IL252291B - A cationic lipid that undergoes ionization to provide RNA - Google Patents
A cationic lipid that undergoes ionization to provide RNAInfo
- Publication number
- IL252291B IL252291B IL252291A IL25229117A IL252291B IL 252291 B IL252291 B IL 252291B IL 252291 A IL252291 A IL 252291A IL 25229117 A IL25229117 A IL 25229117A IL 252291 B IL252291 B IL 252291B
- Authority
- IL
- Israel
- Prior art keywords
- cationic lipid
- rna delivery
- ionizable cationic
- ionizable
- rna
- Prior art date
Links
- -1 cationic lipid Chemical class 0.000 title 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
- A61K9/10—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
-
- 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
-
- 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)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Dispersion Chemistry (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Otolaryngology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Pulmonology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (4)
| 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| IL252291A0 IL252291A0 (en) | 2017-07-31 |
| IL252291B true IL252291B (en) | 2021-08-31 |
Family
ID=56014367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL252291A IL252291B (en) | 2014-11-18 | 2017-05-15 | A cationic lipid that undergoes ionization to provide RNA |
Country Status (20)
| Country | Link |
|---|---|
| EP (1) | EP3221293B1 (enExample) |
| JP (1) | JP6637988B2 (enExample) |
| KR (1) | KR102380363B1 (enExample) |
| CN (1) | CN107207428A (enExample) |
| AU (1) | AU2015350561B2 (enExample) |
| CA (1) | CA2968060C (enExample) |
| DK (1) | DK3221293T3 (enExample) |
| ES (1) | ES2946110T3 (enExample) |
| FI (1) | FI3221293T3 (enExample) |
| HK (1) | HK1244479A1 (enExample) |
| HR (1) | HRP20230342T1 (enExample) |
| HU (1) | HUE062130T2 (enExample) |
| IL (1) | IL252291B (enExample) |
| LT (1) | LT3221293T (enExample) |
| NZ (1) | NZ732777A (enExample) |
| PL (1) | PL3221293T3 (enExample) |
| PT (1) | PT3221293T (enExample) |
| RS (1) | RS64078B1 (enExample) |
| SI (1) | SI3221293T1 (enExample) |
| WO (1) | WO2016081029A1 (enExample) |
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| JP6486955B2 (ja) | 2013-11-18 | 2019-03-20 | アークトゥルス セラピューティクス， インコーポレイテッド | Ｒｎａ送達のためのイオン化可能なカチオン性脂質 |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| JP7317706B2 (ja) | 2016-12-14 | 2023-07-31 | リガンダル インコーポレイテッド | 核酸およびタンパク質ペイロード送達のための方法および組成物 |
| WO2018119163A1 (en) * | 2016-12-21 | 2018-06-28 | Payne Joseph E | Ionizable cationic lipid for rna delivery |
| US10526284B2 (en) | 2016-12-21 | 2020-01-07 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| US10383952B2 (en) * | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | 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 |
| ES3039647T3 (en) * | 2017-05-31 | 2025-10-23 | Arcturus Therapeutics Inc | Synthesis and structure of high potency rna therapeutics |
| US12083224B2 (en) | 2018-03-30 | 2024-09-10 | Arcturus Therapeutics, Inc. | Lipid particles for nucleic acid delivery |
| AU2019258678B2 (en) | 2018-04-25 | 2024-08-22 | Ethris Gmbh | Lipid-based formulations for the delivery of RNA |
| KR20190127277A (ko) * | 2018-05-04 | 2019-11-13 | 주식회사 삼양바이오팜 | mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법 |
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| JPS58500845A (ja) | 1981-06-04 | 1983-05-26 | デラウェア・ケミカルス・コーポレーション | ポンプ |
| US4778810A (en) | 1987-01-08 | 1988-10-18 | Nastech Pharmaceutical Co., Inc. | Nasal delivery of caffeine |
| US5672695A (en) | 1990-10-12 | 1997-09-30 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Modified ribozymes |
| 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 |
| EP2567951B1 (en) * | 2010-04-28 | 2018-09-19 | Kyowa Hakko Kirin Co., Ltd. | Cationic lipid |
| CA2837101C (en) * | 2011-06-08 | 2020-12-15 | Priya Karmali | 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 |
| WO2013185116A1 (en) * | 2012-06-08 | 2013-12-12 | Payne Joseph E | Lipids for therapeutic agent delivery formulations |
| JP6486955B2 (ja) * | 2013-11-18 | 2019-03-20 | アークトゥルス セラピューティクス， インコーポレイテッド | Ｒｎａ送達のためのイオン化可能なカチオン性脂質 |
-
2015
- 2015-05-11 JP JP2017545520A patent/JP6637988B2/ja active Active
- 2015-05-11 KR KR1020177016742A patent/KR102380363B1/ko active Active
- 2015-05-11 CN CN201580073670.9A patent/CN107207428A/zh active Pending
- 2015-05-11 NZ NZ732777A patent/NZ732777A/en unknown
- 2015-05-11 FI FIEP15724466.6T patent/FI3221293T3/fi active
- 2015-05-11 AU AU2015350561A patent/AU2015350561B2/en active Active
- 2015-05-11 HR HRP20230342TT patent/HRP20230342T1/hr unknown
- 2015-05-11 LT LTEPPCT/US2015/030218T patent/LT3221293T/lt unknown
- 2015-05-11 CA CA2968060A patent/CA2968060C/en active Active
- 2015-05-11 RS RS20230231A patent/RS64078B1/sr unknown
- 2015-05-11 SI SI201531935T patent/SI3221293T1/sl unknown
- 2015-05-11 DK DK15724466.6T patent/DK3221293T3/da active
- 2015-05-11 PT PT157244666T patent/PT3221293T/pt unknown
- 2015-05-11 PL PL15724466.6T patent/PL3221293T3/pl unknown
- 2015-05-11 WO PCT/US2015/030218 patent/WO2016081029A1/en not_active Ceased
- 2015-05-11 HK HK18103903.0A patent/HK1244479A1/zh unknown
- 2015-05-11 HU HUE15724466A patent/HUE062130T2/hu unknown
- 2015-05-11 ES ES15724466T patent/ES2946110T3/es active Active
- 2015-05-11 EP EP15724466.6A patent/EP3221293B1/en active Active
-
2017
- 2017-05-15 IL IL252291A patent/IL252291B/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| SI3221293T1 (sl) | 2023-05-31 |
| HRP20230342T1 (hr) | 2023-06-09 |
| KR102380363B1 (ko) | 2022-03-29 |
| PT3221293T (pt) | 2023-03-16 |
| DK3221293T3 (da) | 2023-04-03 |
| NZ732777A (en) | 2023-06-30 |
| ES2946110T3 (es) | 2023-07-12 |
| KR20170095241A (ko) | 2017-08-22 |
| FI3221293T3 (fi) | 2023-04-05 |
| LT3221293T (lt) | 2023-03-27 |
| PL3221293T3 (pl) | 2023-07-10 |
| EP3221293B1 (en) | 2023-03-01 |
| CA2968060C (en) | 2023-06-20 |
| CA2968060A1 (en) | 2016-05-26 |
| JP2017535610A (ja) | 2017-11-30 |
| AU2015350561B2 (en) | 2020-01-30 |
| RS64078B1 (sr) | 2023-04-28 |
| HUE062130T2 (hu) | 2023-09-28 |
| JP6637988B2 (ja) | 2020-01-29 |
| IL252291A0 (en) | 2017-07-31 |
| EP3221293A1 (en) | 2017-09-27 |
| HK1244479A1 (zh) | 2018-08-10 |
| WO2016081029A1 (en) | 2016-05-26 |
| CN107207428A (zh) | 2017-09-26 |
| AU2015350561A1 (en) | 2017-07-06 |
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