SI3558943T1 - Ionizabilen kationski lipid za dostavo rna - Google Patents

Ionizabilen kationski lipid za dostavo rna

Info

Publication number
SI3558943T1
SI3558943T1 SI201731472T SI201731472T SI3558943T1 SI 3558943 T1 SI3558943 T1 SI 3558943T1 SI 201731472 T SI201731472 T SI 201731472T SI 201731472 T SI201731472 T SI 201731472T SI 3558943 T1 SI3558943 T1 SI 3558943T1
Authority
SI
Slovenia
Prior art keywords
cationic lipid
rna delivery
ionizable cationic
ionizable
rna
Prior art date
Application number
SI201731472T
Other languages
English (en)
Inventor
Joseph E. Payne
Padmanabh Chivikula
Steven P. 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.,
Priority claimed from PCT/US2017/067756 external-priority patent/WO2018119163A1/en
Publication of SI3558943T1 publication Critical patent/SI3558943T1/sl

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)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (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)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (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)
SI201731472T 2016-12-21 2017-12-20 Ionizabilen kationski lipid za dostavo rna SI3558943T1 (sl)

Applications Claiming Priority (4)

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
PCT/US2017/067756 WO2018119163A1 (en) 2016-12-21 2017-12-20 Ionizable cationic lipid for rna delivery
EP17826404.0A EP3558943B1 (en) 2016-12-21 2017-12-20 Ionizable cationic lipid for rna delivery

Publications (1)

Publication Number Publication Date
SI3558943T1 true SI3558943T1 (sl) 2024-03-29

Family

ID=58191571

Family Applications (1)

Application Number Title Priority Date Filing Date
SI201731472T SI3558943T1 (sl) 2016-12-21 2017-12-20 Ionizabilen kationski lipid za dostavo rna

Country Status (18)

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

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US9593077B2 (en) 2013-11-18 2017-03-14 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
US10383952B2 (en) * 2016-12-21 2019-08-20 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
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US11939600B2 (en) 2017-05-31 2024-03-26 Arcturus Therapeutics, Inc. Compositions and methods for treating phenylketonuria
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CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
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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
EP4117720A4 (en) * 2020-03-09 2024-04-24 Arcturus Therapeutics, Inc. COMPOSITIONS AND METHODS FOR INDUCING IMMUNE RESPONSES
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TW202245809A (zh) 2020-12-18 2022-12-01 美商詹森藥物公司 用於治療b型肝炎病毒感染之組合療法
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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
EP4395748A1 (en) 2021-09-03 2024-07-10 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
AR127312A1 (es) 2021-10-08 2024-01-10 Suzhou Abogen Biosciences Co Ltd Compuestos lipídicos ycomposiciones de nanopartículas lipídicas
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
WO2023129915A2 (en) * 2021-12-27 2023-07-06 The Board Of Regents Of The University Of Texas System 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
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
WO2024040194A1 (en) 2022-08-17 2024-02-22 Capstan Therapeutics, Inc. Conditioning for in vivo immune cell engineering
DE202023106198U1 (de) 2022-10-28 2024-03-21 CureVac SE Impfstoff auf Nukleinsäurebasis
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
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Also Published As

Publication number Publication date
AU2021200663B2 (en) 2023-09-07
CN110325511A (zh) 2019-10-11
CN110325511B (zh) 2022-04-19
US20190388562A1 (en) 2019-12-26
DK3558943T3 (en) 2024-01-15
IL267317B1 (en) 2023-07-01
JP6905062B2 (ja) 2021-07-21
EP4310075A3 (en) 2024-04-24
EP3558942B1 (en) 2023-09-06
IL267317A (en) 2019-08-29
IL267317B2 (en) 2023-11-01
JP2023071947A (ja) 2023-05-23
HUE065439T2 (hu) 2024-05-28
JP7437935B2 (ja) 2024-02-26
AU2017379059A1 (en) 2019-08-08
EP3558942A1 (en) 2019-10-30
US20210252163A1 (en) 2021-08-19
IL290592A (en) 2022-04-01
KR20210112396A (ko) 2021-09-14
KR102299053B1 (ko) 2021-09-07
RS65078B1 (sr) 2024-02-29
JP2020504730A (ja) 2020-02-13
KR20190134593A (ko) 2019-12-04
AU2017379059B2 (en) 2020-11-12
CA3046885C (en) 2023-03-07
CA3046885A1 (en) 2018-06-28
WO2018118102A1 (en) 2018-06-28
FI3558943T3 (fi) 2024-01-12
AU2021200663A1 (en) 2021-03-04
IL267511A (en) 2019-08-29
IL306099A (en) 2023-11-01
CN114917203A (zh) 2022-08-19
EP3558943B1 (en) 2023-10-18
PT3558943T (pt) 2024-01-12
CN110337429B (zh) 2021-12-21
KR20190122653A (ko) 2019-10-30
IL290592B1 (en) 2023-10-01
EP4310075A2 (en) 2024-01-24
JP2020504728A (ja) 2020-02-13
US10980895B2 (en) 2021-04-20
US10383952B2 (en) 2019-08-20
AU2017379587B2 (en) 2020-12-10
AU2017379587A1 (en) 2019-08-08
CN110337429A (zh) 2019-10-15
HRP20231742T1 (hr) 2024-03-15
CA3047033A1 (en) 2018-06-28
JP2021073263A (ja) 2021-05-13
JP2023071950A (ja) 2023-05-23
IL267511B (en) 2022-04-01
IL290592B2 (en) 2024-02-01
JP2021088588A (ja) 2021-06-10
ES2969232T3 (es) 2024-05-17
EP3558943A1 (en) 2019-10-30
US20180169268A1 (en) 2018-06-21
EP3558942C0 (en) 2023-09-06
KR102385562B1 (ko) 2022-04-11
PL3558943T3 (pl) 2024-04-02

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