TWI921322B - 用於激肽釋放酶(klkb1)基因編輯之組合物及方法 - Google Patents
用於激肽釋放酶(klkb1)基因編輯之組合物及方法Info
- Publication number
- TWI921322B TWI921322B TW110104494A TW110104494A TWI921322B TW I921322 B TWI921322 B TW I921322B TW 110104494 A TW110104494 A TW 110104494A TW 110104494 A TW110104494 A TW 110104494A TW I921322 B TWI921322 B TW I921322B
- Authority
- TW
- Taiwan
- Prior art keywords
- seq
- composition
- sequence
- rna
- guide
- Prior art date
Links
Classifications
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Fodder In General (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062971906P | 2020-02-07 | 2020-02-07 | |
| US62/971,906 | 2020-02-07 | ||
| US202062981965P | 2020-02-26 | 2020-02-26 | |
| US62/981,965 | 2020-02-26 | ||
| US202063019076P | 2020-05-01 | 2020-05-01 | |
| US63/019,076 | 2020-05-01 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| TW202146030A TW202146030A (zh) | 2021-12-16 |
| TWI921322B true TWI921322B (zh) | 2026-04-11 |
Family
ID=74873798
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW110104494A TWI921322B (zh) | 2020-02-07 | 2021-02-05 | 用於激肽釋放酶(klkb1)基因編輯之組合物及方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (5) | US20230295587A1 (enExample) |
| EP (1) | EP4100528A1 (enExample) |
| JP (1) | JP7883437B2 (enExample) |
| KR (1) | KR20220139352A (enExample) |
| CN (1) | CN115362256A (enExample) |
| AU (1) | AU2021216451A1 (enExample) |
| BR (1) | BR112022015380A2 (enExample) |
| CA (1) | CA3169813A1 (enExample) |
| CL (1) | CL2022002095A1 (enExample) |
| CO (1) | CO2022012196A2 (enExample) |
| IL (1) | IL295280A (enExample) |
| MX (1) | MX2022009658A (enExample) |
| PH (1) | PH12022552009A1 (enExample) |
| SA (1) | SA522440054B1 (enExample) |
| TW (1) | TWI921322B (enExample) |
| WO (1) | WO2021158858A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115362256A (zh) | 2020-02-07 | 2022-11-18 | 因特利亚治疗公司 | 用于激肽释放酶（klkb1）基因编辑的组合物和方法 |
| AU2022357561A1 (en) | 2021-10-01 | 2024-04-18 | Adarx Pharmaceuticals, Inc. | Prekallikrein-modulating compositions and methods of use thereof |
| WO2023086953A1 (en) * | 2021-11-11 | 2023-05-19 | Beam Therapeutics Inc. | Compositions and methods for the treatment of hereditary angioedema (hae) |
| TW202346586A (zh) * | 2022-01-28 | 2023-12-01 | 大陸商上海舶望製藥有限公司 | 用於抑制前激肽釋放酶（pkk）蛋白表達的組合物和方法 |
| AU2023304427A1 (en) * | 2022-07-08 | 2025-01-30 | Intellia Therapeutics, Inc. | Methods for in vivo editing of klkb1 |
| WO2024012435A1 (en) * | 2022-07-11 | 2024-01-18 | CorrectSequence Therapeutics Co., Ltd | Gene editing systems and methods for treating hereditary angioedema |
| CN122095094A (zh) * | 2023-11-02 | 2026-05-26 | 波西达治疗公司 | 靶向klkb1的组合物及其使用方法 |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| CN118303422B (zh) * | 2024-04-17 | 2024-11-12 | 华南师范大学 | Ppk基因在控制德国小蠊繁殖行为中的应用 |
| WO2026008078A1 (en) * | 2024-07-05 | 2026-01-08 | Shanghai Vitalgen Biopharma Co., Ltd. | Methods for kallikrein (klkb1) gene editing |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20170002359A1 (en) * | 2013-08-28 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modulation of prekallikrein (pkk) expression |
| WO2019237069A1 (en) * | 2018-06-08 | 2019-12-12 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
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| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| ATE515510T1 (de) | 1991-12-24 | 2011-07-15 | Isis Pharmaceuticals Inc | Durch dna-abschnitte unterbrochene modifizierte oligonukleotide |
| EP0760008A1 (en) | 1994-05-19 | 1997-03-05 | Dako A/S | Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| WO2012170945A2 (en) * | 2011-06-10 | 2012-12-13 | Isis Pharmaceuticals, Inc. | Methods for modulating kallikrein (klkb1) expression |
| AU2012275096B2 (en) | 2011-06-29 | 2016-02-04 | Ionis Pharmaceuticals, Inc. | Methods for modulating kallikrein (KLKB1) expression |
| US20150141497A1 (en) | 2012-06-15 | 2015-05-21 | Joslin Diabetes Center, Inc. | Methods for modulating kallikrein (klkb1) expression |
| PT2931898E (pt) | 2012-12-12 | 2016-06-16 | Harvard College | Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| IL308158A (en) | 2012-12-17 | 2023-12-01 | Harvard College | Rna-guided human genome engineering |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| US10059655B2 (en) * | 2013-12-19 | 2018-08-28 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| HUE052709T2 (hu) | 2014-05-01 | 2021-05-28 | Ionis Pharmaceuticals Inc | Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására |
| WO2015200555A2 (en) | 2014-06-25 | 2015-12-30 | Caribou Biosciences, Inc. | Rna modification to engineer cas9 activity |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| GB201421083D0 (en) | 2014-11-27 | 2015-01-14 | Kalvista Pharmaceuticals Ltd | Enzyme inhibitors |
| AU2016226077B2 (en) | 2015-03-03 | 2021-12-23 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| CA2984636A1 (en) * | 2015-05-06 | 2016-11-10 | Alnylam Pharmaceuticals, Inc. | Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof |
| FI4019022T3 (fi) | 2015-10-01 | 2024-04-19 | Biocryst Pharm Inc | Ihmisen plasman kallikreiinin estäjiä |
| AU2017212713B2 (en) | 2016-01-27 | 2023-01-19 | Oncorus, Inc. | Oncolytic viral vectors and uses thereof |
| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| WO2017153936A1 (en) | 2016-03-10 | 2017-09-14 | Novartis Ag | Chemically modified messenger rna's |
| US20180112234A9 (en) * | 2016-03-14 | 2018-04-26 | Intellia Therapeutics, Inc. | Methods and compositions for gene editing |
| ES3030658T3 (en) | 2016-03-30 | 2025-07-01 | Intellia Therapeutics Inc | Lipid nanoparticle formulations for crispr/cas components |
| JP7256739B2 (ja) * | 2016-09-07 | 2023-04-12 | サンガモ セラピューティクス， インコーポレイテッド | 肝臓遺伝子のモジュレーション |
| EP3551757A1 (en) | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
| EP3649243A1 (en) * | 2017-07-07 | 2020-05-13 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing contact-activation pathway-associated diseases using irna compositions targeting factor xii (hageman factor) (f12) |
| MX2020000993A (es) | 2017-07-26 | 2020-07-22 | Oncorus Inc | Vectores virales oncolíticos y usos de estos. |
| EP3688162B1 (en) | 2017-09-29 | 2024-03-06 | Intellia Therapeutics, Inc. | Formulations |
| IL311278B2 (en) | 2017-09-29 | 2026-05-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing |
| KR102327512B1 (ko) | 2018-01-12 | 2021-11-17 | 주식회사 큐로셀 | 이원 shrna를 이용한 향상된 면역 세포 및 이를 포함하는 조성물 |
| WO2021028645A1 (en) | 2019-08-09 | 2021-02-18 | Kalvista Pharmaceuticals Limited | Plasma kallikrein inhibitors |
| US12521451B2 (en) * | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| CN115362256A (zh) | 2020-02-07 | 2022-11-18 | 因特利亚治疗公司 | 用于激肽释放酶（klkb1）基因编辑的组合物和方法 |
-
2021
- 2021-02-05 CN CN202180026687.4A patent/CN115362256A/zh active Pending
- 2021-02-05 CA CA3169813A patent/CA3169813A1/en active Pending
- 2021-02-05 MX MX2022009658A patent/MX2022009658A/es unknown
- 2021-02-05 IL IL295280A patent/IL295280A/en unknown
- 2021-02-05 AU AU2021216451A patent/AU2021216451A1/en active Pending
- 2021-02-05 KR KR1020227030659A patent/KR20220139352A/ko active Pending
- 2021-02-05 PH PH1/2022/552009A patent/PH12022552009A1/en unknown
- 2021-02-05 BR BR112022015380A patent/BR112022015380A2/pt unknown
- 2021-02-05 JP JP2022547680A patent/JP7883437B2/ja active Active
- 2021-02-05 TW TW110104494A patent/TWI921322B/zh active
- 2021-02-05 WO PCT/US2021/016730 patent/WO2021158858A1/en not_active Ceased
- 2021-02-05 EP EP21711973.4A patent/EP4100528A1/en active Pending
-
2022
- 2022-08-03 CL CL2022002095A patent/CL2022002095A1/es unknown
- 2022-08-04 SA SA522440054A patent/SA522440054B1/ar unknown
- 2022-08-05 US US17/882,099 patent/US20230295587A1/en not_active Abandoned
- 2022-08-26 CO CONC2022/0012196A patent/CO2022012196A2/es unknown
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2023
- 2023-07-31 US US18/362,675 patent/US20240018496A1/en not_active Abandoned
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2025
- 2025-01-17 US US19/028,692 patent/US12480109B2/en active Active
- 2025-09-12 US US19/327,529 patent/US20260125661A1/en active Pending
- 2025-09-17 US US19/331,096 patent/US12674151B2/en active Active
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20170002359A1 (en) * | 2013-08-28 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modulation of prekallikrein (pkk) expression |
| WO2019237069A1 (en) * | 2018-06-08 | 2019-12-12 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
Non-Patent Citations (1)
| Title |
|---|
| 網路文獻 Anonymous, " Intellia Therapeutics Highlights Recent Progress and Anticipated 2020 Milestones" Intellia Therapeutics, Inc. January 9, 2020 * |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112022015380A2 (pt) | 2022-09-27 |
| IL295280A (en) | 2022-10-01 |
| AU2021216451A1 (en) | 2022-09-29 |
| US20230295587A1 (en) | 2023-09-21 |
| US12674151B2 (en) | 2026-07-07 |
| PH12022552009A1 (en) | 2024-02-05 |
| JP2023512758A (ja) | 2023-03-29 |
| MX2022009658A (es) | 2022-10-27 |
| US20260022358A1 (en) | 2026-01-22 |
| SA522440054B1 (ar) | 2025-04-21 |
| US12480109B2 (en) | 2025-11-25 |
| JP7883437B2 (ja) | 2026-07-01 |
| WO2021158858A1 (en) | 2021-08-12 |
| US20260125661A1 (en) | 2026-05-07 |
| US20250197828A1 (en) | 2025-06-19 |
| US20240018496A1 (en) | 2024-01-18 |
| CA3169813A1 (en) | 2021-08-12 |
| CL2022002095A1 (es) | 2023-03-10 |
| KR20220139352A (ko) | 2022-10-14 |
| TW202146030A (zh) | 2021-12-16 |
| CN115362256A (zh) | 2022-11-18 |
| EP4100528A1 (en) | 2022-12-14 |
| CO2022012196A2 (es) | 2022-09-09 |
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