FI3221293T3 - Ionisoituva kationinen lipidi rna:n antamiseen - Google Patents
Ionisoituva kationinen lipidi rna:n antamiseen Download PDFInfo
- Publication number
- FI3221293T3 FI3221293T3 FIEP15724466.6T FI15724466T FI3221293T3 FI 3221293 T3 FI3221293 T3 FI 3221293T3 FI 15724466 T FI15724466 T FI 15724466T FI 3221293 T3 FI3221293 T3 FI 3221293T3
- Authority
- FI
- Finland
- 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
-
- 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
- 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
- A61K9/10—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
- C07C333/10—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
- A61K9/007—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
- 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
- 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)
- 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 (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 |
| US14/707,796 US9567296B2 (en) | 2013-11-18 | 2015-05-08 | Ionizable cationic lipid for RNA delivery |
| US14/707,876 US9365610B2 (en) | 2013-11-18 | 2015-05-08 | Asymmetric ionizable cationic lipid for RNA delivery |
| PCT/US2015/030218 WO2016081029A1 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for rna delivery |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| FI3221293T3 true FI3221293T3 (fi) | 2023-04-05 |
Family
ID=56014367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| FIEP15724466.6T FI3221293T3 (fi) | 2014-11-18 | 2015-05-11 | Ionisoituva kationinen lipidi rna:n antamiseen |
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) |
Families Citing this family (62)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014348212C1 (en) | 2013-11-18 | 2018-11-29 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| CN110582302A (zh) | 2016-12-14 | 2019-12-17 | 利甘达尔股份有限公司 | 用于核酸和/或蛋白有效负载递送的组合物和方法 |
| 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 |
| 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 |
| WO2018222890A1 (en) * | 2017-05-31 | 2018-12-06 | Arcturus Therapeutics, Inc. | Synthesis and structure of high potency rna therapeutics |
| WO2019191780A1 (en) * | 2018-03-30 | 2019-10-03 | Arcturus Therapeutics, Inc. | Lipid particles for nucleic acid delivery |
| WO2019207060A1 (en) | 2018-04-25 | 2019-10-31 | 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 |
| KR102591743B1 (ko) * | 2018-06-08 | 2023-10-19 | 후지필름 가부시키가이샤 | 화합물 또는 그 염 및 지질 입자 |
| HUE064076T2 (hu) | 2018-12-06 | 2024-02-28 | Arcturus Therapeutics Inc | Készítmények és módszerek az ornitin-transzkarbamiláz hiányának kezelésére |
| SG11202110036RA (en) * | 2019-03-19 | 2021-10-28 | Arcturus Therapeutics Inc | Method of making lipid-encapsulated rna nanoparticles |
| KR102660546B1 (ko) | 2019-06-07 | 2024-04-24 | 후지필름 가부시키가이샤 | 지질 조성물 |
| US20220347298A1 (en) | 2019-10-04 | 2022-11-03 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| JP7389134B2 (ja) | 2019-11-15 | 2023-11-29 | 富士フイルム株式会社 | 脂質組成物 |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| WO2021195218A1 (en) | 2020-03-24 | 2021-09-30 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing gaucher therapeutics |
| AU2021244555A1 (en) | 2020-03-24 | 2022-11-24 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing factor ix therapeutics |
| JP2023530487A (ja) * | 2020-06-18 | 2023-07-18 | アークトゥラス・セラピューティクス・インコーポレイテッド | ポリグルタミン病の治療のためのｕｎａオリゴマー |
| CA3189740A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| CA3194951A1 (en) | 2020-09-13 | 2022-03-17 | Arcturus Therapeutics, Inc. | Lipid nanoparticles encapsulation of large rna |
| IL305064A (en) * | 2021-02-10 | 2023-10-01 | Oncorus Inc | Compounds, preparations and methods of using them |
| JP2024517427A (ja) | 2021-04-20 | 2024-04-22 | アンジャリウム バイオサイエンシズ エージー | アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法 |
| WO2022232289A1 (en) | 2021-04-27 | 2022-11-03 | Generation Bio Co. | Non-viral dna vectors expressing therapeutic antibodies and uses thereof |
| EP4329884A1 (en) | 2021-04-27 | 2024-03-06 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| IL309505A (en) | 2021-09-03 | 2024-02-01 | CureVac SE | Novel lipid nanoparticles for delivery of nucleic acids |
| EP4422698A1 (en) | 2021-10-29 | 2024-09-04 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| CN118541346A (zh) | 2021-11-08 | 2024-08-23 | 奥纳治疗公司 | 用于递送环状多核苷酸的脂质纳米颗粒组合物 |
| WO2023135273A2 (en) | 2022-01-14 | 2023-07-20 | Anjarium Biosciences Ag | Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof |
| EP4469091A1 (en) | 2022-01-28 | 2024-12-04 | CureVac SE | Nucleic acid encoded transcription factor inhibitors |
| CN114213295B (zh) * | 2022-02-22 | 2022-05-20 | 中国科学院基础医学与肿瘤研究所(筹) | 一种阳离子化合物、制备方法及其复合物和用途 |
| WO2023177655A1 (en) | 2022-03-14 | 2023-09-21 | Generation Bio Co. | Heterologous prime boost vaccine compositions and methods of use |
| CA3248209A1 (en) | 2022-05-25 | 2023-11-30 | CureVac SE | Escherichia coli Antigenic Polypeptide-Based Nucleic Acid Vaccine (FIMH) |
| WO2023239756A1 (en) | 2022-06-07 | 2023-12-14 | Generation Bio Co. | Lipid nanoparticle compositions and uses thereof |
| WO2024040222A1 (en) | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Cleavable closed-ended dna (cedna) and methods of use thereof |
| IL319704A (en) | 2022-09-23 | 2025-05-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of HBV gene expression |
| DE202023106198U1 (de) | 2022-10-28 | 2024-03-21 | CureVac SE | Impfstoff auf Nukleinsäurebasis |
| WO2024102730A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Lipids and nanoparticle compositions for delivering polynucleotides |
| WO2024102762A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Lipids and lipid nanoparticle compositions for delivering polynucleotides |
| WO2024102677A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Circular rna compositions |
| CN120615013A (zh) | 2022-12-01 | 2025-09-09 | 世代生物公司 | 用于细胞靶向的隐形脂质纳米颗粒组合物 |
| AU2023406321A1 (en) | 2022-12-01 | 2025-05-29 | Generation Bio Co. | Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same |
| WO2024119039A2 (en) | 2022-12-01 | 2024-06-06 | Generation Bio Co. | Stealth lipid nanoparticles and uses thereof |
| EP4626402A1 (en) | 2022-12-01 | 2025-10-08 | Generation Bio Co. | Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers |
| DE112024001143T5 (de) | 2023-03-08 | 2025-12-18 | CureVac SE | Neue lipid-nanopartikel-formeln für die abgabe von nukleinsäuren |
| WO2024205657A2 (en) | 2023-03-29 | 2024-10-03 | Orna Therapeutics, Inc. | Lipids and lipid nanoparticle compositions for delivering polynucleotides |
| AU2024269222A1 (en) | 2023-05-05 | 2025-10-09 | Orna Therapeutics, Inc. | Circular rna compositions and methods |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| CN116270543B (zh) * | 2023-05-19 | 2023-09-26 | 清华大学 | 脂质纳米颗粒在制备通过雾吸或鼻滴给药递送核酸的药物中的用途 |
| US12311033B2 (en) | 2023-05-31 | 2025-05-27 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025049690A1 (en) | 2023-08-29 | 2025-03-06 | Orna Therapeutics, Inc. | Circular polyethylene glycol lipids |
| WO2025052180A2 (en) | 2023-09-07 | 2025-03-13 | Axelyf ehf. | Lipids and lipid nanoparticles |
| WO2025080939A1 (en) | 2023-10-13 | 2025-04-17 | Ultragenyx Pharmaceutical, Inc. | Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations |
| WO2025101501A1 (en) | 2023-11-07 | 2025-05-15 | Orna Therapeutics, Inc. | Circular rna compositions |
| WO2025128853A2 (en) | 2023-12-13 | 2025-06-19 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with ube3a overexpression |
| WO2025129201A1 (en) | 2023-12-15 | 2025-06-19 | Capstan Therapeutics, Inc. | Humanized anti-cd8 antibodies and uses thereof |
| WO2025166325A1 (en) | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | MODIFIED GUIDE RNAs |
| WO2025189064A1 (en) | 2024-03-08 | 2025-09-12 | 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 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4511069A (en) | 1981-06-04 | 1985-04-16 | The Pharmasol Corporation | Dispensing system |
| US4778810A (en) | 1987-01-08 | 1988-10-18 | Nastech Pharmaceutical Co., Inc. | Nasal delivery of caffeine |
| CA2093664C (en) | 1990-10-12 | 2003-07-29 | Fritz Eckstein | 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 |
| US9408914B2 (en) * | 2010-04-28 | 2016-08-09 | Kyowa Hakko Kirin Co., Ltd. | Cationic lipid |
| KR102038300B1 (ko) * | 2011-06-08 | 2019-10-31 | 닛토덴코 가부시키가이샤 | 표적 약물 전달체 및 ｓｉＲＮＡ 활성을 증가시키는 화합물 |
| WO2013086373A1 (en) * | 2011-12-07 | 2013-06-13 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| PT2858974T (pt) * | 2012-06-08 | 2018-11-29 | Nitto Denko Corp | Lípidos para formulações de administração de agentes terapêuticos |
| AU2014348212C1 (en) * | 2013-11-18 | 2018-11-29 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
-
2015
- 2015-05-11 PL PL15724466.6T patent/PL3221293T3/pl unknown
- 2015-05-11 CA CA2968060A patent/CA2968060C/en active Active
- 2015-05-11 FI FIEP15724466.6T patent/FI3221293T3/fi active
- 2015-05-11 EP EP15724466.6A patent/EP3221293B1/en active Active
- 2015-05-11 RS RS20230231A patent/RS64078B1/sr unknown
- 2015-05-11 KR KR1020177016742A patent/KR102380363B1/ko active Active
- 2015-05-11 WO PCT/US2015/030218 patent/WO2016081029A1/en not_active Ceased
- 2015-05-11 AU AU2015350561A patent/AU2015350561B2/en 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 HK HK18103903.0A patent/HK1244479A1/zh unknown
- 2015-05-11 PT PT157244666T patent/PT3221293T/pt unknown
- 2015-05-11 LT LTEPPCT/US2015/030218T patent/LT3221293T/lt unknown
- 2015-05-11 DK DK15724466.6T patent/DK3221293T3/da active
- 2015-05-11 SI SI201531935T patent/SI3221293T1/sl unknown
- 2015-05-11 HU HUE15724466A patent/HUE062130T2/hu unknown
- 2015-05-11 ES ES15724466T patent/ES2946110T3/es active Active
- 2015-05-11 HR HRP20230342TT patent/HRP20230342T1/hr unknown
- 2015-05-11 JP JP2017545520A patent/JP6637988B2/ja active Active
-
2017
- 2017-05-15 IL IL252291A patent/IL252291B/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| HK1244479A1 (zh) | 2018-08-10 |
| WO2016081029A1 (en) | 2016-05-26 |
| NZ732777A (en) | 2023-06-30 |
| JP2017535610A (ja) | 2017-11-30 |
| AU2015350561B2 (en) | 2020-01-30 |
| CA2968060A1 (en) | 2016-05-26 |
| IL252291B (en) | 2021-08-31 |
| DK3221293T3 (da) | 2023-04-03 |
| IL252291A0 (en) | 2017-07-31 |
| EP3221293A1 (en) | 2017-09-27 |
| EP3221293B1 (en) | 2023-03-01 |
| RS64078B1 (sr) | 2023-04-28 |
| HRP20230342T1 (hr) | 2023-06-09 |
| CN107207428A (zh) | 2017-09-26 |
| AU2015350561A1 (en) | 2017-07-06 |
| HUE062130T2 (hu) | 2023-09-28 |
| SI3221293T1 (sl) | 2023-05-31 |
| PL3221293T3 (pl) | 2023-07-10 |
| CA2968060C (en) | 2023-06-20 |
| LT3221293T (lt) | 2023-03-27 |
| JP6637988B2 (ja) | 2020-01-29 |
| KR20170095241A (ko) | 2017-08-22 |
| KR102380363B1 (ko) | 2022-03-29 |
| PT3221293T (pt) | 2023-03-16 |
| ES2946110T3 (es) | 2023-07-12 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| FI3221293T3 (fi) | Ionisoituva kationinen lipidi rna:n antamiseen | |
| EP4310075A3 (en) | Ionizable cationic lipid for rna delivery | |
| NZ720174A (en) | Ionizable cationic lipid for rna delivery | |
| EP3996716A4 (en) | COMPOUNDS FOR TREATMENT OF INFLUENCE VIRUS INFECTION | |
| JO3387B1 (ar) | مشتقات بيتولين | |
| MX377255B (es) | Lipidos y composiciones de lipidos para el suministro de agentes activos. | |
| CR20130023A (es) | Pirazolo[1,5-a]pirimidinas para tratamiento antiviral | |
| MX394360B (es) | Inhibidores de jak2 y alk2 y metodos para su uso. | |
| EA026667B9 (ru) | Фармацевтическая композиция для лечения вируса гепатита с | |
| MX2016005855A (es) | Compuestos de conjugados antisentido de apolipoproteina b (apob). | |
| MX361499B (es) | Baricitinib deuterado. | |
| FR3007969B1 (fr) | Distributeur d'une dose unitaire d'une substance active sous une forme galenique solide | |
| IN2015DN00598A (enExample) | ||
| MX2015012743A (es) | Compuesto de pirazol-amida y sus usos farmaceuticos. | |
| WO2014031872A3 (en) | Small molecule inhibitors for treating parasitic infections | |
| MX2015003594A (es) | Agente terapeutico o profilactico para sindrome de lisis tumoral. | |
| MX372963B (es) | Reordenamiento del virus de influenza b. | |
| EA201690911A1 (ru) | Соединения диметилбензойной кислоты | |
| IN2015DN00446A (enExample) | ||
| MX2015015562A (es) | Dihidropiridinona como inhibidores de monoacilglicerol aciltransferasa 2. | |
| MX379437B (es) | Derivados de hidrazina-bencilo sustituidos, como inhibidores de ppp1r15a y ppp1r15b, y el uso de los mismos para el tratamiento de trastorno de proteostasis. | |
| IN2014DN07996A (enExample) | ||
| SMT201500068B (it) | Nuovi analoghi di piperazina come antivirali ad ampio spettro per l'influenza | |
| WO2015068175A3 (en) | An improved process for the preparation of pazopanib or a pharmaceutically acceptable salt thereof | |
| AR091888A1 (es) | Compuestos de urea y su uso como inhibidores de enzimas |