HRP20230342T1 - Kationski lipid koji se može ionizirati za isporuku rna - Google Patents
Kationski lipid koji se može ionizirati za isporuku rna Download PDFInfo
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- HRP20230342T1 HRP20230342T1 HRP20230342TT HRP20230342T HRP20230342T1 HR P20230342 T1 HRP20230342 T1 HR P20230342T1 HR P20230342T T HRP20230342T T HR P20230342TT HR P20230342 T HRP20230342 T HR P20230342T HR P20230342 T1 HRP20230342 T1 HR P20230342T1
- Authority
- HR
- Croatia
- Prior art keywords
- compound according
- consist
- branched alkyl
- cationic lipid
- carbon atoms
- Prior art date
Links
- -1 cationic lipid Chemical class 0.000 title 1
- 150000001875 compounds Chemical class 0.000 claims 10
- 125000000217 alkyl group Chemical group 0.000 claims 7
- 125000004432 carbon atom Chemical group C* 0.000 claims 4
- VGGSQFUCUMXWEO-UHFFFAOYSA-N Ethene Chemical compound C=C VGGSQFUCUMXWEO-UHFFFAOYSA-N 0.000 claims 2
- 239000005977 Ethylene Substances 0.000 claims 2
- 125000004805 propylene group Chemical group [H]C([H])([H])C([H])([*:1])C([H])([H])[*:2] 0.000 claims 2
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 125000001570 methylene group Chemical group [H]C([H])([*:1])[*:2] 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C323/00—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
- C07C323/50—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton
- C07C323/51—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
- C07C323/60—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton with the carbon atom of at least one of the carboxyl groups bound to nitrogen atoms
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0043—Nose
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/007—Pulmonary tract; Aromatherapy
- A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
-
- 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
- A61K9/12—Aerosols; Foams
-
- 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
-
- 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
- C07C333/02—Monothiocarbamic acids; Derivatives thereof
- C07C333/04—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
- C07C333/02—Monothiocarbamic acids; Derivatives thereof
- C07C333/10—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups being part of any of the groups, X being a hetero atom, Y being any atom, e.g., N-acyl-thiocarbamates
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J1/00—Normal steroids containing carbon, hydrogen, halogen or oxygen, not substituted in position 17 beta by a carbon atom, e.g. estrane, androstane
- C07J1/0003—Androstane derivatives
- C07J1/0007—Androstane derivatives not substituted in position 17
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J31/00—Normal steroids containing one or more sulfur atoms not belonging to a hetero ring
- C07J31/006—Normal steroids containing one or more sulfur atoms not belonging to a hetero ring not covered by C07J31/003
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
- C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
- C07J41/0055—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
-
- 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
-
- 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
-
- 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 nucleic acids [NA]
-
- 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
Landscapes
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Dispersion Chemistry (AREA)
- Biophysics (AREA)
- Otolaryngology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Pulmonology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US14/546,105 US9593077B2 (en) | 2013-11-18 | 2014-11-18 | Ionizable cationic lipid for RNA delivery |
| US14/707,876 US9365610B2 (en) | 2013-11-18 | 2015-05-08 | Asymmetric ionizable cationic lipid for RNA delivery |
| US14/707,796 US9567296B2 (en) | 2013-11-18 | 2015-05-08 | Ionizable cationic lipid for RNA delivery |
| PCT/US2015/030218 WO2016081029A1 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for rna delivery |
| EP15724466.6A EP3221293B1 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for rna delivery |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20230342T1 true HRP20230342T1 (hr) | 2023-06-09 |
Family
ID=56014367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20230342TT HRP20230342T1 (hr) | 2014-11-18 | 2015-05-11 | Kationski lipid koji se može ionizirati za isporuku rna |
Country Status (20)
| Country | Link |
|---|---|
| EP (1) | EP3221293B1 (OSRAM) |
| JP (1) | JP6637988B2 (OSRAM) |
| KR (1) | KR102380363B1 (OSRAM) |
| CN (1) | CN107207428A (OSRAM) |
| AU (1) | AU2015350561B2 (OSRAM) |
| CA (1) | CA2968060C (OSRAM) |
| DK (1) | DK3221293T3 (OSRAM) |
| ES (1) | ES2946110T3 (OSRAM) |
| FI (1) | FI3221293T3 (OSRAM) |
| HK (1) | HK1244479A1 (OSRAM) |
| HR (1) | HRP20230342T1 (OSRAM) |
| HU (1) | HUE062130T2 (OSRAM) |
| IL (1) | IL252291B (OSRAM) |
| LT (1) | LT3221293T (OSRAM) |
| NZ (1) | NZ732777A (OSRAM) |
| PL (1) | PL3221293T3 (OSRAM) |
| PT (1) | PT3221293T (OSRAM) |
| RS (1) | RS64078B1 (OSRAM) |
| SI (1) | SI3221293T1 (OSRAM) |
| WO (1) | WO2016081029A1 (OSRAM) |
Families Citing this family (61)
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| KR102095085B1 (ko) | 2013-11-18 | 2020-03-31 | 아크투루스 쎄라퓨틱스, 인크. | Rna 전달을 위한 이온화가능한 양이온성 지질 |
| EP3126495A1 (en) | 2014-04-02 | 2017-02-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| KR20210035022A (ko) | 2016-12-14 | 2021-03-31 | 리간달 인코포레이티드 | 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법 |
| US10383952B2 (en) * | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| US10526284B2 (en) | 2016-12-21 | 2020-01-07 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| WO2018119163A1 (en) * | 2016-12-21 | 2018-06-28 | Payne Joseph E | Ionizable cationic lipid for rna delivery |
| EP3600396A4 (en) * | 2017-03-30 | 2021-01-13 | The Government of the United States of America as represented by the Secretary of the Army | VACCINE COMPOSITION OF NUCLEIC ACID INCLUDING A LIPIDIC FORMULATION, AND PROCESS FOR INCREASING THE EFFECTIVENESS OF NUCLEIC ACID VACCINES |
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| ES2960936T3 (es) | 2018-04-25 | 2024-03-07 | Ethris Gmbh | Formulaciones a base de lípidos para el suministro de ARN |
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| WO2025128853A2 (en) | 2023-12-13 | 2025-06-19 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with ube3a overexpression |
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| WO2025166323A2 (en) | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting lipoprotein (a) expression |
| US20250381150A1 (en) | 2024-03-08 | 2025-12-18 | Genzyme Corporation | Lipid nanoparticles |
| WO2025213138A1 (en) | 2024-04-05 | 2025-10-09 | Editas Medicine, Inc. | Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0079963B1 (en) | 1981-06-04 | 1986-08-27 | Delaware Chemicals Corporation | Pressurized container with dispensing pump |
| US4778810A (en) | 1987-01-08 | 1988-10-18 | Nastech Pharmaceutical Co., Inc. | Nasal delivery of caffeine |
| PL169576B1 (pl) | 1990-10-12 | 1996-08-30 | Max Planck Gesellschaft | Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| US5849902A (en) | 1996-09-26 | 1998-12-15 | Oligos Etc. Inc. | Three component chimeric antisense oligonucleotides |
| US9408914B2 (en) * | 2010-04-28 | 2016-08-09 | Kyowa Hakko Kirin Co., Ltd. | Cationic lipid |
| EP2998289B1 (en) * | 2011-06-08 | 2019-08-07 | Nitto Denko Corporation | Compounds for targeting drug delivery and enhancing sirna activity |
| US20140308304A1 (en) * | 2011-12-07 | 2014-10-16 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| BR112014030714B1 (pt) * | 2012-06-08 | 2020-12-22 | Nitto Denko Corporation | composto de lipídeo ionizável, composição, veículo de fármaco e formulação farmacêutica |
| KR102095085B1 (ko) * | 2013-11-18 | 2020-03-31 | 아크투루스 쎄라퓨틱스, 인크. | Rna 전달을 위한 이온화가능한 양이온성 지질 |
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2015
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- 2015-05-11 PL PL15724466.6T patent/PL3221293T3/pl unknown
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- 2015-05-11 KR KR1020177016742A patent/KR102380363B1/ko active Active
- 2015-05-11 SI SI201531935T patent/SI3221293T1/sl unknown
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| DK3221293T3 (da) | 2023-04-03 |
| CA2968060A1 (en) | 2016-05-26 |
| SI3221293T1 (sl) | 2023-05-31 |
| IL252291A0 (en) | 2017-07-31 |
| WO2016081029A1 (en) | 2016-05-26 |
| AU2015350561A1 (en) | 2017-07-06 |
| HK1244479A1 (zh) | 2018-08-10 |
| KR102380363B1 (ko) | 2022-03-29 |
| PT3221293T (pt) | 2023-03-16 |
| ES2946110T3 (es) | 2023-07-12 |
| RS64078B1 (sr) | 2023-04-28 |
| LT3221293T (lt) | 2023-03-27 |
| EP3221293B1 (en) | 2023-03-01 |
| AU2015350561B2 (en) | 2020-01-30 |
| FI3221293T3 (fi) | 2023-04-05 |
| CA2968060C (en) | 2023-06-20 |
| IL252291B (en) | 2021-08-31 |
| PL3221293T3 (pl) | 2023-07-10 |
| CN107207428A (zh) | 2017-09-26 |
| JP6637988B2 (ja) | 2020-01-29 |
| NZ732777A (en) | 2023-06-30 |
| EP3221293A1 (en) | 2017-09-27 |
| KR20170095241A (ko) | 2017-08-22 |
| JP2017535610A (ja) | 2017-11-30 |
| HUE062130T2 (hu) | 2023-09-28 |
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