polypeptide containing a fusion for enhanced stability/folding during expression, e.g. fusions with chaperones or thioredoxin
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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
- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Neurology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Neurosurgery (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Immunology (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063016707P | 2020-04-28 | 2020-04-28 | |
| US63/016707 | 2020-04-28 | ||
| US202063035437P | 2020-06-05 | 2020-06-05 | |
| US63/035437 | 2020-06-05 | ||
| PCT/US2021/029289 WO2021222168A2 (en) | 2020-04-28 | 2021-04-27 | Compositions and methods for the treatment of tdp-43 proteinopathies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN115836129A true CN115836129A (zh) | 2023-03-21 |
Family
ID=75905076
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202180045995.1A Pending CN115836129A (zh) | 2020-04-28 | 2021-04-27 | 用于治疗tdp-43蛋白病的组合物和方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240327477A1 (https=) |
| EP (1) | EP4143215A2 (https=) |
| JP (1) | JP2023524414A (https=) |
| KR (1) | KR20230025659A (https=) |
| CN (1) | CN115836129A (https=) |
| AU (1) | AU2021265088A1 (https=) |
| BR (1) | BR112022021604A2 (https=) |
| MX (1) | MX2022013288A (https=) |
| TW (1) | TW202206446A (https=) |
| WO (1) | WO2021222168A2 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2023548632A (ja) * | 2020-10-26 | 2023-11-17 | ソラ・バイオサイエンシズ・エルエルシー | アルツハイマー病の処置のための組成物および方法 |
| WO2023147473A2 (en) * | 2022-01-27 | 2023-08-03 | The Trustees Of Columbia University In The City Of New York | Systems, methods, and compositions for rescuing protein misfolding |
| WO2023185017A1 (zh) * | 2022-03-30 | 2023-10-05 | 呈诺再生医学科技(珠海横琴新区)有限公司 | 经修饰的rnf112或包含其的外泌体递送系统 |
| CN114874332B (zh) * | 2022-03-30 | 2023-01-10 | 呈诺再生医学科技(珠海横琴新区)有限公司 | 经修饰的rnf112作为治疗als药物的应用 |
| AR133834A1 (es) * | 2023-09-15 | 2025-11-05 | Prothena Biosciences Ltd | Agentes de penetración célular y sus usos |
| WO2025264861A1 (en) * | 2024-06-18 | 2025-12-26 | Yale University | Compositions of bi-functional alpha helical peptides and methods thereof for treating tdp-43 proteinopathies |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010111587A1 (en) * | 2009-03-26 | 2010-09-30 | Trustees Of The University Of Pennsylvania | Modulators of tdp-43 mediated toxicity |
| WO2014038884A1 (ko) * | 2012-09-07 | 2014-03-13 | 주식회사 마크로젠 | 융합 단백질 및 이를 포함하는 암 진단용 조성물 |
| WO2014089375A1 (en) * | 2012-12-05 | 2014-06-12 | Boston Strategics Corporation | Protein expression enhancing polypeptides |
| WO2016086320A1 (en) * | 2014-12-05 | 2016-06-09 | UNIVERSITé LAVAL | Tdp-43-binding polypeptides useful for the treatment of neurodegenerative diseases |
| CN106163540A (zh) * | 2013-09-13 | 2016-11-23 | 安达卢西亚进步与健康公共基金会 | 用于治疗蛋白病或构象病的聚集蛋白和分子伴侣的组合 |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB8918616D0 (en) | 1989-08-15 | 1989-09-27 | Univ Glasgow | Herpes simplex virus type 1 mutant |
| US5804413A (en) | 1992-07-31 | 1998-09-08 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Herpes simplex virus strains for gene transfer |
| GB9415319D0 (en) | 1994-07-29 | 1994-09-21 | Medical Res Council | HSV viral vector |
| US5846782A (en) | 1995-11-28 | 1998-12-08 | Genvec, Inc. | Targeting adenovirus with use of constrained peptide motifs |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| AU8605598A (en) | 1997-07-31 | 1999-02-22 | University Of Pittsburgh | Targeted hsv vectors |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| JP5136766B2 (ja) | 2004-12-15 | 2013-02-06 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | キメラベクター |
| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| US9169492B2 (en) | 2010-02-05 | 2015-10-27 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| US9587014B2 (en) | 2011-10-28 | 2017-03-07 | Biogen International Neuroscience Gmbh | TDP-43 specific binding molecules |
| WO2014053879A1 (en) | 2012-10-04 | 2014-04-10 | Centre National De La Recherche Scientifique | Cell penetrating peptides for intracellular delivery of molecules |
| WO2016053610A1 (en) | 2014-10-03 | 2016-04-07 | Academia Sinica | Antibodies against pathological forms of tdp-43 and uses thereof |
| WO2018218252A1 (en) | 2017-05-26 | 2018-11-29 | California Institute Of Technology | Method and apparatus for dynamic rf lens focusing and tracking of wireless power recovery unit |
| AU2019205090A1 (en) | 2018-01-05 | 2020-08-06 | Ac Immune Sa | Misfolded TDP-43 binding molecules |
| EP3770262B1 (en) | 2018-03-16 | 2026-01-14 | National University Corporation Shiga University of Medical Science | Antibody fragment degrading and removing abnormal tdp-43 |
-
2021
- 2021-04-27 WO PCT/US2021/029289 patent/WO2021222168A2/en not_active Ceased
- 2021-04-27 AU AU2021265088A patent/AU2021265088A1/en active Pending
- 2021-04-27 CN CN202180045995.1A patent/CN115836129A/zh active Pending
- 2021-04-27 BR BR112022021604A patent/BR112022021604A2/pt unknown
- 2021-04-27 JP JP2022565544A patent/JP2023524414A/ja active Pending
- 2021-04-27 US US17/997,142 patent/US20240327477A1/en active Pending
- 2021-04-27 KR KR1020227041465A patent/KR20230025659A/ko active Pending
- 2021-04-27 EP EP21725359.0A patent/EP4143215A2/en active Pending
- 2021-04-27 MX MX2022013288A patent/MX2022013288A/es unknown
- 2021-04-28 TW TW110115416A patent/TW202206446A/zh unknown
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010111587A1 (en) * | 2009-03-26 | 2010-09-30 | Trustees Of The University Of Pennsylvania | Modulators of tdp-43 mediated toxicity |
| WO2014038884A1 (ko) * | 2012-09-07 | 2014-03-13 | 주식회사 마크로젠 | 융합 단백질 및 이를 포함하는 암 진단용 조성물 |
| WO2014089375A1 (en) * | 2012-12-05 | 2014-06-12 | Boston Strategics Corporation | Protein expression enhancing polypeptides |
| CN106163540A (zh) * | 2013-09-13 | 2016-11-23 | 安达卢西亚进步与健康公共基金会 | 用于治疗蛋白病或构象病的聚集蛋白和分子伴侣的组合 |
| WO2016086320A1 (en) * | 2014-12-05 | 2016-06-09 | UNIVERSITé LAVAL | Tdp-43-binding polypeptides useful for the treatment of neurodegenerative diseases |
Non-Patent Citations (3)
| Title |
|---|
| HAN-JOU CHEN等: "The heat shock response plays an important role in TDP-43 clearance: evidence for dysfunction in amyotrophic lateral sclerosis", BRAIN, vol. 139, no. 5, 31 May 2016 (2016-05-31), pages 1417 * |
| HISHIYA AKINORI等: "Artificial DnaJ Protein for protein production and conformational diseases", SCIENTIFIC REPORTS, vol. 7, no. 1, 17 August 2017 (2017-08-17), pages 055841518 * |
| 岳宏伟;胡红雨;: "多聚谷氨酰胺延伸蛋白募集细胞内正常蛋白质或RNA的分子机制", 生物化学与生物物理进展, no. 12, 20 December 2018 (2018-12-20), pages 13 - 22 * |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023524414A (ja) | 2023-06-12 |
| KR20230025659A (ko) | 2023-02-22 |
| EP4143215A2 (en) | 2023-03-08 |
| AU2021265088A1 (en) | 2022-11-03 |
| WO2021222168A3 (en) | 2021-12-30 |
| MX2022013288A (es) | 2023-02-22 |
| BR112022021604A2 (pt) | 2022-12-06 |
| TW202206446A (zh) | 2022-02-16 |
| US20240327477A1 (en) | 2024-10-03 |
| WO2021222168A2 (en) | 2021-11-04 |
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