KR20210105889A - 지질 나노입자 제제 - Google Patents

지질 나노입자 제제 Download PDF

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Publication number
KR20210105889A
KR20210105889A KR1020217017641A KR20217017641A KR20210105889A KR 20210105889 A KR20210105889 A KR 20210105889A KR 1020217017641 A KR1020217017641 A KR 1020217017641A KR 20217017641 A KR20217017641 A KR 20217017641A KR 20210105889 A KR20210105889 A KR 20210105889A
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KR
South Korea
Prior art keywords
lipid
nucleic acid
sirna
nucleotides
sequence
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.)
Pending
Application number
KR1020217017641A
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English (en)
Korean (ko)
Inventor
제임스 헤이스
아담 저지
키우 몽 램
론 랄프 팔머
페트라 슈라이너
Original Assignee
아뷰터스 바이오파마 코포레이션
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Publication of KR20210105889A publication Critical patent/KR20210105889A/ko
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K9/10—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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/505—Erythropoietin [EPO]
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Dispersion Chemistry (AREA)
  • Dermatology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
KR1020217017641A 2018-11-09 2019-11-08 지질 나노입자 제제 Pending KR20210105889A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862758088P 2018-11-09 2018-11-09
US62/758,088 2018-11-09
PCT/US2019/060582 WO2020097540A1 (en) 2018-11-09 2019-11-08 Lipid nanoparticle formulations

Publications (1)

Publication Number Publication Date
KR20210105889A true KR20210105889A (ko) 2021-08-27

Family

ID=70611206

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217017641A Pending KR20210105889A (ko) 2018-11-09 2019-11-08 지질 나노입자 제제

Country Status (14)

Country Link
US (1) US12251470B2 (https=)
EP (1) EP3877521A4 (https=)
JP (1) JP2022506956A (https=)
KR (1) KR20210105889A (https=)
CN (1) CN113286882A (https=)
AU (1) AU2019376161B2 (https=)
BR (1) BR112021008982A2 (https=)
EA (1) EA202191313A1 (https=)
IL (1) IL282995B1 (https=)
MX (1) MX2021005389A (https=)
SG (1) SG11202104807YA (https=)
UA (1) UA130104C2 (https=)
WO (1) WO2020097540A1 (https=)
ZA (1) ZA202103233B (https=)

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KR20230145791A (ko) 2022-04-11 2023-10-18 연세대학교 원주산학협력단 mRNA 백신을 위한 양이온성 지질 나노입자

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AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
KR20230011913A (ko) 2020-03-04 2023-01-25 버브 테라퓨틱스, 인크. 표적화된 rna 전달을 위한 조성물 및 방법
CN116096886A (zh) 2020-03-11 2023-05-09 欧米茄治疗公司 用于调节叉头框p3(foxp3)基因表达的组合物和方法
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
EP4132478A1 (en) 2020-04-09 2023-02-15 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
JP2023546175A (ja) 2020-10-14 2023-11-01 ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド 脂質ナノ粒子製造の方法及びそれに由来する組成物
WO2022099003A1 (en) 2020-11-06 2022-05-12 Sanofi Lipid nanoparticles for delivering mrna vaccines
CN113509542A (zh) * 2021-04-20 2021-10-19 嘉晨西海(杭州)生物技术有限公司 一种基于mRNA的表达白介素12针对肿瘤的药物及其制备方法
EP4367242A2 (en) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
CA3226019A1 (en) 2021-07-20 2023-01-26 Ags Therapeutics Sas Extracellular vesicles from microalgae, their preparation, and uses
JP2024530647A (ja) 2021-08-03 2024-08-23 ヴァーヴ・セラピューティクス，インコーポレーテッド 標的ｒｎａ送達のための組成物および方法
CA3132191A1 (en) * 2021-09-28 2023-03-28 Providence Therapeutics Holdings Inc. Compositions and methods for the prevention and/or treatment of covid-19
KR20230053998A (ko) 2021-10-15 2023-04-24 (주)지노믹트리 mRNA 발현을 위한 유전자 구조체
MX2024005462A (es) * 2021-11-05 2024-05-22 Sanofi Sa Vacuna de arn del virus respiratorio sincitial.
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
CN118804743A (zh) * 2022-03-04 2024-10-18 益杰立科(上海)生物科技有限公司 靶向递送系统和方法
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
US20250302747A1 (en) * 2022-06-08 2025-10-02 Academia Sinica Lipids, nanoparticles comprising the same and uses thereof
CN121038801A (zh) 2022-10-24 2025-11-28 Ags治疗简易股份公司 来自微藻的细胞外囊泡、它们在鼻内施用时的生物分布及其用途
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
EP4722368A1 (en) 2023-05-26 2026-04-08 Samsung Biologics Co., Ltd. Novel nucleic acid construct comprising poly(a) tail having secondary or tertiary structure and uses thereof
KR20250018367A (ko) 2023-07-28 2025-02-05 한국과학기술원 히스톤 코드 리더 도메인을 포함하는 폴리펩티드 및 이의 용도
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WO2025210520A1 (en) * 2024-04-04 2025-10-09 Genevant Sciences Gmbh Lipid nanoparticles for inducing an immunological response
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CN121287885A (zh) * 2025-12-11 2026-01-09 中国人民解放军军事科学院军事医学研究院 一种HGF mRNA脂质纳米颗粒（LNP）及其应用

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230145791A (ko) 2022-04-11 2023-10-18 연세대학교 원주산학협력단 mRNA 백신을 위한 양이온성 지질 나노입자
KR20250056147A (ko) 2022-04-11 2025-04-25 연세대학교 원주산학협력단 mRNA 백신을 위한 양이온성 지질 나노입자

Also Published As

Publication number Publication date
EP3877521A1 (en) 2021-09-15
MX2021005389A (es) 2021-07-21
EA202191313A1 (ru) 2022-01-26
WO2020097540A1 (en) 2020-05-14
JP2022506956A (ja) 2022-01-17
SG11202104807YA (en) 2021-06-29
US12251470B2 (en) 2025-03-18
ZA202103233B (en) 2025-08-27
CN113286882A (zh) 2021-08-20
IL282995A (en) 2021-06-30
IL282995B1 (en) 2026-03-01
UA130104C2 (uk) 2025-11-12
CA3119071A1 (en) 2020-05-14
AU2019376161B2 (en) 2025-04-24
BR112021008982A2 (pt) 2021-08-10
AU2019376161A1 (en) 2021-06-03
US20220000778A1 (en) 2022-01-06
EP3877521A4 (en) 2022-07-27

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