CR20230536A - Composiciones de nanopartículas lipídicas - Google Patents
Composiciones de nanopartículas lipídicasInfo
- Publication number
- CR20230536A CR20230536A CR20230536A CR20230536A CR20230536A CR 20230536 A CR20230536 A CR 20230536A CR 20230536 A CR20230536 A CR 20230536A CR 20230536 A CR20230536 A CR 20230536A CR 20230536 A CR20230536 A CR 20230536A
- Authority
- CR
- Costa Rica
- Prior art keywords
- lipids
- compositions
- methods
- biologically active
- delivery
- Prior art date
Links
- 150000002632 lipids Chemical group 0.000 title abstract 6
- 239000000203 mixture Substances 0.000 title abstract 3
- 239000002105 nanoparticle Substances 0.000 title abstract 2
- 239000013543 active substance Substances 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 3
- 238000010362 genome editing Methods 0.000 abstract 1
- 230000007935 neutral effect Effects 0.000 abstract 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
- A61K9/48—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
-
- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
-
- 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
- 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
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Nanotechnology (AREA)
- Mycology (AREA)
- Optics & Photonics (AREA)
- Cell Biology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Dispersion Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Manufacturing Of Micro-Capsules (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163176227P | 2021-04-17 | 2021-04-17 | |
| US202163254948P | 2021-10-12 | 2021-10-12 | |
| US202163274153P | 2021-11-01 | 2021-11-01 | |
| US202263316568P | 2022-03-04 | 2022-03-04 | |
| PCT/US2022/025076 WO2022221697A1 (en) | 2021-04-17 | 2022-04-15 | Lipid nanoparticle compositions |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CR20230536A true CR20230536A (es) | 2024-03-08 |
Family
ID=81580499
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CR20230536A CR20230536A (es) | 2021-04-17 | 2022-04-15 | Composiciones de nanopartículas lipídicas |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20250090471A1 (https=) |
| EP (1) | EP4322920A1 (https=) |
| JP (1) | JP2024515650A (https=) |
| KR (1) | KR20240017792A (https=) |
| AU (1) | AU2022257050A1 (https=) |
| BR (1) | BR112023021477A2 (https=) |
| CA (1) | CA3216877A1 (https=) |
| CL (1) | CL2023003077A1 (https=) |
| CO (1) | CO2023015485A2 (https=) |
| CR (1) | CR20230536A (https=) |
| IL (1) | IL307738A (https=) |
| MX (1) | MX2023012235A (https=) |
| TW (1) | TW202308597A (https=) |
| WO (1) | WO2022221697A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024536406A (ja) * | 2021-10-08 | 2024-10-04 | スージョウ・アボジェン・バイオサイエンシズ・カンパニー・リミテッド | 脂質化合物及び脂質ナノ粒子組成物 |
| EP4638783A2 (en) | 2022-12-22 | 2025-10-29 | Intellia Therapeutics, Inc. | Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies |
| WO2024199282A1 (zh) * | 2023-03-28 | 2024-10-03 | 苏州艾博生物科技有限公司 | 脂质化合物和脂质纳米颗粒组合物 |
| AU2024279278A1 (en) | 2023-05-31 | 2025-12-18 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025049432A1 (en) * | 2023-08-28 | 2025-03-06 | Intellia Therapeutics, Inc. | Methods for editing hla-a in cells pre-screened for the absence of one or both alleles of hla-h*01 |
| WO2025049481A1 (en) * | 2023-08-28 | 2025-03-06 | Intellia Therapeutics, Inc. | Methods of editing an hla-a gene in vitro |
| WO2025064401A1 (en) | 2023-09-18 | 2025-03-27 | Intellia Therapeutics, Inc. | Nuclease resistant single stranded dna product for non-viral delivery to a cell and methods of production thereof |
| WO2025064396A1 (en) | 2023-09-18 | 2025-03-27 | Intellia Therapeutics, Inc. | Nuclease resistant double stranded dna product for non-viral delivery to a cell and methods of production thereof |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025137301A1 (en) * | 2023-12-20 | 2025-06-26 | Intellia Therapeutics, Inc. | Methods for rapid engineering of cells |
| WO2025240946A1 (en) | 2024-05-17 | 2025-11-20 | Intellia Therapeutics, Inc. | Lipid nanoparticles and lipid nanoparticle compositions |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030083272A1 (en) | 1997-09-19 | 2003-05-01 | Lahive & Cockfield, Llp | Sense mrna therapy |
| US20060051405A1 (en) | 2004-07-19 | 2006-03-09 | Protiva Biotherapeutics, Inc. | Compositions for the delivery of therapeutic agents and uses thereof |
| CA3009891C (en) | 2009-12-23 | 2020-09-15 | Novartis Ag | Lipids, lipid compositions, and methods of using them |
| EP2526199A4 (en) | 2010-01-22 | 2013-08-07 | Scripps Research Inst | PROCESS FOR PRODUCING ZINC FINGERUCLEASES WITH CHANGED EFFECT |
| CN103668470B (zh) | 2012-09-12 | 2015-07-29 | 上海斯丹赛生物技术有限公司 | 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法 |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| CN107879960B (zh) | 2013-03-08 | 2021-06-22 | 诺华股份有限公司 | 用于传递活性成分的脂质和脂质组合物 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| JP6948313B6 (ja) | 2015-09-17 | 2022-01-14 | モデルナティエックス インコーポレイテッド | 治療剤の細胞内送達のための化合物および組成物 |
| WO2018073393A2 (en) | 2016-10-19 | 2018-04-26 | Cellectis | Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy |
| WO2019147805A2 (en) | 2018-01-26 | 2019-08-01 | The Board Of Trustees Of The Leland Stanford Junior University | Regulatory t cells targeted with chimeric antigen receptors |
| AU2020248337A1 (en) * | 2019-03-28 | 2021-11-04 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof |
| CN114127044A (zh) * | 2019-04-25 | 2022-03-01 | 英特利亚治疗股份有限公司 | 可电离的胺类脂质和脂质纳米颗粒 |
| CA3145913A1 (en) * | 2019-07-29 | 2021-02-04 | James Everett Dahlman | Nanomaterials containing constrained lipids and uses thereof |
-
2022
- 2022-04-15 EP EP22721589.4A patent/EP4322920A1/en active Pending
- 2022-04-15 KR KR1020237039429A patent/KR20240017792A/ko active Pending
- 2022-04-15 MX MX2023012235A patent/MX2023012235A/es unknown
- 2022-04-15 IL IL307738A patent/IL307738A/en unknown
- 2022-04-15 US US18/287,239 patent/US20250090471A1/en active Pending
- 2022-04-15 WO PCT/US2022/025076 patent/WO2022221697A1/en not_active Ceased
- 2022-04-15 CR CR20230536A patent/CR20230536A/es unknown
- 2022-04-15 CA CA3216877A patent/CA3216877A1/en active Pending
- 2022-04-15 JP JP2023563210A patent/JP2024515650A/ja active Pending
- 2022-04-15 AU AU2022257050A patent/AU2022257050A1/en active Pending
- 2022-04-15 TW TW111114501A patent/TW202308597A/zh unknown
- 2022-04-15 BR BR112023021477A patent/BR112023021477A2/pt not_active Application Discontinuation
-
2023
- 2023-10-16 CL CL2023003077A patent/CL2023003077A1/es unknown
- 2023-11-16 CO CONC2023/0015485A patent/CO2023015485A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20240017792A (ko) | 2024-02-08 |
| JP2024515650A (ja) | 2024-04-10 |
| EP4322920A1 (en) | 2024-02-21 |
| BR112023021477A2 (pt) | 2024-01-30 |
| CL2023003077A1 (es) | 2024-04-12 |
| WO2022221697A1 (en) | 2022-10-20 |
| AU2022257050A1 (en) | 2023-11-30 |
| TW202308597A (zh) | 2023-03-01 |
| MX2023012235A (es) | 2024-01-23 |
| IL307738A (en) | 2023-12-01 |
| US20250090471A1 (en) | 2025-03-20 |
| CO2023015485A2 (es) | 2025-08-19 |
| CA3216877A1 (en) | 2022-10-20 |
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