JP6637988B2 - Rna送達のためのイオン化可能カチオン性脂質 - Google Patents
Rna送達のためのイオン化可能カチオン性脂質 Download PDFInfo
- Publication number
- JP6637988B2 JP6637988B2 JP2017545520A JP2017545520A JP6637988B2 JP 6637988 B2 JP6637988 B2 JP 6637988B2 JP 2017545520 A JP2017545520 A JP 2017545520A JP 2017545520 A JP2017545520 A JP 2017545520A JP 6637988 B2 JP6637988 B2 JP 6637988B2
- Authority
- JP
- Japan
- Prior art keywords
- carbons
- mol
- compound
- item
- lipid
- 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.)
- Active
Links
Classifications
-
- 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
- A61K9/127—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
- A61K9/0012—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
- 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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2017535610A JP2017535610A (ja) | 2017-11-30 |
| JP2017535610A5 JP2017535610A5 (OSRAM) | 2018-09-27 |
| JP6637988B2 true JP6637988B2 (ja) | 2020-01-29 |
Family
ID=56014367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017545520A Active JP6637988B2 (ja) | 2014-11-18 | 2015-05-11 | Rna送達のためのイオン化可能カチオン性脂質 |
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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 lípidos para el suministro de ARN |
| KR20190127277A (ko) * | 2018-05-04 | 2019-11-13 | 주식회사 삼양바이오팜 | mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법 |
| CN116172975B (zh) * | 2018-06-08 | 2025-03-28 | 富士胶片株式会社 | 脂质颗粒、包含该脂质颗粒的核酸药物以及核酸递送用组合物 |
| WO2020118115A1 (en) | 2018-12-06 | 2020-06-11 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| WO2020191103A1 (en) | 2019-03-19 | 2020-09-24 | Arcturus Therapeutics, Inc. | Method of making lipid-encapsulated rna nanoparticles |
| CA3143865A1 (en) | 2019-06-07 | 2020-12-10 | Fujifilm Corporation | Lipid composition |
| JP2022550435A (ja) | 2019-10-04 | 2022-12-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えaavの改善された治療的使用のための方法 |
| 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 | 世代生物公司 | 非病毒dna载体和其用于表达因子ix治疗剂的用途 |
| JP2023520763A (ja) | 2020-03-24 | 2023-05-19 | ジェネレーション バイオ カンパニー | ゴーシェ治療薬を発現するための非ウイルス性dnaベクター及びその使用 |
| 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 |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Dna分子の組成物、その作製方法、及びその使用方法 |
| AU2021340876A1 (en) | 2020-09-13 | 2023-04-13 | Arcturus Therapeutics, Inc. | Lipid nanoparticles encapsulation of large RNA |
| MX2023009345A (es) | 2021-02-10 | 2023-10-24 | Oncorus Inc | Compuestos, composiciones y metodos de uso de estos. |
| 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 |
| EP4329885A1 (en) | 2021-04-27 | 2024-03-06 | Generation Bio Co. | Non-viral dna vectors expressing therapeutic antibodies and uses thereof |
| WO2022232286A1 (en) | 2021-04-27 | 2022-11-03 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| AU2022336209B2 (en) | 2021-09-03 | 2025-10-16 | CureVac SE | Novel lipid nanoparticles for delivery of nucleic acids |
| US20250027108A1 (en) | 2021-10-29 | 2025-01-23 | 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 |
| US20250099614A1 (en) | 2022-01-28 | 2025-03-27 | 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 |
| JP2025509607A (ja) | 2022-03-14 | 2025-04-11 | ジェネレーション バイオ カンパニー | 異種プライムブーストワクチン組成物及び使用の方法 |
| CN119212720A (zh) | 2022-05-25 | 2024-12-27 | 库瑞瓦格欧洲股份公司 | 编码大肠杆菌FimH抗原性多肽的基于核酸的疫苗 |
| 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 |
| AU2023347320A1 (en) | 2022-09-23 | 2025-04-10 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2024089638A1 (en) | 2022-10-28 | 2024-05-02 | Glaxosmithkline Biologicals Sa | Nucleic acid based vaccine |
| TW202425959A (zh) | 2022-11-08 | 2024-07-01 | 美商歐納醫療公司 | 遞送多核苷酸的脂質及奈米顆粒組合物 |
| WO2024102762A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Lipids and lipid nanoparticle compositions for delivering polynucleotides |
| AU2023375897A1 (en) | 2022-11-08 | 2025-06-12 | Orna Therapeutics, Inc. | Circular rna compositions |
| WO2024119103A1 (en) | 2022-12-01 | 2024-06-06 | Generation Bio Co. | Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers |
| KR20250129819A (ko) | 2022-12-01 | 2025-08-29 | 제너레이션 바이오 컴퍼니 | 핵산, 이온화 가능한 지질, 스테롤, 지질에 고정된 중합체, 및 헬퍼 지질을 포함하는 지질 나노입자 및 이의 용도 |
| KR20250131271A (ko) | 2022-12-01 | 2025-09-02 | 제너레이션 바이오 컴퍼니 | 신규한 폴리글리세롤-접합된 지질 및 이를 포함하는 지질 나노입자 조성물 |
| KR20250114116A (ko) | 2022-12-01 | 2025-07-28 | 제너레이션 바이오 컴퍼니 | 세포 표적화를 위한 스텔스 지질 나노입자 조성물 |
| WO2024184500A1 (en) | 2023-03-08 | 2024-09-12 | CureVac SE | Novel lipid nanoparticle formulations for delivery of nucleic acids |
| 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 |
| WO2024249954A1 (en) | 2023-05-31 | 2024-12-05 | 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 |
| 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 |
Family Cites Families (10)
| 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 |
-
2015
- 2015-05-11 LT LTEPPCT/US2015/030218T patent/LT3221293T/lt unknown
- 2015-05-11 ES ES15724466T patent/ES2946110T3/es active Active
- 2015-05-11 HU HUE15724466A patent/HUE062130T2/hu unknown
- 2015-05-11 WO PCT/US2015/030218 patent/WO2016081029A1/en not_active Ceased
- 2015-05-11 PL PL15724466.6T patent/PL3221293T3/pl unknown
- 2015-05-11 EP EP15724466.6A patent/EP3221293B1/en active Active
- 2015-05-11 KR KR1020177016742A patent/KR102380363B1/ko active Active
- 2015-05-11 SI SI201531935T patent/SI3221293T1/sl unknown
- 2015-05-11 NZ NZ732777A patent/NZ732777A/en unknown
- 2015-05-11 CA CA2968060A patent/CA2968060C/en active Active
- 2015-05-11 AU AU2015350561A patent/AU2015350561B2/en active Active
- 2015-05-11 CN CN201580073670.9A patent/CN107207428A/zh active Pending
- 2015-05-11 FI FIEP15724466.6T patent/FI3221293T3/fi active
- 2015-05-11 HK HK18103903.0A patent/HK1244479A1/zh unknown
- 2015-05-11 PT PT157244666T patent/PT3221293T/pt unknown
- 2015-05-11 DK DK15724466.6T patent/DK3221293T3/da active
- 2015-05-11 RS RS20230231A patent/RS64078B1/sr unknown
- 2015-05-11 JP JP2017545520A patent/JP6637988B2/ja active Active
- 2015-05-11 HR HRP20230342TT patent/HRP20230342T1/hr unknown
-
2017
- 2017-05-15 IL IL252291A patent/IL252291B/en unknown
Also Published As
| 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 |
| 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 |
| 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 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7641308B2 (ja) | Rna送達のためのイオン化可能なカチオン性脂質 | |
| JP6637988B2 (ja) | Rna送達のためのイオン化可能カチオン性脂質 | |
| US9896413B2 (en) | Ionizable cationic lipid for RNA delivery | |
| US9580711B2 (en) | Lipid particles with asymmetric cationic lipids for RNA delivery | |
| WO2017117530A9 (en) | Ionizable cationic lipid | |
| HK40078649A (en) | Ionizable cationic lipid for rna delivery |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20180409 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20180409 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20180814 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20181204 |
|
| A977 | Report on retrieval |
Free format text: JAPANESE INTERMEDIATE CODE: A971007 Effective date: 20181206 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20190301 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20190507 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20190603 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20191202 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20191223 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 6637988 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |