HUE065439T2 - Ionizálható kationos lipid RNS szállítására - Google Patents

Ionizálható kationos lipid RNS szállítására

Info

Publication number
HUE065439T2
HUE065439T2 HUE17826404A HUE17826404A HUE065439T2 HU E065439 T2 HUE065439 T2 HU E065439T2 HU E17826404 A HUE17826404 A HU E17826404A HU E17826404 A HUE17826404 A HU E17826404A HU E065439 T2 HUE065439 T2 HU E065439T2
Authority
HU
Hungary
Prior art keywords
cationic lipid
rna delivery
ionizable cationic
ionizable
rna
Prior art date
Application number
HUE17826404A
Other languages
English (en)
Inventor
Joseph Payne
Padmanabh Chivikula
Steven Tanis
Priya Karmali
Original Assignee
Arcturus Therapeutics Inc
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 Arcturus Therapeutics Inc filed Critical Arcturus Therapeutics Inc
Publication of HUE065439T2 publication Critical patent/HUE065439T2/hu

Links

Classifications

    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02Monothiocarbamic acids; Derivatives thereof
    • C07C333/04Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/20Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
    • 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
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61K47/16Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
HUE17826404A 2016-12-21 2017-12-20 Ionizálható kationos lipid RNS szállítására HUE065439T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US15/387,067 US10383952B2 (en) 2016-12-21 2016-12-21 Ionizable cationic lipid for RNA delivery
PCT/US2017/015886 WO2018118102A1 (en) 2016-12-21 2017-01-31 Ionizable cationic lipid for rna delivery

Publications (1)

Publication Number Publication Date
HUE065439T2 true HUE065439T2 (hu) 2024-05-28

Family

ID=58191571

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE17826404A HUE065439T2 (hu) 2016-12-21 2017-12-20 Ionizálható kationos lipid RNS szállítására

Country Status (18)

Country Link
US (3) US10383952B2 (hu)
EP (3) EP3558942B1 (hu)
JP (6) JP6905062B2 (hu)
KR (3) KR20190134593A (hu)
CN (3) CN110337429B (hu)
AU (3) AU2017379587B2 (hu)
CA (2) CA3047033A1 (hu)
DK (1) DK3558943T3 (hu)
ES (1) ES2969232T3 (hu)
FI (1) FI3558943T3 (hu)
HR (1) HRP20231742T1 (hu)
HU (1) HUE065439T2 (hu)
IL (4) IL267317B2 (hu)
PL (1) PL3558943T3 (hu)
PT (1) PT3558943T (hu)
RS (1) RS65078B1 (hu)
SI (1) SI3558943T1 (hu)
WO (1) WO2018118102A1 (hu)

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CA2930602C (en) 2013-11-18 2019-05-28 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery
US10383952B2 (en) * 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US11433143B2 (en) * 2017-05-18 2022-09-06 The Regents Of The University Of California Nano-enabled immunotherapy in cancer
US11939600B2 (en) 2017-05-31 2024-03-26 Arcturus Therapeutics, Inc. Compositions and methods for treating phenylketonuria
AR112706A1 (es) 2017-05-31 2019-12-04 Ultragenyx Pharmaceutical Inc Productos terapéuticos para la glucogenosis tipo iii
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EP4299750A3 (en) 2018-12-06 2024-07-10 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
TW202114732A (zh) 2019-06-20 2021-04-16 愛爾蘭商健生科學愛爾蘭無限公司 遞送b型肝炎病毒(hbv)疫苗之脂質奈米顆粒或脂質體
US20220347298A1 (en) 2019-10-04 2022-11-03 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
KR102433471B1 (ko) * 2019-10-17 2022-08-18 한국과학기술원 지질이 접합된 유전자편집 단백질 기반 crispr 복합체 및 이의 제조방법
US11744887B2 (en) * 2020-03-09 2023-09-05 Arcturus Therapeutics, Inc. Coronavirus vaccine compositions and methods
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TW202313557A (zh) * 2021-05-06 2023-04-01 美商亞克圖羅斯醫療公司 用於rna遞送之可離子化陽離子脂質
CN113633766B (zh) * 2021-08-11 2023-06-30 通用生物(安徽)股份有限公司 一种脂质体核酸疫苗佐剂及其制备方法
IL309505A (en) 2021-09-03 2024-02-01 CureVac SE Lipid nanoparticles for nucleic acid delivery
AR127312A1 (es) 2021-10-08 2024-01-10 Suzhou Abogen Biosciences Co Ltd Compuestos lipídicos ycomposiciones de nanopartículas lipídicas
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
IL313876A (en) * 2021-12-27 2024-08-01 Univ Texas Compositions and methods for targeted delivery to cells
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
CN116354836A (zh) * 2022-03-25 2023-06-30 深圳市新合生物医疗科技有限公司 阳离子脂质化合物及其制备方法和应用、以及mRNA递送系统
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
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WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
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WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
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CN115745820B (zh) * 2023-01-05 2023-04-28 北京悦康科创医药科技股份有限公司 长效低毒的新型阳离子脂质化合物及其组合物
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Also Published As

Publication number Publication date
DK3558943T3 (en) 2024-01-15
IL306099A (en) 2023-11-01
PT3558943T (pt) 2024-01-12
CA3046885A1 (en) 2018-06-28
JP2020504728A (ja) 2020-02-13
IL267317B2 (en) 2023-11-01
JP2021088588A (ja) 2021-06-10
PL3558943T3 (pl) 2024-04-02
US10383952B2 (en) 2019-08-20
US20190388562A1 (en) 2019-12-26
JP6905062B2 (ja) 2021-07-21
IL267317A (en) 2019-08-29
AU2017379587A1 (en) 2019-08-08
IL267511A (en) 2019-08-29
KR102299053B1 (ko) 2021-09-07
IL267511B (en) 2022-04-01
IL290592B2 (en) 2024-02-01
EP3558942C0 (en) 2023-09-06
SI3558943T1 (sl) 2024-03-29
IL290592B1 (en) 2023-10-01
EP3558942B1 (en) 2023-09-06
AU2017379059A1 (en) 2019-08-08
IL267317B1 (en) 2023-07-01
US10980895B2 (en) 2021-04-20
AU2021200663B2 (en) 2023-09-07
CN114917203A (zh) 2022-08-19
FI3558943T3 (fi) 2024-01-12
US20180169268A1 (en) 2018-06-21
EP3558943B1 (en) 2023-10-18
AU2021200663A1 (en) 2021-03-04
KR20190134593A (ko) 2019-12-04
EP3558942A1 (en) 2019-10-30
JP7437935B2 (ja) 2024-02-26
KR20210112396A (ko) 2021-09-14
KR20190122653A (ko) 2019-10-30
CA3047033A1 (en) 2018-06-28
JP2023071947A (ja) 2023-05-23
ES2969232T3 (es) 2024-05-17
CN110337429A (zh) 2019-10-15
EP3558943A1 (en) 2019-10-30
EP4310075A3 (en) 2024-04-24
JP2021073263A (ja) 2021-05-13
HRP20231742T1 (hr) 2024-03-15
AU2017379059B2 (en) 2020-11-12
JP2020504730A (ja) 2020-02-13
CA3046885C (en) 2023-03-07
CN110337429B (zh) 2021-12-21
JP2023071950A (ja) 2023-05-23
CN110325511A (zh) 2019-10-11
EP4310075A2 (en) 2024-01-24
RS65078B1 (sr) 2024-02-29
WO2018118102A1 (en) 2018-06-28
IL290592A (en) 2022-04-01
CN110325511B (zh) 2022-04-19
AU2017379587B2 (en) 2020-12-10
KR102385562B1 (ko) 2022-04-11
US20210252163A1 (en) 2021-08-19

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