IL296763B2 - New lipids and their nanoparticle compositions - Google Patents

New lipids and their nanoparticle compositions

Info

Publication number
IL296763B2
IL296763B2 IL296763A IL29676322A IL296763B2 IL 296763 B2 IL296763 B2 IL 296763B2 IL 296763 A IL296763 A IL 296763A IL 29676322 A IL29676322 A IL 29676322A IL 296763 B2 IL296763 B2 IL 296763B2
Authority
IL
Israel
Prior art keywords
nanoparticle compositions
novel lipids
lipids
novel
nanoparticle
Prior art date
Application number
IL296763A
Other languages
English (en)
Hebrew (he)
Other versions
IL296763A (en
IL296763B1 (en
Original Assignee
Generation Bio Co
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Generation Bio Co filed Critical Generation Bio Co
Publication of IL296763A publication Critical patent/IL296763A/en
Publication of IL296763B1 publication Critical patent/IL296763B1/en
Publication of IL296763B2 publication Critical patent/IL296763B2/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D211/00Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/08Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
    • C07D211/18Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D211/20Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms
    • C07D211/22Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms by oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Obesity (AREA)
  • Biochemistry (AREA)
  • Optics & Photonics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nanotechnology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (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)
IL296763A 2020-03-27 2021-03-26 New lipids and their nanoparticle compositions IL296763B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063000990P 2020-03-27 2020-03-27
PCT/US2021/024413 WO2021195529A2 (en) 2020-03-27 2021-03-26 Novel lipids and nanoparticle compositions thereof

Publications (3)

Publication Number Publication Date
IL296763A IL296763A (en) 2022-11-01
IL296763B1 IL296763B1 (en) 2025-12-01
IL296763B2 true IL296763B2 (en) 2026-04-01

Family

ID=77890624

Family Applications (1)

Application Number Title Priority Date Filing Date
IL296763A IL296763B2 (en) 2020-03-27 2021-03-26 New lipids and their nanoparticle compositions

Country Status (11)

Country Link
US (3) US12534435B2 (enExample)
EP (1) EP4125821A4 (enExample)
JP (2) JP7746280B2 (enExample)
KR (1) KR20220160647A (enExample)
CN (1) CN115916163A (enExample)
AU (1) AU2021241696A1 (enExample)
BR (1) BR112022019369A2 (enExample)
CA (1) CA3173126A1 (enExample)
IL (1) IL296763B2 (enExample)
MX (1) MX2022011988A (enExample)
WO (1) WO2021195529A2 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
JP7746280B2 (ja) 2020-03-27 2025-09-30 ジェネレーション バイオ カンパニー 新規脂質及びそれらのナノ粒子組成物
AU2021307952A1 (en) * 2020-07-17 2023-03-02 Generation Bio Co. Methods for encapsulating polynucleotides into reduced sizes of Lipid nanoparticles and novel formulation thereof
WO2022115645A1 (en) 2020-11-25 2022-06-02 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
CA3234321A1 (en) * 2021-09-30 2023-04-06 Nof Corporation Lipid nanoparticles used to transport nucleic acids into lymphatic endothelium cells
EP4452942A1 (en) * 2021-12-20 2024-10-30 Beam Therapeutics Inc. Nanomaterials comprising disulfides
WO2023190175A1 (ja) 2022-03-28 2023-10-05 日油株式会社 末梢血単核細胞へ核酸を送達するための脂質ナノ粒子およびこれを用いて末梢血単核細胞へ核酸を送達する方法
US20260115316A1 (en) 2022-03-28 2026-04-30 Nof Corporation Lipid nanoparticles for delivering nucleic acid to splenic tissue, and method for delivering nucleic acid to splenic tissue using same
JPWO2023190164A1 (enExample) * 2022-03-28 2023-10-05
WO2023190170A1 (ja) 2022-03-28 2023-10-05 日油株式会社 核酸内封脂質ナノ粒子の製造方法及びこれを含む医薬品組成物の製造方法、並びに、核酸を細胞内又は標的細胞内に導入する方法
CA3256897A1 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for the administration of nucleic acids and their methods of use
CN119604276A (zh) 2022-07-15 2025-03-11 日油株式会社 脂质纳米粒子及其制造方法、封入有核酸的脂质纳米粒子及其制造方法以及使用封入有核酸的脂质纳米粒子赋予生物体获得性免疫的方法
CA3262131A1 (en) 2022-07-15 2025-06-13 Nof Corporation FREEZE-DRIED COMPOSITION OF LIPID NANOPARTICLES CHARGED WITH NUCLEIC ACID
EP4683905A1 (en) * 2023-03-22 2026-01-28 Nanovation Therapeutics Inc. Ionizable anionic lipids
CN120957730A (zh) 2023-03-29 2025-11-14 国立大学法人东北大学 核酸封装脂质纳米粒子的制造方法
CN116082184B (zh) * 2023-04-12 2023-06-30 山东大学 基于环己二胺的可电离脂质、脂质纳米颗粒及其制备方法与应用
AU2024279278A1 (en) 2023-05-31 2025-12-18 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
CN121263400A (zh) * 2023-12-15 2026-01-02 厦门赛诺邦格生物科技股份有限公司 一种含二硫键的可降解阳离子脂质及其应用
CN117964577B (zh) * 2024-03-29 2024-06-21 天津全和诚生物技术有限公司 阳离子脂质化合物、其制备方法、包含其的组合物及应用
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026070952A1 (ja) * 2024-09-27 2026-04-02 日油株式会社 ジスルフィド結合を有する化合物およびその製造方法

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5928906A (en) 1996-05-09 1999-07-27 Sequenom, Inc. Process for direct sequencing during template amplification
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
CN101267805A (zh) 2005-07-27 2008-09-17 普洛体维生物治疗公司 制造脂质体的系统和方法
CA2689042A1 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US8883211B2 (en) 2008-07-10 2014-11-11 Serina Therapeutics, Inc. Polyoxazolines with inert terminating groups, polyoxazolines prepared from protected initiating groups and related compounds
US20130037977A1 (en) 2010-04-08 2013-02-14 Paul A. Burke Preparation of Lipid Nanoparticles
US20130156845A1 (en) 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012024526A2 (en) 2010-08-20 2012-02-23 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
WO2012046265A1 (ja) * 2010-10-05 2012-04-12 トヨタ自動車株式会社 推定装置および推定方法
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP3485875A1 (en) 2011-11-04 2019-05-22 Nitto Denko Corporation Single use system for sterelily producing lipid-nucleic acid particles
WO2013073480A1 (ja) 2011-11-18 2013-05-23 日油株式会社 細胞内動態を改善したカチオン性脂質
TW201808342A (zh) 2012-05-02 2018-03-16 喜納製藥公司 包含四galnac之新穎結合物及傳送寡核苷酸之方法
US10426737B2 (en) 2013-12-19 2019-10-01 Novartis Ag Lipids and lipid compositions for the delivery of active agents
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
BR112018003910B1 (pt) 2015-11-16 2022-03-22 F. Hoffmann-La Roche Ag Derivado de fosforamidita galnac, processo para a preparação de um derivado de fosforamidita galnac de fórmula i e uso dos derivados de fosforamidita galnac de fórmula i
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
CN111902397B (zh) 2018-03-27 2024-04-12 日油株式会社 改进了细胞内动力学的阳离子脂质
JP7075256B2 (ja) * 2018-03-28 2022-05-25 三菱重工業株式会社 配管の余寿命評価方法
WO2021046265A1 (en) 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
JP7746280B2 (ja) * 2020-03-27 2025-09-30 ジェネレーション バイオ カンパニー 新規脂質及びそれらのナノ粒子組成物
AU2021307952A1 (en) * 2020-07-17 2023-03-02 Generation Bio Co. Methods for encapsulating polynucleotides into reduced sizes of Lipid nanoparticles and novel formulation thereof

Also Published As

Publication number Publication date
JP2025181861A (ja) 2025-12-11
US20230159459A1 (en) 2023-05-25
IL296763A (en) 2022-11-01
JP2023518985A (ja) 2023-05-09
IL296763B1 (en) 2025-12-01
BR112022019369A2 (pt) 2022-12-13
MX2022011988A (es) 2023-01-04
EP4125821A2 (en) 2023-02-08
WO2021195529A3 (en) 2021-11-18
KR20220160647A (ko) 2022-12-06
AU2021241696A1 (en) 2022-12-01
JP7746280B2 (ja) 2025-09-30
EP4125821A4 (en) 2024-06-26
CA3173126A1 (en) 2021-09-30
CN115916163A (zh) 2023-04-04
US20250361208A1 (en) 2025-11-27
US12252470B2 (en) 2025-03-18
US12534435B2 (en) 2026-01-27
WO2021195529A2 (en) 2021-09-30
US20240294474A1 (en) 2024-09-05

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