CO2023015745A2 - Administración oral de oligonucleótidos - Google Patents
Administración oral de oligonucleótidosInfo
- Publication number
- CO2023015745A2 CO2023015745A2 CONC2023/0015745A CO2023015745A CO2023015745A2 CO 2023015745 A2 CO2023015745 A2 CO 2023015745A2 CO 2023015745 A CO2023015745 A CO 2023015745A CO 2023015745 A2 CO2023015745 A2 CO 2023015745A2
- Authority
- CO
- Colombia
- Prior art keywords
- oligonucleotide
- oral administration
- cnac
- aso
- rna
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 8
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 title 1
- 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
- 239000002679 microRNA Substances 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
- 239000004055 small Interfering RNA Substances 0.000 abstract 1
- 239000013598 vector Substances 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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- 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
- 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
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- 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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- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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- 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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- 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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- 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
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- A61K9/4841—Filling excipients; Inactive ingredients
- A61K9/4858—Organic compounds
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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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- 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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- 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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- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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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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- 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
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- 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
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- 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)
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Abstract
La presente descripción proporciona composiciones de oligonucleótidos que comprenden (1) un oligonucleótido de la presente descripción, por ejemplo, un aptámero de ASO, ARNsi, ARNsh, ADN o ARN, vector de terapia génica, ARNmi, imitador de ARNmi, antimiR, señuelo de ADN o ARN, oligonucleótido CpG, o cualquier oligonucleótido terapéutico o de diagnóstico conocido en la técnica, y (ii) un derivado del ácido caprílico, por ejemplo, 5-CNAC. En algunos aspectos, la composición de oligonucleótidos se formula para su administración al tracto gastrointestinal. Por lo tanto, en algunos aspectos, la presente descripción proporciona composiciones de oligonucleótidos para administración oral que comprenden un oligonucleótido terapéutico o de diagnóstico (por ejemplo, un ASO) y un derivado del ácido caprílico (por ejemplo, 5-CNAC o su derivado).
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 |
---|---|
CO2023015745A2 true CO2023015745A2 (es) | 2023-11-30 |
Family
ID=83723158
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2023/0015745A CO2023015745A2 (es) | 2021-04-22 | 2023-11-21 | Administración oral de oligonucleótidos |
Country Status (15)
Country | Link |
---|---|
US (1) | US20220380761A1 (es) |
EP (1) | EP4326329A1 (es) |
JP (1) | JP2024514345A (es) |
KR (1) | KR20230173712A (es) |
AU (1) | AU2022261982A1 (es) |
BR (1) | BR112023021766A2 (es) |
CA (1) | CA3216091A1 (es) |
CL (1) | CL2023003110A1 (es) |
CO (1) | CO2023015745A2 (es) |
CR (1) | CR20230471A (es) |
IL (1) | IL307875A (es) |
MX (1) | MX2023012437A (es) |
PE (1) | PE20240640A1 (es) |
TW (1) | TW202308661A (es) |
WO (1) | WO2022226217A1 (es) |
Family Cites Families (33)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
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2022
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- 2022-04-21 WO PCT/US2022/025807 patent/WO2022226217A1/en active Application Filing
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- 2022-04-21 EP EP22792517.9A patent/EP4326329A1/en active Pending
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BR112023021766A2 (pt) | 2024-01-23 |
AU2022261982A1 (en) | 2023-12-07 |
CR20230471A (es) | 2023-11-01 |
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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 |
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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