IL291038A - Preparations of lipid nanoparticles containing closed-end DNA and cleavable lipids and methods of using them - Google Patents
Preparations of lipid nanoparticles containing closed-end DNA and cleavable lipids and methods of using themInfo
- Publication number
- IL291038A IL291038A IL291038A IL29103822A IL291038A IL 291038 A IL291038 A IL 291038A IL 291038 A IL291038 A IL 291038A IL 29103822 A IL29103822 A IL 29103822A IL 291038 A IL291038 A IL 291038A
- Authority
- IL
- Israel
- Prior art keywords
- closed
- methods
- lipid nanoparticle
- nanoparticle compositions
- ended dna
- Prior art date
Links
- 150000002632 lipids Chemical class 0.000 title 2
- 239000000203 mixture Substances 0.000 title 1
- 239000002105 nanoparticle Substances 0.000 title 1
Classifications
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/575—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7048—Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin, digitoxin or digoxin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
-
- 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
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
- C12N2710/14141—Use of virus, viral particle or viral elements as a vector
- C12N2710/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Dispersion Chemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962896980P | 2019-09-06 | 2019-09-06 | |
| US201962910720P | 2019-10-04 | 2019-10-04 | |
| US201962940104P | 2019-11-25 | 2019-11-25 | |
| PCT/US2020/049266 WO2021046265A1 (en) | 2019-09-06 | 2020-09-03 | Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL291038A true IL291038A (en) | 2022-05-01 |
Family
ID=74853052
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL291038A IL291038A (en) | 2019-09-06 | 2022-03-01 | Preparations of lipid nanoparticles containing closed-end DNA and cleavable lipids and methods of using them |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220280427A1 (https=) |
| EP (1) | EP4025196A4 (https=) |
| JP (1) | JP7700101B2 (https=) |
| CN (1) | CN114929205B (https=) |
| AU (1) | AU2020342668B2 (https=) |
| CA (1) | CA3150452A1 (https=) |
| IL (1) | IL291038A (https=) |
| WO (1) | WO2021046265A1 (https=) |
Families Citing this family (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021060440A1 (ja) * | 2019-09-26 | 2021-04-01 | 日油株式会社 | 脂質ナノ粒子の凍結乾燥組成物 |
| IL296763B2 (en) | 2020-03-27 | 2026-04-01 | Generation Bio Co | New lipids and their nanoparticle compositions |
| US20230181764A1 (en) * | 2020-05-18 | 2023-06-15 | Generation Bio Co. | Novel lipids and nanoparticle compositions thereof |
| MX2023000806A (es) * | 2020-07-17 | 2023-04-11 | Generation Bio Co | Métodos para encapsular polinucleótidos en tamaños reducidos de nanopartículas lipídicas y formulación novedosa de estos. |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| CZ310613B6 (cs) | 2020-09-23 | 2026-01-28 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Lipidoidy pro transfekci nukleových kyselin a jejich použití |
| IL303195A (en) | 2020-11-25 | 2023-07-01 | Akagera Medicines Inc | Lipid nanoparticles for delivery of nucleic acids and related methods of use |
| CA3221454A1 (en) * | 2021-06-07 | 2022-12-15 | Michelle LEBLANC | Apoe and apob modified lipid nanoparticle compositions and uses thereof |
| WO2022266032A1 (en) * | 2021-06-14 | 2022-12-22 | Generation Bio Co. | Cationic lipids and compositions thereof |
| KR20240038705A (ko) * | 2021-06-22 | 2024-03-25 | 인텔리아 테라퓨틱스, 인크. | 간 유전자의 생체 내 편집 방법 |
| CZ310443B6 (cs) | 2021-07-19 | 2025-06-25 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití |
| US12251471B2 (en) * | 2021-07-26 | 2025-03-18 | Oregon State University | Inhalable therapeutics |
| WO2023054242A1 (ja) * | 2021-09-30 | 2023-04-06 | 日油株式会社 | 核酸をリンパ管内皮細胞に送達するために用いられる脂質ナノ粒子 |
| CN114432502B (zh) * | 2021-12-31 | 2023-02-03 | 中山大学附属第八医院（深圳福田） | 一种负载op3-4多肽、抗cxcl9抗体的水凝胶及其制备方法与应用 |
| CN118973614A (zh) * | 2022-03-28 | 2024-11-15 | 日油株式会社 | 用于向脾脏组织递送核酸的脂质纳米粒子以及使用其向脾脏组织递送核酸的方法 |
| EP4501361A4 (en) * | 2022-03-28 | 2026-01-28 | Nof Corp | Lipid nanoparticles for delivering nucleic acid to peripheral blood mononuclear cells, and a method for delivering nucleic acid to peripheral blood mononuclear cells using them |
| EP4531819A2 (en) | 2022-05-25 | 2025-04-09 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids and methods of use thereof |
| WO2024112943A1 (en) * | 2022-11-22 | 2024-05-30 | University Of Maryland, Baltimore | Activated nanotherapy for sickle cell disease |
| EP4628496A1 (en) | 2022-12-29 | 2025-10-08 | Jenkem Technology Co., Ltd. (Liaoning) | Steroid-cationic lipid compound and use thereof |
| US12311033B2 (en) | 2023-05-31 | 2025-05-27 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025188836A1 (en) * | 2024-03-05 | 2025-09-12 | Saliogen Therapeutics, Inc. | Compositions and methods of using gene coding for treating ocular disease |
| WO2025213139A1 (en) * | 2024-04-04 | 2025-10-09 | The General Hospital Corporation | Enpp1 gene therapy for the treatment of vascular disease |
| WO2026003582A2 (en) | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
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| US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
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| CA2289702C (en) | 1997-05-14 | 2008-02-19 | Inex Pharmaceuticals Corp. | High efficiency encapsulation of charged therapeutic agents in lipid vesicles |
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| JP2020532981A (ja) * | 2017-09-08 | 2020-11-19 | ジェネレーション バイオ カンパニー | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
| AU2018330208B2 (en) | 2017-09-08 | 2025-04-17 | Generation Bio Co. | Lipid nanoparticle formulations of non-viral, capsid-free DNA vectors |
| MA51842A (fr) * | 2018-02-14 | 2020-12-23 | Generation Bio Co | Vecteurs d'adn non viraux et utilisations associées pour la production d'anticorps et de protéines de fusion |
| WO2019188867A1 (ja) * | 2018-03-27 | 2019-10-03 | 日油株式会社 | 細胞内動態を改善した新規カチオン性脂質 |
-
2020
- 2020-09-03 JP JP2022514708A patent/JP7700101B2/ja active Active
- 2020-09-03 EP EP20860233.4A patent/EP4025196A4/en active Pending
- 2020-09-03 WO PCT/US2020/049266 patent/WO2021046265A1/en not_active Ceased
- 2020-09-03 CA CA3150452A patent/CA3150452A1/en active Pending
- 2020-09-03 CN CN202080076963.3A patent/CN114929205B/zh active Active
- 2020-09-03 AU AU2020342668A patent/AU2020342668B2/en active Active
- 2020-09-03 US US17/632,262 patent/US20220280427A1/en active Pending
-
2022
- 2022-03-01 IL IL291038A patent/IL291038A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2020342668B2 (en) | 2025-10-09 |
| JP2022546597A (ja) | 2022-11-04 |
| EP4025196A4 (en) | 2023-07-12 |
| US20220280427A1 (en) | 2022-09-08 |
| CN114929205A (zh) | 2022-08-19 |
| CA3150452A1 (en) | 2021-03-11 |
| JP7700101B2 (ja) | 2025-06-30 |
| AU2020342668A1 (en) | 2022-03-03 |
| WO2021046265A1 (en) | 2021-03-11 |
| CN114929205B (zh) | 2024-09-03 |
| EP4025196A1 (en) | 2022-07-13 |
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