AR115416A1 - Moduladores de la expresión de apol1 - Google Patents
Moduladores de la expresión de apol1Info
- Publication number
- AR115416A1 AR115416A1 ARP190101365A ARP190101365A AR115416A1 AR 115416 A1 AR115416 A1 AR 115416A1 AR P190101365 A ARP190101365 A AR P190101365A AR P190101365 A ARP190101365 A AR P190101365A AR 115416 A1 AR115416 A1 AR 115416A1
- Authority
- AR
- Argentina
- Prior art keywords
- segment
- modified oligonucleotide
- linked nucleosides
- cet
- nucleoside
- Prior art date
Links
- 102100030762 Apolipoprotein L1 Human genes 0.000 title abstract 3
- 101100323521 Homo sapiens APOL1 gene Proteins 0.000 title abstract 3
- 239000002777 nucleoside Substances 0.000 abstract 9
- 125000003835 nucleoside group Chemical group 0.000 abstract 5
- 108091034117 Oligonucleotide Proteins 0.000 abstract 4
- 150000003833 nucleoside derivatives Chemical class 0.000 abstract 4
- 150000001875 compounds Chemical class 0.000 abstract 3
- QJGQUHMNIGDVPM-UHFFFAOYSA-N nitrogen group Chemical group [N] QJGQUHMNIGDVPM-UHFFFAOYSA-N 0.000 abstract 3
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 abstract 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 abstract 1
- 229940104302 cytosine Drugs 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 abstract 1
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
- 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
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Urology & Nephrology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Pain & Pain Management (AREA)
- Rheumatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Plural Heterocyclic Compounds (AREA)
Abstract
Las presentes realizaciones proporcionan métodos, compuestos y composiciones útiles para inhibir la expresión de APOL1, lo que puede ser útil para tratar, prevenir o mejorar una enfermedad asociada con APOL1. Reivindicación 1: Un compuesto que comprende un oligonucleótido modificado de 8 a 80 nucleósidos unidos de longitud que tiene una secuencia de bases nitrogenadas que comprende al menos 8, al menos 9, al menos 10, al menos 11 o al menos 12 bases nitrogenadas contiguas de cualquiera de las secuencias de bases nitrogenadas de las SEQ ID Nº 13 - 1941. Reivindicación 30: Un compuesto que consiste en un oligonucleótido modificado, en donde el oligonucleótido modificado tiene 16 nucleósidos unidos de longitud y consiste en la secuencia de SEQ ID Nº 1164, en donde el oligonucleótido modificado consta de un segmento gap constituido por nueve desoxinucleósidos unidos; un segmento lateral 5 constituido por tres nucleósidos unidos; y un segmento lateral 3 constituido por cuatro nucleósidos unidos; en donde el segmento gap está situado entre el segmento lateral 5 y el segmento lateral 3; en donde el segmento lateral 5 consiste en nucleósidos cEt; en donde el segmento lateral 3 consiste en un nucleósido cEt, un nucleósido cEt, un nucleósido cEt y un nucleósido 2-O-metoxietilo en la dirección de 5 a 3; donde cada unión entre nucleósidos es una unión de tipo fosforotioato; y en donde cada citosina es una 5-metilcitosina.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862674865P | 2018-05-22 | 2018-05-22 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR115416A1 true AR115416A1 (es) | 2021-01-13 |
Family
ID=68613945
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190101365A AR115416A1 (es) | 2018-05-22 | 2019-05-22 | Moduladores de la expresión de apol1 |
Country Status (25)
| Country | Link |
|---|---|
| US (3) | US10927377B2 (es) |
| EP (1) | EP3799570A4 (es) |
| JP (2) | JP7247227B2 (es) |
| KR (1) | KR20210011981A (es) |
| CN (3) | CN118127019A (es) |
| AR (1) | AR115416A1 (es) |
| AU (1) | AU2019274461A1 (es) |
| BR (1) | BR112020023436A2 (es) |
| CA (1) | CA3099750A1 (es) |
| CL (1) | CL2020003010A1 (es) |
| CO (1) | CO2020015377A2 (es) |
| CR (1) | CR20200631A (es) |
| EA (1) | EA202092748A1 (es) |
| EC (1) | ECSP20082339A (es) |
| IL (1) | IL278785B1 (es) |
| JO (1) | JOP20200291A1 (es) |
| MA (1) | MA53924A (es) |
| MX (1) | MX2020012497A (es) |
| NI (1) | NI202000085A (es) |
| PE (1) | PE20211397A1 (es) |
| PH (1) | PH12020551995A1 (es) |
| SG (1) | SG11202011397QA (es) |
| TW (2) | TW202430638A (es) |
| WO (1) | WO2019226611A1 (es) |
| ZA (1) | ZA202007537B (es) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE49076E1 (en) | 2010-04-18 | 2022-05-17 | Beth Israel Deaconess Medical Center, Inc. | Compositions and methods for treating renal disease |
| TW202430638A (zh) | 2018-05-22 | 2024-08-01 | 美商Ionis製藥公司 | Apol1表現之調節劑 |
| CN113453760B (zh) | 2018-12-17 | 2024-05-24 | 弗特克斯药品有限公司 | Apol1抑制剂及其使用方法 |
| CN116348447A (zh) | 2020-01-29 | 2023-06-27 | 弗特克斯药品有限公司 | Apol1的抑制剂及其使用其的方法 |
| AU2023208684A1 (en) | 2022-01-18 | 2024-09-05 | Maze Therapeutics, Inc. | Apol1 inhibitors and methods of use |
| WO2024040235A1 (en) * | 2022-08-19 | 2024-02-22 | Maze Therapeutics, Inc. | Apol1 inhibitors and methods of use |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6833361B2 (en) * | 1998-05-26 | 2004-12-21 | Ribapharm, Inc. | Nucleosides having bicyclic sugar moiety |
| JP2010507387A (ja) * | 2006-10-25 | 2010-03-11 | クアーク・ファーマスーティカルス、インコーポレイテッド | 新規のｓｉＲＮＡおよびその使用方法 |
| US9023355B2 (en) | 2010-04-13 | 2015-05-05 | Beth Israel Deaconess Medical Center, Inc. | Compositions and methods for treating renal disease |
| CA2863958A1 (en) | 2012-02-08 | 2013-08-15 | Isis Pharmaceuticals, Inc. | Methods and compositions for modulating factor vii expression |
| US10130632B2 (en) * | 2012-11-27 | 2018-11-20 | Beth Israel Deaconess Medical Center, Inc. | Methods for treating renal disease |
| EP2972381A2 (en) | 2013-03-14 | 2016-01-20 | Galapagos NV | Molecular targets and compounds, and methods to identify the same, useful in the treatment of diseases associated with epithelial mesenchymal transition |
| TW202246503A (zh) | 2013-07-19 | 2022-12-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| TW201718010A (zh) * | 2015-06-19 | 2017-06-01 | 健臻公司 | 治療原發性局部節段性腎小球硬化症之方法 |
| KR20180051626A (ko) | 2015-09-24 | 2018-05-16 | 아이오니스 파마수티컬즈, 인코포레이티드 | Kras 발현의 조절제 |
| TW202430638A (zh) | 2018-05-22 | 2024-08-01 | 美商Ionis製藥公司 | Apol1表現之調節劑 |
-
2019
- 2019-05-21 TW TW113114934A patent/TW202430638A/zh unknown
- 2019-05-21 CN CN202410386618.XA patent/CN118127019A/zh active Pending
- 2019-05-21 CR CR20200631A patent/CR20200631A/es unknown
- 2019-05-21 TW TW108117504A patent/TWI841564B/zh active
- 2019-05-21 JO JOP/2020/0291A patent/JOP20200291A1/ar unknown
- 2019-05-21 WO PCT/US2019/033244 patent/WO2019226611A1/en active Application Filing
- 2019-05-21 IL IL278785A patent/IL278785B1/en unknown
- 2019-05-21 AU AU2019274461A patent/AU2019274461A1/en active Pending
- 2019-05-21 SG SG11202011397QA patent/SG11202011397QA/en unknown
- 2019-05-21 BR BR112020023436-2A patent/BR112020023436A2/pt unknown
- 2019-05-21 EA EA202092748A patent/EA202092748A1/ru unknown
- 2019-05-21 JP JP2020565431A patent/JP7247227B2/ja active Active
- 2019-05-21 CN CN202410386621.1A patent/CN118127020A/zh active Pending
- 2019-05-21 MA MA053924A patent/MA53924A/fr unknown
- 2019-05-21 EP EP19807789.3A patent/EP3799570A4/en active Pending
- 2019-05-21 CA CA3099750A patent/CA3099750A1/en active Pending
- 2019-05-21 KR KR1020207036583A patent/KR20210011981A/ko active Search and Examination
- 2019-05-21 PE PE2020001880A patent/PE20211397A1/es unknown
- 2019-05-21 MX MX2020012497A patent/MX2020012497A/es unknown
- 2019-05-21 CN CN201980033544.9A patent/CN112423792B/zh active Active
- 2019-05-21 US US16/418,060 patent/US10927377B2/en active Active
- 2019-05-22 AR ARP190101365A patent/AR115416A1/es unknown
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2020
- 2020-09-18 US US17/025,239 patent/US11525136B2/en active Active
- 2020-11-18 PH PH12020551995A patent/PH12020551995A1/en unknown
- 2020-11-19 CL CL2020003010A patent/CL2020003010A1/es unknown
- 2020-11-20 NI NI202000085A patent/NI202000085A/es unknown
- 2020-12-03 ZA ZA2020/07537A patent/ZA202007537B/en unknown
- 2020-12-09 CO CONC2020/0015377A patent/CO2020015377A2/es unknown
- 2020-12-18 EC ECSENADI202082339A patent/ECSP20082339A/es unknown
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2022
- 2022-11-08 US US18/053,447 patent/US20230265428A1/en active Pending
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2023
- 2023-03-15 JP JP2023040313A patent/JP2023075283A/ja active Pending
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