AU2015350561B2 - Ionizable cationic lipid for RNA delivery - Google Patents
Ionizable cationic lipid for RNA delivery Download PDFInfo
- Publication number
- AU2015350561B2 AU2015350561B2 AU2015350561A AU2015350561A AU2015350561B2 AU 2015350561 B2 AU2015350561 B2 AU 2015350561B2 AU 2015350561 A AU2015350561 A AU 2015350561A AU 2015350561 A AU2015350561 A AU 2015350561A AU 2015350561 B2 AU2015350561 B2 AU 2015350561B2
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- AU
- Australia
- Prior art keywords
- cationic lipid
- rna delivery
- ionizable cationic
- ionizable
- rna
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- -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
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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
- A61K48/0008—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
-
- 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
-
- 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
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US14/546,105 | 2014-11-18 | ||
| 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 |
| US14/707,796 | 2015-05-08 | ||
| US14/707,876 | 2015-05-08 | ||
| PCT/US2015/030218 WO2016081029A1 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for rna delivery |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2015350561A1 AU2015350561A1 (en) | 2017-07-06 |
| AU2015350561B2 true AU2015350561B2 (en) | 2020-01-30 |
Family
ID=56014367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2015350561A Active AU2015350561B2 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for RNA delivery |
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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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102095085B1 (ko) | 2013-11-18 | 2020-03-31 | ์ํฌํฌ๋ฃจ์ค ์๋ผํจํฑ์ค, ์ธํฌ. | Rna ์ ๋ฌ์ ์ํ ์ด์จํ๊ฐ๋ฅํ ์์ด์จ์ฑ ์ง์ง |
| 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 | ๋ฆฌ๊ฐ๋ฌ ์ธ์ฝํฌ๋ ์ดํฐ๋ | ํต์ฐ ๋ฐ/๋๋ ๋จ๋ฐฑ์ง ์ ์ฌ๋ฌผ ์ ๋ฌ์ ์ํ ์กฐ์ฑ๋ฌผ ๋ฐ ๋ฐฉ๋ฒ |
| 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 |
| US12083224B2 (en) | 2018-03-30 | 2024-09-10 | Arcturus Therapeutics, Inc. | Lipid particles for nucleic acid delivery |
| ES2960936T3 (es) | 2018-04-25 | 2024-03-07 | Ethris Gmbh | Formulaciones a base de lรญpidos para el suministro de ARN |
| KR20190127277A (ko) * | 2018-05-04 | 2019-11-13 | ์ฃผ์ํ์ฌ ์ผ์๋ฐ์ด์คํ | mRNA ์ ๋ฌ์ฉ ๊ณ ๋ถ์ ๋๋ ธ์ ์ ์กฐ์ฑ๋ฌผ ๋ฐ ๊ทธ ์ ์กฐ๋ฐฉ๋ฒ |
| CN116172975B (zh) * | 2018-06-08 | 2025-03-28 | ๅฏๅฃซ่ถ็ๆ ชๅผไผ็คพ | ่่ดจ้ข็ฒใๅ ๅซ่ฏฅ่่ดจ้ข็ฒ็ๆ ธ้ ธ่ฏ็ฉไปฅๅๆ ธ้ ธ้้็จ็ปๅ็ฉ |
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| JP2022550435A (ja) | 2019-10-04 | 2022-12-01 | ใฆใซใใฉใธใงใใใฏใน ใใกใผใใทใฅใผใใฃใซใซ ใคใณใณใผใใฌใคใใใ | ็ตๆใ๏ฝ๏ฝ๏ฝใฎๆนๅใใใๆฒป็็ไฝฟ็จใฎใใใฎๆนๆณ |
| CA3158293C (en) | 2019-11-15 | 2024-06-25 | Fujifilm Corporation | Lipid composition |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| CN115667530A (zh) | 2020-03-24 | 2023-01-31 | ไธไปฃ็็ฉๅ ฌๅธ | ้็ ๆฏdna่ฝฝไฝๅๅ ถ็จไบ่กจ่พพๅ ๅญixๆฒป็ๅ็็จ้ |
| JP2023520763A (ja) | 2020-03-24 | 2023-05-19 | ใธใงใใฌใผใทใงใณ ใใคใช ใซใณใใใผ | ใดใผใทใงๆฒป็่ฌใ็บ็พใใใใใฎ้ใฆใคใซในๆง๏ฝ๏ฝ๏ฝใใฏใฟใผๅใณใใฎไฝฟ็จ |
| JP2023530487A (ja) * | 2020-06-18 | 2023-07-18 | ใขใผใฏใใฅใฉในใปใปใฉใใฅใผใใฃใฏในใปใคใณใณใผใใฌใคใใใ | ใใชใฐใซใฟใใณ็ ใฎๆฒป็ใฎใใใฎ๏ฝ๏ฝ๏ฝใชใชใดใใผ |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | ใขใณใธใฃใชใฆใ ใใคใชใตใคใจใณใทใบ ใจใผใธใผ | ๏ผค๏ฝ๏ฝๅๅญใฎ็ตๆ็ฉใใใฎไฝ่ฃฝๆนๆณใๅใณใใฎไฝฟ็จๆนๆณ |
| AU2021340876A1 (en) | 2020-09-13 | 2023-04-13 | Arcturus Therapeutics, Inc. | Lipid nanoparticles encapsulation of large RNA |
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| AU2022260111A1 (en) | 2021-04-20 | 2023-11-30 | Anjarium Biosciences Ag | Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof |
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| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
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| WO2013086373A1 (en) * | 2011-12-07 | 2013-06-13 | 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 |
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| Publication number | Publication date |
|---|---|
| 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 |
| 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 |
| HRP20230342T1 (hr) | 2023-06-09 |
| HUE062130T2 (hu) | 2023-09-28 |
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