BR112023021766A2 - Dispensação oral de oligonucleotídeo - Google Patents
Dispensação oral de oligonucleotídeoInfo
- Publication number
- BR112023021766A2 BR112023021766A2 BR112023021766A BR112023021766A BR112023021766A2 BR 112023021766 A2 BR112023021766 A2 BR 112023021766A2 BR 112023021766 A BR112023021766 A BR 112023021766A BR 112023021766 A BR112023021766 A BR 112023021766A BR 112023021766 A2 BR112023021766 A2 BR 112023021766A2
- Authority
- BR
- Brazil
- Prior art keywords
- oligonucleotide
- oral
- cnac
- aso
- therapeutic
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 9
- 239000000203 mixture Substances 0.000 abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 2
- XRTHAPZDZPADIL-UHFFFAOYSA-N 8-[(5-chloro-2-hydroxybenzoyl)amino]octanoic acid Chemical compound OC(=O)CCCCCCCNC(=O)C1=CC(Cl)=CC=C1O XRTHAPZDZPADIL-UHFFFAOYSA-N 0.000 abstract 2
- 108020004414 DNA Proteins 0.000 abstract 2
- 108091070501 miRNA Proteins 0.000 abstract 2
- WWZKQHOCKIZLMA-UHFFFAOYSA-N octanoic acid Chemical class CCCCCCCC(O)=O WWZKQHOCKIZLMA-UHFFFAOYSA-N 0.000 abstract 2
- 230000001225 therapeutic effect Effects 0.000 abstract 2
- 108091023037 Aptamer Proteins 0.000 abstract 1
- 108091027967 Small hairpin RNA Proteins 0.000 abstract 1
- 108020004459 Small interfering RNA Proteins 0.000 abstract 1
- 210000001035 gastrointestinal tract Anatomy 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 230000003278 mimic effect Effects 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
- A61K9/4841—Filling excipients; Inactive ingredients
- A61K9/4858—Organic compounds
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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
- A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
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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
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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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
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
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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
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
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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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/20—Pills, tablets, discs, rods
- A61K9/2004—Excipients; Inactive ingredients
- A61K9/2013—Organic compounds, e.g. phospholipids, fats
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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
- A61K9/4816—Wall or shell material
- A61K9/4825—Proteins, e.g. gelatin
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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
- A61K9/4891—Coated capsules; Multilayered drug free capsule shells
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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
- 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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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/16—Aptamers
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—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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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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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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- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Abstract
dispensação oral de oligonucleotídeo. a presente descrição provê composições de oligonucleotídeo compreendendo (i) um oligonucleotídeo da presente descrição, por exemplo, um aso, sirna, shrna, aptâmero de dna ou rna, vetor de terapia gênica, mirna, imitador de mirna, antimir, isca de dna ou rna, oligonucleotídeo cpg ou qualquer oligonucleotídeo terapêutico ou de diagnóstico conhecido na técnica e (ii) um derivado de ácido caprílico, por exemplo, 5-cnac. em alguns aspectos, a composição de oligonucleotídeo é formulada para dispensação ao trato gastrointestinal. assim, em alguns aspectos, a presente descrição provê composições de oligonucleotídeo para dispensação oral compreendendo um oligonucleotídeo terapêutico ou de diagnóstico (por exemplo, um aso) e um derivado de ácido caprílico (por exemplo, 5-cnac ou de derivado do mesmo).
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163178361P | 2021-04-22 | 2021-04-22 | |
US202163261506P | 2021-09-22 | 2021-09-22 | |
US202163288379P | 2021-12-10 | 2021-12-10 | |
PCT/US2022/025807 WO2022226217A1 (en) | 2021-04-22 | 2022-04-21 | Oral delivery of oligonucleotides |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112023021766A2 true BR112023021766A2 (pt) | 2024-01-23 |
Family
ID=83723158
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112023021766A BR112023021766A2 (pt) | 2021-04-22 | 2022-04-21 | Dispensação oral de oligonucleotídeo |
Country Status (15)
Country | Link |
---|---|
US (1) | US20220380761A1 (pt) |
EP (1) | EP4326329A1 (pt) |
JP (1) | JP2024514345A (pt) |
KR (1) | KR20230173712A (pt) |
AU (1) | AU2022261982A1 (pt) |
BR (1) | BR112023021766A2 (pt) |
CA (1) | CA3216091A1 (pt) |
CL (1) | CL2023003110A1 (pt) |
CO (1) | CO2023015745A2 (pt) |
CR (1) | CR20230471A (pt) |
IL (1) | IL307875A (pt) |
MX (1) | MX2023012437A (pt) |
PE (1) | PE20240640A1 (pt) |
TW (1) | TW202308661A (pt) |
WO (1) | WO2022226217A1 (pt) |
Family Cites Families (33)
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US4043996A (en) | 1975-01-27 | 1977-08-23 | Swift & Company | Gelatin manufacture-peroxide liquefaction process |
GB1557005A (en) | 1975-07-11 | 1979-12-05 | Novo Industri As | Gelatine extraction |
US4176117A (en) | 1977-03-11 | 1979-11-27 | Leon Oudem | Process for obtaining gelatin |
US4232425A (en) | 1980-01-15 | 1980-11-11 | Darling & Company | Method of producing stabilized bone |
ZA816771B (en) | 1980-10-07 | 1982-10-27 | Lensfield Prod Ltd | Protein production |
US4374063A (en) | 1981-09-28 | 1983-02-15 | General Foods Corporation | Process for the preparation and purification of gelatin and pyrogen-free gelatin so prepared |
US4402873A (en) | 1982-09-23 | 1983-09-06 | Sugardale Foods Incorporated | Extraction of protein from pork bones |
DE3726963A1 (de) | 1987-08-13 | 1989-02-23 | Stoess & Co Gelatine | Kaltwasserloesliche, instantisierte gelatine und verfahren zur herstellung derselben |
FR2625412B1 (fr) | 1987-12-30 | 1990-06-29 | Mero Rousselot Satia | Procede continu d'obtention de gelatine a partir de poudre d'os et gelatine obtenue |
IL87344A (en) | 1988-08-04 | 1992-03-29 | Univ Bar Ilan | Process for the production of gelatin from fish skins |
US5210182A (en) | 1992-02-12 | 1993-05-11 | Kraft General Foods, Inc. | Extraction process for gelatin |
US5288408A (en) | 1992-10-26 | 1994-02-22 | Chemical Industry Consultants, Inc. | Method of gelatin recovery and purification from encapsulation processes |
US5650386A (en) | 1995-03-31 | 1997-07-22 | Emisphere Technologies, Inc. | Compositions for oral delivery of active agents |
US5866536A (en) | 1995-03-31 | 1999-02-02 | Emisphere Technologies, Inc. | Compounds and compositions for delivering active agents |
US6090915A (en) | 1996-10-18 | 2000-07-18 | Hormel Foods Corporation | Collagen or gelatin crumble composition and uses |
US5851579A (en) | 1996-10-28 | 1998-12-22 | Eastman Chemical Company | Aqueous enteric coating compositions |
US5773647A (en) | 1997-02-07 | 1998-06-30 | Emisphere Technologies, Inc. | Compounds and compositions for delivering active agents |
GB9807331D0 (en) | 1998-04-07 | 1998-06-03 | Cerestar Holding Bv | Gelatin replacement by wheat fiber gel and starch |
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EP1535625B1 (en) * | 1999-04-05 | 2014-01-08 | Novartis AG | Composition containing n-(5-chlorosalicyloyl)-8-aminocaprylic acid and salmon calcitonin |
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2022
- 2022-04-21 KR KR1020237040040A patent/KR20230173712A/ko unknown
- 2022-04-21 MX MX2023012437A patent/MX2023012437A/es unknown
- 2022-04-21 IL IL307875A patent/IL307875A/en unknown
- 2022-04-21 WO PCT/US2022/025807 patent/WO2022226217A1/en active Application Filing
- 2022-04-21 BR BR112023021766A patent/BR112023021766A2/pt unknown
- 2022-04-21 CA CA3216091A patent/CA3216091A1/en active Pending
- 2022-04-21 EP EP22792517.9A patent/EP4326329A1/en active Pending
- 2022-04-21 CR CR20230471A patent/CR20230471A/es unknown
- 2022-04-21 AU AU2022261982A patent/AU2022261982A1/en active Pending
- 2022-04-21 JP JP2023564094A patent/JP2024514345A/ja active Pending
- 2022-04-21 PE PE2023002902A patent/PE20240640A1/es unknown
- 2022-04-21 US US17/726,371 patent/US20220380761A1/en active Pending
- 2022-04-22 TW TW111115515A patent/TW202308661A/zh unknown
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2023
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IL307875A (en) | 2023-12-01 |
JP2024514345A (ja) | 2024-04-01 |
AU2022261982A1 (en) | 2023-12-07 |
CR20230471A (es) | 2023-11-01 |
CA3216091A1 (en) | 2022-10-27 |
AU2022261982A9 (en) | 2023-12-14 |
KR20230173712A (ko) | 2023-12-27 |
CL2023003110A1 (es) | 2024-02-16 |
TW202308661A (zh) | 2023-03-01 |
WO2022226217A1 (en) | 2022-10-27 |
CO2023015745A2 (es) | 2023-11-30 |
AU2022261982A2 (en) | 2024-01-11 |
MX2023012437A (es) | 2023-11-07 |
PE20240640A1 (es) | 2024-04-04 |
EP4326329A1 (en) | 2024-02-28 |
US20220380761A1 (en) | 2022-12-01 |
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