KR20240099377A - 올리고뉴클레오티드 전달용 리피드 나노입자 - Google Patents

올리고뉴클레오티드 전달용 리피드 나노입자 Download PDF

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Publication number
KR20240099377A
KR20240099377A KR1020247018058A KR20247018058A KR20240099377A KR 20240099377 A KR20240099377 A KR 20240099377A KR 1020247018058 A KR1020247018058 A KR 1020247018058A KR 20247018058 A KR20247018058 A KR 20247018058A KR 20240099377 A KR20240099377 A KR 20240099377A
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KR
South Korea
Prior art keywords
lipid
rna
mmol
ethyl
compound
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Pending
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KR1020247018058A
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English (en)
Korean (ko)
Inventor
에이케이엠 아시쿨 하크
소피 발렘보이스
카페르티 세안 엠씨
이티스리 사휘
크리스티안 카르돈
Original Assignee
지피우스 엔브이
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Publication of KR20240099377A publication Critical patent/KR20240099377A/ko
Pending legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D295/00Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/12Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
    • C07D295/125Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/13Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • 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
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • 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
    • A61K47/24Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • 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
    • 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
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • 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
    • 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
    • A61K9/513Organic macromolecular compounds; Dendrimers
    • A61K9/5146Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53DNA (RNA) vaccination
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/60Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6018Lipids, e.g. in lipopeptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Optics & Photonics (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247018058A 2021-11-02 2022-11-02 올리고뉴클레오티드 전달용 리피드 나노입자 Pending KR20240099377A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP21205904 2021-11-02
EP21205904.2 2021-11-02
EP22179399.5 2022-06-16
EP22179399 2022-06-16
PCT/EP2022/080581 WO2023078950A1 (en) 2021-11-02 2022-11-02 Lipid nanoparticles for oligonucleotide delivery

Publications (1)

Publication Number Publication Date
KR20240099377A true KR20240099377A (ko) 2024-06-28

Family

ID=84364268

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247018058A Pending KR20240099377A (ko) 2021-11-02 2022-11-02 올리고뉴클레오티드 전달용 리피드 나노입자

Country Status (14)

Country Link
US (1) US20250236599A1 (https=)
EP (2) EP4613741A3 (https=)
JP (1) JP2024540108A (https=)
KR (1) KR20240099377A (https=)
AU (1) AU2022381463A1 (https=)
CA (1) CA3236653A1 (https=)
DK (1) DK4426270T3 (https=)
ES (1) ES3042660T3 (https=)
FI (1) FI4426270T3 (https=)
IL (1) IL312432B1 (https=)
MX (1) MX2024005109A (https=)
PL (1) PL4426270T3 (https=)
WO (1) WO2023078950A1 (https=)
ZA (1) ZA202403360B (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
CN118125994A (zh) * 2022-11-25 2024-06-04 艾斯拓康医药科技(北京)有限公司 可离子化脂质及其应用
WO2024136309A1 (en) * 2022-12-23 2024-06-27 Green Cross Corporation Ionizable lipid and use thereof
CN116514696B (zh) * 2023-06-29 2023-12-01 艾斯拓康医药科技(北京)有限公司 可离子化脂质及其应用
WO2025088152A1 (en) 2023-10-27 2025-05-01 Ziphius Nv MODIFIED 5'UTRs
US12599560B2 (en) 2023-11-02 2026-04-14 Longuide Biopharma Corporation Lipids and lipid nanoparticle formulations
WO2025104023A1 (en) 2023-11-13 2025-05-22 Ziphius Nv MODIFIED ALPHAVIRAL nsP3
WO2025128696A1 (en) * 2023-12-12 2025-06-19 Acuitas Therapeutics, Inc. Cationic lipid compounds for use in lipid nanoparticles
WO2025255534A1 (en) * 2024-06-07 2025-12-11 Innovac Therapeutics Hk Limited Diol-based novel ionizable lipids and their uses thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6451592B1 (en) 1996-04-05 2002-09-17 Chiron Corporation Recombinant alphavirus-based vectors with reduced inhibition of cellular macromolecular synthesis
CN1989250B (zh) 2004-05-18 2013-11-20 阿尔法瓦克斯公司 Tc-83衍生的甲病毒属载体、颗粒和方法
US7332322B2 (en) 2004-09-14 2008-02-19 Ilya Frolov Venezuelan equine encephalitis virus replicons with adaptive mutations in the genome and uses thereof
WO2014028487A1 (en) * 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
WO2018232120A1 (en) * 2017-06-14 2018-12-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents

Also Published As

Publication number Publication date
EP4613741A2 (en) 2025-09-10
EP4426270B1 (en) 2025-07-23
US20250236599A1 (en) 2025-07-24
IL312432B1 (en) 2026-01-01
EP4426270A1 (en) 2024-09-11
JP2024540108A (ja) 2024-10-31
MX2024005109A (es) 2024-05-31
WO2023078950A1 (en) 2023-05-11
IL312432A (en) 2024-06-01
ES3042660T3 (en) 2025-11-24
FI4426270T3 (fi) 2025-09-12
DK4426270T3 (da) 2025-09-22
PL4426270T3 (pl) 2025-11-24
AU2022381463A1 (en) 2024-05-16
EP4613741A3 (en) 2025-11-12
ZA202403360B (en) 2025-08-27
CA3236653A1 (en) 2023-05-11

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Date Code Title Description
PA0105 International application

Patent event date: 20240529

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application