JP7820823B2 - 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター - Google Patents
骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクターInfo
- Publication number
- JP7820823B2 JP7820823B2 JP2022564746A JP2022564746A JP7820823B2 JP 7820823 B2 JP7820823 B2 JP 7820823B2 JP 2022564746 A JP2022564746 A JP 2022564746A JP 2022564746 A JP2022564746 A JP 2022564746A JP 7820823 B2 JP7820823 B2 JP 7820823B2
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- C—CHEMISTRY; METALLURGY
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- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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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
- 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
- A61K38/19—Cytokines; Lymphokines; Interferons
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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
- 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
- A61K38/19—Cytokines; Lymphokines; Interferons
- A61K38/193—Colony stimulating factors [CSF]
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
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- 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
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/53—Colony-stimulating factor [CSF]
- C07K14/535—Granulocyte CSF; Granulocyte-macrophage CSF
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
- C07K14/5434—IL-12
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
- C07K14/5443—IL-15
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
- C07K14/55—IL-2
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/555—Interferons [IFN]
- C07K14/56—IFN-alpha
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/555—Interferons [IFN]
- C07K14/57—IFN-gamma
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Toxicology (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Psychiatry (AREA)
- Developmental Biology & Embryology (AREA)
- Cell Biology (AREA)
- Hematology (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025239487A JP2026062601A (ja) | 2020-05-27 | 2025-12-08 | 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20176939 | 2020-05-27 | ||
| EP20176939.5 | 2020-05-27 | ||
| PCT/EP2021/059070 WO2021239308A1 (en) | 2020-05-27 | 2021-04-07 | Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025239487A Division JP2026062601A (ja) | 2020-05-27 | 2025-12-08 | 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023526773A JP2023526773A (ja) | 2023-06-23 |
| JP2023526773A5 JP2023526773A5 (https=) | 2024-04-01 |
| JP7820823B2 true JP7820823B2 (ja) | 2026-02-26 |
Family
ID=70918223
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022564746A Active JP7820823B2 (ja) | 2020-05-27 | 2021-04-07 | 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター |
| JP2025239487A Pending JP2026062601A (ja) | 2020-05-27 | 2025-12-08 | 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025239487A Pending JP2026062601A (ja) | 2020-05-27 | 2025-12-08 | 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230227847A1 (https=) |
| EP (1) | EP4158035A1 (https=) |
| JP (2) | JP7820823B2 (https=) |
| KR (1) | KR20230018378A (https=) |
| CN (1) | CN115552019A (https=) |
| AU (1) | AU2021280943A1 (https=) |
| BR (1) | BR112022016715A2 (https=) |
| CA (1) | CA3165462A1 (https=) |
| IL (1) | IL298093A (https=) |
| MX (1) | MX2022014766A (https=) |
| WO (1) | WO2021239308A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US12071633B2 (en) | 2020-10-13 | 2024-08-27 | Kriya Therapeutics, Inc. | Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| WO2023109911A1 (en) * | 2021-12-15 | 2023-06-22 | National Institute Of Biological Sciences, Beijing | Microglia having car and use thereof |
| WO2024036106A1 (en) * | 2022-08-08 | 2024-02-15 | The Board Of Trustees Of The Leland Stanford Junior University | Embryo microglia complementation for in vivo microglia manipulation and production of a non-human animal model for validation of gene function and therapeutic screening |
Citations (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001513508A (ja) | 1997-08-12 | 2001-09-04 | ジョージタウン ユニバーシティー | 腫瘍治療を目的とするヘルペスベクターの使用 |
| US20110223635A1 (en) | 2008-08-11 | 2011-09-15 | Karl Deisseroth | Method and Composition for Controlling Gene Expression |
| JP2017525364A (ja) | 2014-08-18 | 2017-09-07 | アプセト ゲーエムベーハー アンド ツェーオー.カーゲー | 免疫細胞を惹起及び/又は活性化するための免疫応答刺激性サイトカインを発現する遺伝子改変型間葉系幹細胞 |
| WO2019021233A1 (en) | 2017-07-27 | 2019-01-31 | Novartis Ag | TREM2 VARIANTS RESISTANT TO SHEDDASE |
| JP2019511570A (ja) | 2016-03-02 | 2019-04-25 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 前頭側頭型認知症の治療 |
| WO2019173756A1 (en) | 2018-03-09 | 2019-09-12 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| US20190300870A1 (en) | 2018-04-03 | 2019-10-03 | The Broad Institute, Inc. | Target recognition motifs and uses thereof |
| WO2020160458A1 (en) | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20180186855A1 (en) * | 2016-03-23 | 2018-07-05 | Alector Llc | Chimeric receptors and methods of use thereof |
| WO2018169901A1 (en) * | 2017-03-13 | 2018-09-20 | Massachusetts Institute Of Technology | Synthetic promoters |
| CN109868286B (zh) * | 2017-12-05 | 2022-09-16 | 深圳先进技术研究院 | 一种慢病毒载体、慢病毒及其制备方法和应用 |
| MX2022003934A (es) * | 2019-10-02 | 2022-04-25 | Univ Zuerich | Tratamiento de la enfermedad granulomatosa cronica. |
-
2021
- 2021-04-07 US US17/998,587 patent/US20230227847A1/en active Pending
- 2021-04-07 BR BR112022016715A patent/BR112022016715A2/pt unknown
- 2021-04-07 MX MX2022014766A patent/MX2022014766A/es unknown
- 2021-04-07 KR KR1020227041496A patent/KR20230018378A/ko active Pending
- 2021-04-07 CA CA3165462A patent/CA3165462A1/en active Pending
- 2021-04-07 IL IL298093A patent/IL298093A/en unknown
- 2021-04-07 CN CN202180034310.3A patent/CN115552019A/zh active Pending
- 2021-04-07 JP JP2022564746A patent/JP7820823B2/ja active Active
- 2021-04-07 AU AU2021280943A patent/AU2021280943A1/en active Pending
- 2021-04-07 WO PCT/EP2021/059070 patent/WO2021239308A1/en not_active Ceased
- 2021-04-07 EP EP21716740.2A patent/EP4158035A1/en active Pending
-
2025
- 2025-12-08 JP JP2025239487A patent/JP2026062601A/ja active Pending
Patent Citations (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001513508A (ja) | 1997-08-12 | 2001-09-04 | ジョージタウン ユニバーシティー | 腫瘍治療を目的とするヘルペスベクターの使用 |
| US20110223635A1 (en) | 2008-08-11 | 2011-09-15 | Karl Deisseroth | Method and Composition for Controlling Gene Expression |
| JP2017525364A (ja) | 2014-08-18 | 2017-09-07 | アプセト ゲーエムベーハー アンド ツェーオー.カーゲー | 免疫細胞を惹起及び/又は活性化するための免疫応答刺激性サイトカインを発現する遺伝子改変型間葉系幹細胞 |
| JP2019511570A (ja) | 2016-03-02 | 2019-04-25 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 前頭側頭型認知症の治療 |
| WO2019021233A1 (en) | 2017-07-27 | 2019-01-31 | Novartis Ag | TREM2 VARIANTS RESISTANT TO SHEDDASE |
| WO2019173756A1 (en) | 2018-03-09 | 2019-09-12 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| US20190300870A1 (en) | 2018-04-03 | 2019-10-03 | The Broad Institute, Inc. | Target recognition motifs and uses thereof |
| WO2020160458A1 (en) | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
Non-Patent Citations (1)
| Title |
|---|
| HUMAN GENE THERAPY METHODS,2013年,Vol.24,pp.151-159 |
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230018378A (ko) | 2023-02-07 |
| JP2023526773A (ja) | 2023-06-23 |
| EP4158035A1 (en) | 2023-04-05 |
| CN115552019A (zh) | 2022-12-30 |
| US20230227847A1 (en) | 2023-07-20 |
| BR112022016715A2 (pt) | 2022-11-16 |
| JP2026062601A (ja) | 2026-04-09 |
| CA3165462A1 (en) | 2021-12-02 |
| AU2021280943A1 (en) | 2022-07-28 |
| IL298093A (en) | 2023-01-01 |
| MX2022014766A (es) | 2023-02-22 |
| WO2021239308A1 (en) | 2021-12-02 |
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