AU2015350561B2 - Ionizable cationic lipid for RNA delivery - Google Patents

Ionizable cationic lipid for RNA delivery Download PDF

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Publication number
AU2015350561B2
AU2015350561B2 AU2015350561A AU2015350561A AU2015350561B2 AU 2015350561 B2 AU2015350561 B2 AU 2015350561B2 AU 2015350561 A AU2015350561 A AU 2015350561A AU 2015350561 A AU2015350561 A AU 2015350561A AU 2015350561 B2 AU2015350561 B2 AU 2015350561B2
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cationic lipid
rna delivery
ionizable cationic
ionizable
rna
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AU2015350561A
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AU2015350561A1 (en
Inventor
Padmanabh Chivukula
Joseph E. Payne
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Arcturus Therapeutics Inc
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Arcturus Therapeutics Inc
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Priority claimed from US14/546,105 external-priority patent/US9593077B2/en
Priority claimed from US14/707,876 external-priority patent/US9365610B2/en
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Publication of AU2015350561A1 publication Critical patent/AU2015350561A1/en
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C323/00Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
    • C07C323/50Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton
    • C07C323/51Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C323/60Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton with the carbon atom of at least one of the carboxyl groups bound to nitrogen atoms
    • 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
    • A61K48/0033Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • 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
    • C07C333/10Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups being part of any of the groups, X being a hetero atom, Y being any atom, e.g., N-acyl-thiocarbamates
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J1/00Normal steroids containing carbon, hydrogen, halogen or oxygen, not substituted in position 17 beta by a carbon atom, e.g. estrane, androstane
    • C07J1/0003Androstane derivatives
    • C07J1/0007Androstane derivatives not substituted in position 17
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    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J31/00Normal steroids containing one or more sulfur atoms not belonging to a hetero ring
    • C07J31/006Normal steroids containing one or more sulfur atoms not belonging to a hetero ring not covered by C07J31/003
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    • C07JSTEROIDS
    • C07J41/00Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0055Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
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    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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
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    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • Molecular Biology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Otolaryngology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
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AU2015350561A 2014-11-18 2015-05-11 Ionizable cationic lipid for RNA delivery Active AU2015350561B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US14/546,105 2014-11-18
US14/546,105 US9593077B2 (en) 2013-11-18 2014-11-18 Ionizable cationic lipid for RNA delivery
US14/707,876 US9365610B2 (en) 2013-11-18 2015-05-08 Asymmetric ionizable cationic lipid for RNA delivery
US14/707,796 US9567296B2 (en) 2013-11-18 2015-05-08 Ionizable cationic lipid for RNA delivery
US14/707,796 2015-05-08
US14/707,876 2015-05-08
PCT/US2015/030218 WO2016081029A1 (en) 2014-11-18 2015-05-11 Ionizable cationic lipid for rna delivery

Publications (2)

Publication Number Publication Date
AU2015350561A1 AU2015350561A1 (en) 2017-07-06
AU2015350561B2 true AU2015350561B2 (en) 2020-01-30

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AU2015350561A Active AU2015350561B2 (en) 2014-11-18 2015-05-11 Ionizable cationic lipid for RNA delivery

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EP (1) EP3221293B1 (enExample)
JP (1) JP6637988B2 (enExample)
KR (1) KR102380363B1 (enExample)
CN (1) CN107207428A (enExample)
AU (1) AU2015350561B2 (enExample)
CA (1) CA2968060C (enExample)
DK (1) DK3221293T3 (enExample)
ES (1) ES2946110T3 (enExample)
FI (1) FI3221293T3 (enExample)
HK (1) HK1244479A1 (enExample)
HR (1) HRP20230342T1 (enExample)
HU (1) HUE062130T2 (enExample)
IL (1) IL252291B (enExample)
LT (1) LT3221293T (enExample)
NZ (1) NZ732777A (enExample)
PL (1) PL3221293T3 (enExample)
PT (1) PT3221293T (enExample)
RS (1) RS64078B1 (enExample)
SI (1) SI3221293T1 (enExample)
WO (1) WO2016081029A1 (enExample)

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KR102095085B1 (ko) 2013-11-18 2020-03-31 ์•„ํฌํˆฌ๋ฃจ์Šค ์Ž„๋ผํ“จํ‹ฑ์Šค, ์ธํฌ. Rna ์ „๋‹ฌ์„ ์œ„ํ•œ ์ด์˜จํ™”๊ฐ€๋Šฅํ•œ ์–‘์ด์˜จ์„ฑ ์ง€์งˆ
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KR20210035022A (ko) 2016-12-14 2021-03-31 ๋ฆฌ๊ฐ„๋‹ฌ ์ธ์ฝ”ํฌ๋ ˆ์ดํ‹ฐ๋“œ ํ•ต์‚ฐ ๋ฐ/๋˜๋Š” ๋‹จ๋ฐฑ์งˆ ์ ์žฌ๋ฌผ ์ „๋‹ฌ์„ ์œ„ํ•œ ์กฐ์„ฑ๋ฌผ ๋ฐ ๋ฐฉ๋ฒ•
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CN116172975B (zh) * 2018-06-08 2025-03-28 ๅฏŒๅฃซ่ƒถ็‰‡ๆ ชๅผไผš็คพ ่„‚่ดจ้ข—็ฒ’ใ€ๅŒ…ๅซ่ฏฅ่„‚่ดจ้ข—็ฒ’็š„ๆ ธ้…ธ่ฏ็‰ฉไปฅๅŠๆ ธ้…ธ้€’้€็”จ็ป„ๅˆ็‰ฉ
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JP2022550435A (ja) 2019-10-04 2022-12-01 ใ‚ฆใƒซใƒˆใƒฉใ‚ธใ‚งใƒ‹ใƒƒใ‚ฏใ‚น ใƒ•ใ‚กใƒผใƒžใ‚ทใƒฅใƒผใƒ†ใ‚ฃใ‚ซใƒซ ใ‚คใƒณใ‚ณใƒผใƒใƒฌใ‚คใƒ†ใƒƒใƒ‰ ็ต„ๆ›ใˆ๏ฝ๏ฝ๏ฝ–ใฎๆ”นๅ–„ใ•ใ‚ŒใŸๆฒป็™‚็š„ไฝฟ็”จใฎใŸใ‚ใฎๆ–นๆณ•
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JP2023520763A (ja) 2020-03-24 2023-05-19 ใ‚ธใ‚งใƒใƒฌใƒผใ‚ทใƒงใƒณ ใƒใ‚คใ‚ช ใ‚ซใƒณใƒ‘ใƒ‹ใƒผ ใ‚ดใƒผใ‚ทใ‚งๆฒป็™‚่–ฌใ‚’็™บ็พใ™ใ‚‹ใŸใ‚ใฎ้žใ‚ฆใ‚คใƒซใ‚นๆ€ง๏ฝ„๏ฝŽ๏ฝใƒ™ใ‚ฏใ‚ฟใƒผๅŠใณใใฎไฝฟ็”จ
JP2023530487A (ja) * 2020-06-18 2023-07-18 ใ‚ขใƒผใ‚ฏใƒˆใ‚ฅใƒฉใ‚นใƒปใ‚ปใƒฉใƒ”ใƒฅใƒผใƒ†ใ‚ฃใ‚ฏใ‚นใƒปใ‚คใƒณใ‚ณใƒผใƒใƒฌใ‚คใƒ†ใƒƒใƒ‰ ใƒใƒชใ‚ฐใƒซใ‚ฟใƒŸใƒณ็—…ใฎๆฒป็™‚ใฎใŸใ‚ใฎ๏ฝ•๏ฝŽ๏ฝใ‚ชใƒชใ‚ดใƒžใƒผ
JP2023535632A (ja) 2020-07-27 2023-08-18 ใ‚ขใƒณใ‚ธใƒฃใƒชใ‚ฆใƒ  ใƒใ‚คใ‚ชใ‚ตใ‚คใ‚จใƒณใ‚ทใ‚บ ใ‚จใƒผใ‚ธใƒผ ๏ผค๏ฝŽ๏ฝๅˆ†ๅญใฎ็ต„ๆˆ็‰ฉใ€ใใฎไฝœ่ฃฝๆ–นๆณ•ใ€ๅŠใณใใฎไฝฟ็”จๆ–นๆณ•
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CN118541346A (zh) 2021-11-08 2024-08-23 ๅฅฅ็บณๆฒป็–—ๅ…ฌๅธ ็”จไบŽ้€’้€็Žฏ็Šถๅคšๆ ธ่‹ท้…ธ็š„่„‚่ดจ็บณ็ฑณ้ข—็ฒ’็ป„ๅˆ็‰ฉ
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
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JP2025509607A (ja) 2022-03-14 2025-04-11 ใ‚ธใ‚งใƒใƒฌใƒผใ‚ทใƒงใƒณ ใƒใ‚คใ‚ช ใ‚ซใƒณใƒ‘ใƒ‹ใƒผ ็•ฐ็จฎใƒ—ใƒฉใ‚คใƒ ใƒ–ใƒผใ‚นใƒˆใƒฏใ‚ฏใƒใƒณ็ต„ๆˆ็‰ฉๅŠใณไฝฟ็”จใฎๆ–นๆณ•
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