CN116437964A - 用于将多核苷酸封装成减小尺寸的脂质纳米颗粒以及其新型调配物的方法 - Google Patents

用于将多核苷酸封装成减小尺寸的脂质纳米颗粒以及其新型调配物的方法 Download PDF

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CN116437964A
CN116437964A CN202180063144.XA CN202180063144A CN116437964A CN 116437964 A CN116437964 A CN 116437964A CN 202180063144 A CN202180063144 A CN 202180063144A CN 116437964 A CN116437964 A CN 116437964A
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lipid
pharmaceutical composition
alkyl
itr
cedna
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N·加拉格
M·G·斯坦顿
G·范斯坦
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Generational Biology Co
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Generational Biology Co
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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
    • 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
    • C12N15/87—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
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • 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
    • 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
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202180063144.XA 2020-07-17 2021-07-16 用于将多核苷酸封装成减小尺寸的脂质纳米颗粒以及其新型调配物的方法 Pending CN116437964A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063053274P 2020-07-17 2020-07-17
US63/053,274 2020-07-17
US202163194620P 2021-05-28 2021-05-28
US63/194,620 2021-05-28
PCT/US2021/042033 WO2022016089A2 (en) 2020-07-17 2021-07-16 Methods for encapsulating polynucleotides into reduced sizes of lipid nanoparticles and novel formulation thereof

Publications (1)

Publication Number Publication Date
CN116437964A true CN116437964A (zh) 2023-07-14

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CN202180063144.XA Pending CN116437964A (zh) 2020-07-17 2021-07-16 用于将多核苷酸封装成减小尺寸的脂质纳米颗粒以及其新型调配物的方法

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Country Link
US (1) US20230320993A1 (https=)
EP (1) EP4181948A4 (https=)
JP (1) JP2023534043A (https=)
KR (1) KR20230052895A (https=)
CN (1) CN116437964A (https=)
AU (1) AU2021307952A1 (https=)
CA (1) CA3186033A1 (https=)
IL (1) IL299896A (https=)
MX (1) MX2023000806A (https=)
WO (1) WO2022016089A2 (https=)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11752213B2 (en) 2015-12-21 2023-09-12 Duke University Surfaces having reduced non-specific binding and antigenicity
EP3515928B1 (en) 2016-09-23 2026-01-28 Duke University Unstructured non-repetitive polypeptides having lcst behavior
WO2019006374A1 (en) 2017-06-30 2019-01-03 Duke University ORDER AND DISORDER AS A DESIGN PRINCIPLE FOR STIMULI-SENSITIVE BIOPOLYMER NETWORKS
US12296018B2 (en) 2018-01-26 2025-05-13 Duke University Albumin binding peptide-drug (AlBiPeD) conjugates and methods of making and using same
EP3788343B1 (en) 2018-04-30 2024-03-27 Duke University Stimuli-responsive peg-like polymer-based drug delivery platform
WO2020051541A1 (en) 2018-09-07 2020-03-12 Duke University Nanoparticulate drug delivery systems
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
US11512314B2 (en) 2019-07-12 2022-11-29 Duke University Amphiphilic polynucleotides
IL296763B2 (en) 2020-03-27 2026-04-01 Generation Bio Co New lipids and their nanoparticle compositions
KR20230172570A (ko) 2021-04-20 2023-12-22 제너레이션 바이오 컴퍼니 양이온성 지질 및 이의 조성물
US12508225B2 (en) 2021-05-28 2025-12-30 Beijing Tricisionbio Therapeutics Inc. Lipid compound and use thereof in delivery of nucleic acid
WO2022266032A1 (en) * 2021-06-14 2022-12-22 Generation Bio Co. Cationic lipids and compositions thereof
US20250281596A1 (en) * 2021-09-28 2025-09-11 Seqirus Inc. Ionizable cationic compound
CN118201605A (zh) * 2021-10-25 2024-06-14 杜克大学 基于poegma的脂质纳米粒子
WO2023190166A1 (ja) * 2022-03-28 2023-10-05 日油株式会社 ジスルフィド結合を有するカチオン性脂質、これを含む脂質膜構造体、これらのいずれかを含む核酸導入剤及び医薬品組成物、核酸を細胞又は標的細胞内へ導入する方法、及び細胞医薬品の製造方法
CN114685784B (zh) * 2022-04-26 2023-09-15 北京清科胜因生物科技有限公司 一种用于核酸递送的聚(2-噁唑啉)脂质与脂质纳米颗粒及应用
EP4536197A1 (en) * 2022-06-07 2025-04-16 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
CN115105584B (zh) * 2022-06-28 2023-03-31 长春生物制品研究所有限责任公司 一种卡式瓶多剂量笔式注射组合包装的干扰素注射液
WO2024037577A1 (en) * 2022-08-18 2024-02-22 Suzhou Abogen Biosciences Co., Ltd. Composition of lipid nanoparticles
EP4594299A1 (en) * 2022-09-27 2025-08-06 Reinvigoron TheraTech, Inc. Compounds with cleavable disulfide moieties
CN120091992A (zh) * 2022-11-08 2025-06-03 三养控股公司 阳离子脂质及其制备方法
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
EP4704855A1 (en) * 2023-04-26 2026-03-11 Saliogen Therapeutics, Inc. Lipid nanoparticle (lnp) formulations
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025043146A2 (en) * 2023-08-24 2025-02-27 Molecular Axiom Inc. Lipid nanoparticle compositions and methods of using same
WO2025064850A1 (en) 2023-09-22 2025-03-27 BioNTech SE Rna constructs with n-terminal degrons to enhance an immune response
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
GB202416789D0 (en) 2023-11-15 2025-01-01 BioNTech SE SARS-COV-2 immunogenic compositions
WO2025149492A1 (en) 2024-01-08 2025-07-17 BioNTech SE Rna encoding an immune inhibitory il-1 family member
WO2025166238A1 (en) * 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025178465A1 (ko) * 2024-02-19 2025-08-28 주식회사 메디치바이오 신규한 이온화 지질 및 이를 이용한 지질 나노입자 조성물
WO2025230906A1 (en) * 2024-04-29 2025-11-06 Saliogen Therapeutics, Inc. Lipid nanoparticle (lnp) formulations
WO2025237830A1 (en) * 2024-05-15 2025-11-20 Evonik Operations Gmbh Polyoxyalkylene-1,2-di(alkyloxy)propanoxy-carbonylaminopropyl compounds, wherein the polyoxyalkylene is a poly(ethylene oxide) having c1 to c3-alkyloxymethyl side chains
WO2025255027A1 (en) * 2024-06-03 2025-12-11 Flagship Labs 114, Inc. Lipid nanoparticles for topical delivery
WO2026047603A1 (en) 2024-08-28 2026-03-05 BioNTech SE Sars-cov-2 immunogenic compositions
WO2026057625A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having a chap domain with engineered active site
WO2026057626A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having multiple enzymatically active domains
WO2026068864A1 (en) 2024-09-30 2026-04-02 BioNTech SE Engineered ch1 and cl domains for the prevention of chain mispairing
CN121421963A (zh) * 2026-01-04 2026-01-30 广州隽沐生物科技股份有限公司 一种用于难溶性活性经皮黏膜给药的装配材料及制备方法

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100015218A1 (en) * 2007-02-16 2010-01-21 Vasant Jadhav Compositions and methods for potentiated activity of biologically active molecules
JP6093710B2 (ja) * 2011-11-18 2017-03-08 日油株式会社 細胞内動態を改善したカチオン性脂質
HK1206644A1 (en) * 2012-03-29 2016-01-15 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
EP4286012A3 (en) * 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US11395798B2 (en) * 2015-10-08 2022-07-26 Nof Corporation O/W type emulsion
WO2017189870A1 (en) * 2016-04-27 2017-11-02 Massachusetts Institute Of Technology Stable nanoscale nucleic acid assemblies and methods thereof
US11583504B2 (en) * 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
AU2018330208B2 (en) * 2017-09-08 2025-04-17 Generation Bio Co. Lipid nanoparticle formulations of non-viral, capsid-free DNA vectors
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
JP7700101B2 (ja) * 2019-09-06 2025-06-30 ジェネレーション バイオ カンパニー 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法
AU2020385378A1 (en) * 2019-11-22 2022-04-07 Generation Bio Co. Ionizable lipids and nanoparticle compositions thereof
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

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EP4181948A2 (en) 2023-05-24
WO2022016089A2 (en) 2022-01-20
KR20230052895A (ko) 2023-04-20
CA3186033A1 (en) 2022-01-20
AU2021307952A1 (en) 2023-03-02
IL299896A (en) 2023-03-01
JP2023534043A (ja) 2023-08-07
WO2022016089A3 (en) 2022-03-03
US20230320993A1 (en) 2023-10-12
EP4181948A4 (en) 2024-12-04
MX2023000806A (es) 2023-04-11

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