JP7700101B2 - 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法 - Google Patents

閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法 Download PDF

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JP7700101B2
JP7700101B2 JP2022514708A JP2022514708A JP7700101B2 JP 7700101 B2 JP7700101 B2 JP 7700101B2 JP 2022514708 A JP2022514708 A JP 2022514708A JP 2022514708 A JP2022514708 A JP 2022514708A JP 7700101 B2 JP7700101 B2 JP 7700101B2
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lipid
cedna
itr
dna
pharmaceutical composition
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JP2022546597A (ja
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ジエ スー，
プルーデンス ユイ トゥン リー，
デブラ クラット，
リア ユー リウ，
マシュー ジェイムズ チオッコ，
マシュー ジー． スタントン，
ジェフ モフィット，
ジョン エドワード チャタートン，
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ジェネレーション バイオ カンパニー
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K9/51—Nanocapsules; Nanoparticles
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022514708A 2019-09-06 2020-09-03 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法 Active JP7700101B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962896980P 2019-09-06 2019-09-06
US62/896,980 2019-09-06
US201962910720P 2019-10-04 2019-10-04
US62/910,720 2019-10-04
US201962940104P 2019-11-25 2019-11-25
US62/940,104 2019-11-25
PCT/US2020/049266 WO2021046265A1 (en) 2019-09-06 2020-09-03 Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof

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JP2022546597A JP2022546597A (ja) 2022-11-04
JP2022546597A5 JP2022546597A5 (enExample) 2023-09-11
JP7700101B2 true JP7700101B2 (ja) 2025-06-30

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US (1) US20220280427A1 (enExample)
EP (1) EP4025196A4 (enExample)
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CN (1) CN114929205B (enExample)
AU (1) AU2020342668B2 (enExample)
CA (1) CA3150452A1 (enExample)
IL (1) IL291038A (enExample)
WO (1) WO2021046265A1 (enExample)

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