MX2019007594A - Composiciones y metodos para tratar la deficiencia de alfa-1 antitripsina. - Google Patents
Composiciones y metodos para tratar la deficiencia de alfa-1 antitripsina.Info
- Publication number
- MX2019007594A MX2019007594A MX2019007594A MX2019007594A MX2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A
- Authority
- MX
- Mexico
- Prior art keywords
- compositions
- methods
- antitrypsin deficiency
- treating alpha
- antitrypsin
- Prior art date
Links
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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
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
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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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/513—Organic macromolecular compounds; Dendrimers
- A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
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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
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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
- 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
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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
- C12N15/63—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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- 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 RNAses, DNAses
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- C—CHEMISTRY; METALLURGY
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- 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 [CRISPRs]
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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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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- 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
- C12N2310/34—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
- C12N2310/3521—Methyl
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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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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
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- Biochemistry (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Virology (AREA)
- Pulmonology (AREA)
- Gastroenterology & Hepatology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Dispersion Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Se proporcionan composiciones y métodos para introducir rupturas de doble cadena dentro del gen de SERPINA 1. Se proporcionan composiciones y métodos para reducir y eliminar las formas mutantes de a1-antitripsina (AAT), tal como se observa en los sujetos que tienen deficiencia de a1-antitripsina (AATD).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662438219P | 2016-12-22 | 2016-12-22 | |
PCT/US2017/067800 WO2018119182A1 (en) | 2016-12-22 | 2017-12-21 | Compositions and methods for treating alpha-1 antitrypsin deficiency |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2019007594A true MX2019007594A (es) | 2019-12-16 |
Family
ID=61054487
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019007594A MX2019007594A (es) | 2016-12-22 | 2017-12-21 | Composiciones y metodos para tratar la deficiencia de alfa-1 antitripsina. |
Country Status (18)
Country | Link |
---|---|
US (2) | US11549107B2 (es) |
EP (1) | EP3559232A1 (es) |
JP (2) | JP7383478B2 (es) |
KR (1) | KR102551664B1 (es) |
CN (2) | CN117737062A (es) |
AU (2) | AU2017379073B2 (es) |
BR (1) | BR112019012825A2 (es) |
CA (1) | CA3047415A1 (es) |
CO (1) | CO2019007776A2 (es) |
EA (1) | EA201991455A1 (es) |
IL (1) | IL267448A (es) |
MX (1) | MX2019007594A (es) |
NZ (1) | NZ754739A (es) |
PH (1) | PH12019501457A1 (es) |
SG (1) | SG10202106412RA (es) |
TW (1) | TW201833331A (es) |
WO (1) | WO2018119182A1 (es) |
ZA (1) | ZA202007632B (es) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
BR112019012825A2 (pt) * | 2016-12-22 | 2019-11-26 | Intellia Therapeutics Inc | composições e métodos para tratar deficiência de alfa-1 antitripsina |
LT3688162T (lt) * | 2017-09-29 | 2024-05-27 | Intellia Therapeutics, Inc. | Preparatai |
WO2020006708A1 (en) * | 2018-07-04 | 2020-01-09 | Yale University | Compositions and methods for enhancement of homology-directed repair mediated precise gene editing by programming dna repair with a single rna-guided endonuclease |
KR20210102883A (ko) * | 2018-10-18 | 2021-08-20 | 인텔리아 테라퓨틱스, 인크. | 알부민 좌위로부터 트랜스진을 발현하기 위한 조성물 및 방법 |
JP2022505381A (ja) | 2018-10-18 | 2022-01-14 | インテリア セラピューティクス,インコーポレーテッド | アルファ1アンチトリプシン欠乏症を治療するための組成物及び方法 |
US20220160847A1 (en) * | 2020-11-20 | 2022-05-26 | Mark Egly | Method of preventing and/or treating a plurality of diseases |
AU2021394998A1 (en) | 2020-12-11 | 2023-06-29 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing involving deamination |
Family Cites Families (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
EP0618925B2 (en) | 1991-12-24 | 2012-04-18 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotides |
US6169169B1 (en) | 1994-05-19 | 2001-01-02 | Dako A/S | PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis |
US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
DK3011031T3 (da) * | 2013-06-17 | 2020-12-21 | Broad Inst Inc | Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi |
LT3036327T (lt) * | 2013-08-22 | 2019-06-25 | Pioneer Hi-Bred International, Inc. | Genomo modifikacijos, panaudojant nukreipiančias polinukleotido/cas endonukleazės sistemas, ir panaudojimo būdai |
CN106459995B (zh) * | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
US20150166984A1 (en) * | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
CN106103706B (zh) * | 2013-12-26 | 2021-08-24 | 通用医疗公司 | 多重引导rna |
CA2969619A1 (en) * | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
EP3280803B1 (en) * | 2015-04-06 | 2021-05-26 | The Board of Trustees of the Leland Stanford Junior University | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
DK3362560T3 (da) * | 2015-10-12 | 2022-11-07 | Dupont Us Holding Llc | Beskyttede dna-templater til genmodificering og forøget homolog rekombination i celler og fremgangsmåder til anvendelse |
US11866727B2 (en) * | 2015-11-06 | 2024-01-09 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1A |
US11851653B2 (en) * | 2015-12-01 | 2023-12-26 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
US11512311B2 (en) * | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
BR112019012825A2 (pt) * | 2016-12-22 | 2019-11-26 | Intellia Therapeutics Inc | composições e métodos para tratar deficiência de alfa-1 antitripsina |
JP2022505381A (ja) * | 2018-10-18 | 2022-01-14 | インテリア セラピューティクス,インコーポレーテッド | アルファ1アンチトリプシン欠乏症を治療するための組成物及び方法 |
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2017
- 2017-12-21 BR BR112019012825A patent/BR112019012825A2/pt unknown
- 2017-12-21 MX MX2019007594A patent/MX2019007594A/es unknown
- 2017-12-21 NZ NZ754739A patent/NZ754739A/en unknown
- 2017-12-21 WO PCT/US2017/067800 patent/WO2018119182A1/en unknown
- 2017-12-21 SG SG10202106412RA patent/SG10202106412RA/en unknown
- 2017-12-21 KR KR1020197020995A patent/KR102551664B1/ko active IP Right Grant
- 2017-12-21 EP EP17835903.0A patent/EP3559232A1/en active Pending
- 2017-12-21 EA EA201991455A patent/EA201991455A1/ru unknown
- 2017-12-21 CN CN202311710568.8A patent/CN117737062A/zh active Pending
- 2017-12-21 CN CN201780086243.3A patent/CN110382697B/zh active Active
- 2017-12-21 TW TW106145118A patent/TW201833331A/zh unknown
- 2017-12-21 AU AU2017379073A patent/AU2017379073B2/en active Active
- 2017-12-21 JP JP2019533577A patent/JP7383478B2/ja active Active
- 2017-12-21 CA CA3047415A patent/CA3047415A1/en active Pending
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2019
- 2019-06-18 IL IL267448A patent/IL267448A/en unknown
- 2019-06-20 US US16/446,764 patent/US11549107B2/en active Active
- 2019-06-21 PH PH12019501457A patent/PH12019501457A1/en unknown
- 2019-07-19 CO CONC2019/0007776A patent/CO2019007776A2/es unknown
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2020
- 2020-12-08 ZA ZA2020/07632A patent/ZA202007632B/en unknown
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2022
- 2022-12-09 US US18/063,817 patent/US20230212575A1/en active Pending
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2023
- 2023-02-24 JP JP2023026853A patent/JP2023075164A/ja active Pending
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2024
- 2024-03-06 AU AU2024201464A patent/AU2024201464A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
CN110382697A (zh) | 2019-10-25 |
AU2017379073B2 (en) | 2023-12-14 |
JP2023075164A (ja) | 2023-05-30 |
EP3559232A1 (en) | 2019-10-30 |
KR20190100943A (ko) | 2019-08-29 |
IL267448A (en) | 2019-08-29 |
TW201833331A (zh) | 2018-09-16 |
NZ754739A (en) | 2024-01-26 |
JP7383478B2 (ja) | 2023-11-20 |
WO2018119182A1 (en) | 2018-06-28 |
PH12019501457A1 (en) | 2020-03-09 |
KR102551664B1 (ko) | 2023-07-05 |
US11549107B2 (en) | 2023-01-10 |
US20190316129A1 (en) | 2019-10-17 |
CA3047415A1 (en) | 2018-06-28 |
SG10202106412RA (en) | 2021-07-29 |
ZA202007632B (en) | 2023-03-29 |
BR112019012825A2 (pt) | 2019-11-26 |
US20230212575A1 (en) | 2023-07-06 |
AU2024201464A1 (en) | 2024-03-28 |
JP2020501582A (ja) | 2020-01-23 |
AU2017379073A1 (en) | 2019-07-11 |
CN110382697B (zh) | 2023-12-29 |
CN117737062A (zh) | 2024-03-22 |
CO2019007776A2 (es) | 2019-10-09 |
EA201991455A1 (ru) | 2020-01-15 |
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