CL2020002401A1 - Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas. - Google Patents
Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas.Info
- Publication number
- CL2020002401A1 CL2020002401A1 CL2020002401A CL2020002401A CL2020002401A1 CL 2020002401 A1 CL2020002401 A1 CL 2020002401A1 CL 2020002401 A CL2020002401 A CL 2020002401A CL 2020002401 A CL2020002401 A CL 2020002401A CL 2020002401 A1 CL2020002401 A1 CL 2020002401A1
- Authority
- CL
- Chile
- Prior art keywords
- hsd17b13
- compositions
- methods
- arni
- hydroxysteroid dehydrogenases
- Prior art date
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- C—CHEMISTRY; METALLURGY
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- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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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
- C12N15/1137—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 against enzymes
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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
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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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
- C12N2310/31—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
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- 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
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- 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
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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
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Abstract
Composiciones de ácidos ribonucleicos de hebra doble (ARNhd) dirigidos al gen HSD17B13, así como métodos para inhibir la expresión de HSD17B13, y métodos para tratar a sujetos que pudieran verse beneficiados con una reducción de la expresión de HSD17B13, tales como sujetos que padecen una enfermedad, trastorno o condición asociada con HSD17B13, mediante el uso de dichas composiciones de ARNhd.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862645941P | 2018-03-21 | 2018-03-21 | |
US201862770298P | 2018-11-21 | 2018-11-21 | |
US201862775590P | 2018-12-05 | 2018-12-05 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2020002401A1 true CL2020002401A1 (es) | 2021-01-29 |
Family
ID=66001358
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2020002401A CL2020002401A1 (es) | 2018-03-21 | 2020-09-16 | Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas. |
Country Status (16)
Country | Link |
---|---|
US (2) | US20210380985A1 (es) |
EP (1) | EP3768838A1 (es) |
JP (1) | JP7507093B2 (es) |
KR (1) | KR20200133373A (es) |
CN (1) | CN112020556A (es) |
AU (1) | AU2019239971A1 (es) |
BR (1) | BR112020018758A2 (es) |
CA (1) | CA3091146A1 (es) |
CL (1) | CL2020002401A1 (es) |
CO (1) | CO2020010342A2 (es) |
IL (1) | IL277329A (es) |
MX (1) | MX2020009812A (es) |
PH (1) | PH12020500622A1 (es) |
SG (1) | SG11202007583SA (es) |
TW (1) | TW202003848A (es) |
WO (1) | WO2019183164A1 (es) |
Families Citing this family (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR102619197B1 (ko) | 2017-01-23 | 2024-01-03 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
AU2018250727A1 (en) | 2017-04-11 | 2019-10-31 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (HSD17B) family |
EP3695012B1 (en) | 2017-10-11 | 2023-03-08 | Regeneron Pharmaceuticals, Inc. | Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation |
CN112020556A (zh) | 2018-03-21 | 2020-12-01 | 瑞泽恩制药公司 | 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法 |
CN113164509A (zh) * | 2018-09-19 | 2021-07-23 | 箭头药业股份有限公司 | 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法 |
US20220273691A1 (en) | 2018-12-21 | 2022-09-01 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
MX2022015169A (es) * | 2020-06-01 | 2023-07-03 | Amgen Inc | Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos. |
WO2022098748A1 (en) * | 2020-11-06 | 2022-05-12 | Inipharm, Inc. | Uses for hsd17b13 inhibitors |
CA3201624A1 (en) | 2020-12-23 | 2022-06-30 | Regeneron Pharmaceuticals, Inc. | Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors |
TW202302852A (zh) * | 2021-04-22 | 2023-01-16 | 大陸商上海拓界生物醫藥科技有限公司 | 靶向第13型17β-羥基類固醇脫氫酶的siRNA和siRNA綴合物 |
CA3230382A1 (en) * | 2021-09-08 | 2023-03-16 | Aligos Therapeutics, Inc. | Modified short interfering nucleic acid (sina) molecules and uses thereof |
WO2023109945A1 (zh) * | 2021-12-16 | 2023-06-22 | 上海拓界生物医药科技有限公司 | 一种dsRNA、其制备方法及应用 |
US20230193392A1 (en) | 2021-12-20 | 2023-06-22 | Regeneron Pharmaceuticals, Inc. | Methods Of Identifying And Evaluating Liver Inflammation And Liver Fibrosis In A Subject By Determining A Stratified Score Based On Gene Expression |
WO2023138650A1 (zh) * | 2022-01-20 | 2023-07-27 | 上海拓界生物医药科技有限公司 | 一种dsRNA、其应用及制备方法 |
WO2023208109A1 (zh) * | 2022-04-29 | 2023-11-02 | 北京福元医药股份有限公司 | 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途 |
TW202400791A (zh) * | 2022-05-06 | 2024-01-01 | 大陸商蘇州瑞博生物技術股份有限公司 | 一種核酸、含有該核酸的組合物與綴合物及製備方法和用途 |
US20240002857A1 (en) * | 2022-05-09 | 2024-01-04 | Sanegene Bio Usa Inc. | Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof |
WO2024059165A1 (en) * | 2022-09-15 | 2024-03-21 | Alnylam Pharmaceuticals, Inc. | 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof |
Family Cites Families (30)
Publication number | Priority date | Publication date | Assignee | Title |
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US5624813A (en) | 1994-04-21 | 1997-04-29 | Mahant; Vijay K. | NAD(P)+ /NAD(P)H based chemiluminescent diagnostics |
AU1067697A (en) | 1995-12-05 | 1997-06-27 | Jouko Antero Oikarinen | Hsd17b1 promoter, enhancer, silencer and use thereof |
US6391311B1 (en) | 1998-03-17 | 2002-05-21 | Genentech, Inc. | Polypeptides having homology to vascular endothelial cell growth factor and bone morphogenetic protein 1 |
FR2801218B1 (fr) | 1999-11-23 | 2001-12-28 | Hoechst Marion Roussel Inc | Compositions pharmaceutiques comprenant de la trimegestone, leurs procedes de preparation ainsi que le conditionnement primaire les renfermant |
US20050119211A1 (en) * | 2001-05-18 | 2005-06-02 | Sirna Therapeutics, Inc. | RNA mediated inhibition connexin gene expression using short interfering nucleic acid (siNA) |
US20050182007A1 (en) * | 2001-05-18 | 2005-08-18 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of interleukin and interleukin receptor gene expression using short interfering nucleic acid (SINA) |
WO2005014811A2 (en) * | 2003-08-08 | 2005-02-17 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
JP2006507841A (ja) * | 2002-11-14 | 2006-03-09 | ダーマコン, インコーポレイテッド | 機能的siRNAおよび超機能的siRNA |
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EP2087110A2 (en) * | 2006-10-11 | 2009-08-12 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Influenza targets |
US20100056384A1 (en) | 2008-09-04 | 2010-03-04 | Board Of Regents, The University Of Texas System | Sequence Variations in PNPLA3 Associated with Hepatic Steatosis |
WO2013177060A2 (en) | 2012-05-20 | 2013-11-28 | Trustees Of Boston University | Methods and systems for monitoring, diagnosing, and treating chronic obstructive polmonary disease |
EP2864791A2 (en) | 2012-06-20 | 2015-04-29 | Leibniz - Institut für Analytische Wissenschaften - ISAS - E.V. | Specific biomarkers for hepatocellular carcinoma (hcc) |
CN103520724B (zh) * | 2013-10-23 | 2016-05-25 | 江苏美迪森生物医药有限公司 | Hsd17b13蛋白或其编码基因的抑制剂的新用途 |
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CN104698108B (zh) | 2015-03-26 | 2016-11-09 | 中国药科大学 | 一种利用固定化酶筛选I型17β羟类固醇脱氢酶抑制剂的方法 |
EP3350328A1 (en) | 2015-09-14 | 2018-07-25 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof |
TWI788312B (zh) * | 2016-11-23 | 2023-01-01 | 美商阿尼拉製藥公司 | 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法 |
KR20210035022A (ko) | 2016-12-14 | 2021-03-31 | 리간달 인코포레이티드 | 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법 |
KR102619197B1 (ko) | 2017-01-23 | 2024-01-03 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
CN112020556A (zh) | 2018-03-21 | 2020-12-01 | 瑞泽恩制药公司 | 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法 |
US20210000906A1 (en) | 2018-03-21 | 2021-01-07 | Ionis Pharmaceuticals, Inc. | Modulation of hsd17b13 expression |
CN113164509A (zh) * | 2018-09-19 | 2021-07-23 | 箭头药业股份有限公司 | 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法 |
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AU2021373262A1 (en) * | 2020-11-04 | 2023-06-01 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of pros1 in a cell |
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2019
- 2019-03-20 CN CN201980020643.3A patent/CN112020556A/zh active Pending
- 2019-03-20 US US16/978,947 patent/US20210380985A1/en active Pending
- 2019-03-20 EP EP19715322.4A patent/EP3768838A1/en active Pending
- 2019-03-20 WO PCT/US2019/023079 patent/WO2019183164A1/en unknown
- 2019-03-20 KR KR1020207030127A patent/KR20200133373A/ko not_active Application Discontinuation
- 2019-03-20 AU AU2019239971A patent/AU2019239971A1/en active Pending
- 2019-03-20 SG SG11202007583SA patent/SG11202007583SA/en unknown
- 2019-03-20 BR BR112020018758-5A patent/BR112020018758A2/pt unknown
- 2019-03-20 MX MX2020009812A patent/MX2020009812A/es unknown
- 2019-03-20 CA CA3091146A patent/CA3091146A1/en active Pending
- 2019-03-20 JP JP2020550112A patent/JP7507093B2/ja active Active
- 2019-03-21 TW TW108109810A patent/TW202003848A/zh unknown
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2020
- 2020-08-19 PH PH12020500622A patent/PH12020500622A1/en unknown
- 2020-08-21 CO CONC2020/0010342A patent/CO2020010342A2/es unknown
- 2020-09-14 IL IL277329A patent/IL277329A/en unknown
- 2020-09-16 CL CL2020002401A patent/CL2020002401A1/es unknown
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2021
- 2021-05-14 US US17/321,001 patent/US11180757B1/en active Active
Also Published As
Publication number | Publication date |
---|---|
TW202003848A (zh) | 2020-01-16 |
AU2019239971A1 (en) | 2020-09-03 |
EP3768838A1 (en) | 2021-01-27 |
KR20200133373A (ko) | 2020-11-27 |
US11180757B1 (en) | 2021-11-23 |
WO2019183164A1 (en) | 2019-09-26 |
BR112020018758A2 (pt) | 2021-01-26 |
MX2020009812A (es) | 2021-01-08 |
CA3091146A1 (en) | 2019-09-26 |
JP2021518125A (ja) | 2021-08-02 |
SG11202007583SA (en) | 2020-09-29 |
JP7507093B2 (ja) | 2024-06-27 |
CO2020010342A2 (es) | 2020-08-31 |
IL277329A (en) | 2020-10-29 |
PH12020500622A1 (en) | 2021-03-15 |
US20210380985A1 (en) | 2021-12-09 |
CN112020556A (zh) | 2020-12-01 |
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