JPWO2020097540A5 - - Google Patents

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JPWO2020097540A5
JPWO2020097540A5 JP2021525115A JP2021525115A JPWO2020097540A5 JP WO2020097540 A5 JPWO2020097540 A5 JP WO2020097540A5 JP 2021525115 A JP2021525115 A JP 2021525115A JP 2021525115 A JP2021525115 A JP 2021525115A JP WO2020097540 A5 JPWO2020097540 A5 JP WO2020097540A5
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Japan
Prior art keywords
lipid
nucleic acid
composition
nanoparticle
nanoparticles
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Pending
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JP2021525115A
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Japanese (ja)
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JP2022506956A (en
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Priority claimed from PCT/US2019/060582 external-priority patent/WO2020097540A1/en
Publication of JP2022506956A publication Critical patent/JP2022506956A/en
Publication of JPWO2020097540A5 publication Critical patent/JPWO2020097540A5/ja
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Claims (15)

（ａ）１つ以上の核酸分子；
（ｂ）コレステロール；
（ｃ）ＤＳＰＣ；
（ｄ）ＰＥＧ－Ｃ－ＤＭＡ；及び
（ｅ）下記式のカチオン性脂質またはその塩：
Figure 2020097540000001
 を含む脂質ナノ粒子であって、総脂質中のＰＥＧ－Ｃ－ＤＭＡ、カチオン性脂質、コレステロール、及びＤＳＰＣのモル百分率が、
ＰＥＧ－Ｃ－ＤＭＡ：約１．５；
カチオン性脂質：約５０．０；
コレステロール：約３８．５；及び
ＤＳＰＣ：約１０．０
である、前記脂質ナノ粒子。 (a) one or more nucleic acid molecules;
(b) cholesterol;
(c) DSPCs;
(d) PEG-C-DMA; and (e) a cationic lipid or salt thereof of the formula:
Figure 2020097540000001
 wherein the molar percentages of PEG-C-DMA, cationic lipids, cholesterol, and DSPC in total lipid are
PEG-C-DMA: about 1.5;
cationic lipid: about 50.0;
Cholesterol: about 38.5; and DSPC: about 10.0
The lipid nanoparticles. 前記１つ以上の核酸分子がｓｉＲＮＡを含む、請求項１に記載の脂質ナノ粒子。 2. The lipid nanoparticle of claim 1, wherein said one or more nucleic acid molecules comprise siRNA. 前記１つ以上の核酸分子がｍＲＮＡを含む、請求項１に記載の脂質ナノ粒子。 2. The lipid nanoparticle of claim 1, wherein said one or more nucleic acid molecules comprise mRNA. 総脂質の核酸に対する重量比が約１７超である、請求項１～３のいずれか１項に記載の脂質ナノ粒子。 4. The lipid nanoparticle of any one of claims 1-3 , wherein the total lipid to nucleic acid weight ratio is greater than about 17. 総脂質の核酸に対する重量比が約１８超である、請求項１～３のいずれか１項に記載の脂質ナノ粒子。 4. The lipid nanoparticle of any one of claims 1-3 , wherein the total lipid to nucleic acid weight ratio is greater than about 18. 総脂質の核酸に対する重量比が約１９超である、請求項１～３のいずれか１項に記載の脂質ナノ粒子。 4. The lipid nanoparticle of any one of claims 1-3 , wherein the total lipid to nucleic acid weight ratio is greater than about 19. 総脂質の核酸に対する重量比が約２２～約２５である、請求項１～３のいずれか１項に記載の脂質ナノ粒子。 4. The lipid nanoparticle of any one of claims 1-3 , wherein the total lipid to nucleic acid weight ratio is from about 22 to about 25. 請求項１～７のいずれか１項に記載の脂質ナノ粒子と、薬学的に許容可能な担体とを含む、医薬組成物。 A pharmaceutical composition comprising the lipid nanoparticles of any one of claims 1-7 and a pharmaceutically acceptable carrier. 皮下投与用に製剤化される、請求項８に記載の医薬組成物。 9. A pharmaceutical composition according to claim 8, formulated for subcutaneous administration. ｉｎ ｖｉｔｒｏで核酸を細胞に送達するための方法であって、請求項１～７のいずれか１項に記載の脂質ナノ粒子と細胞を接触させることを含む、前記方法。 A method for delivering nucleic acids to cells in vitro , said method comprising contacting a cell with a lipid nanoparticle according to any one of claims 1-7. 核酸を細胞に送達するための組成物であって、請求項１～７のいずれか１項に記載の脂質ナノ粒子を含み、前記脂質ナノ粒子と細胞を接触させる、前記組成物。 A composition for delivering nucleic acids to cells, said composition comprising the lipid nanoparticles of any one of claims 1 to 7, wherein said lipid nanoparticles are contacted with said cells . 機能性タンパク質の欠損を生じさせる遺伝的欠陥を特徴とする疾患を治療するための組成物であって、請求項１～７のいずれか１項に記載の脂質ナノ粒子を含み、前記核酸分子が、前記機能性タンパク質または前記機能性タンパク質と同じ生物学的活性を有するタンパク質をコードするｍＲＮＡである、前記組成物。 A composition for treating a disease characterized by a genetic defect resulting in a deficiency of a functional protein, said composition comprising a lipid nanoparticle according to any one of claims 1 to 7 , wherein said nucleic acid molecule comprises , mRNA encoding said functional protein or a protein having the same biological activity as said functional protein . ポリペプチドの過剰発現を特徴とする疾患を治療するための組成物であって、請求項１～７のいずれか１項に記載の脂質ナノ粒子を含み、前記核酸分子が、過剰発現されたポリペプチドの発現を標的とするｓｉＲＮＡである、前記組成物。 A composition for treating a disease characterized by overexpression of a polypeptide, comprising the lipid nanoparticles of any one of claims 1-7, wherein said nucleic acid molecule comprises an overexpressed polypeptide. The above composition , which is an siRNA that targets the expression of the peptide. 機能性タンパク質の欠損を生じさせる遺伝的欠陥を特徴とする疾患の治療的または予防的処置で使用するためのものである、請求項１～７のいずれか１項に記載の脂質ナノ粒子。 Lipid nanoparticles according to any one of claims 1 to 7, for use in the therapeutic or prophylactic treatment of diseases characterized by genetic defects resulting in functional protein deficiencies. ポリペプチドの過剰発現を特徴とする疾患の治療的または予防的処置で使用するためのものである、請求項１～７のいずれか１項に記載の脂質ナノ粒子。 Lipid nanoparticles according to any one of claims 1 to 7, for use in the therapeutic or prophylactic treatment of diseases characterized by overexpression of the polypeptide.
JP2021525115A 2018-11-09 2019-11-08 Lipid nanoparticle preparation Pending JP2022506956A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862758088P 2018-11-09 2018-11-09
US62/758,088 2018-11-09
PCT/US2019/060582 WO2020097540A1 (en) 2018-11-09 2019-11-08 Lipid nanoparticle formulations

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JP2022506956A JP2022506956A (en) 2022-01-17
JPWO2020097540A5 true JPWO2020097540A5 (en) 2022-11-07

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US (1) US20220000778A1 (en)
EP (1) EP3877521A4 (en)
JP (1) JP2022506956A (en)
KR (1) KR20210105889A (en)
CN (1) CN113286882A (en)
AU (1) AU2019376161A1 (en)
BR (1) BR112021008982A2 (en)
CA (1) CA3119071A1 (en)
EA (1) EA202191313A1 (en)
IL (1) IL282995A (en)
MX (1) MX2021005389A (en)
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WO (1) WO2020097540A1 (en)

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WO2021205077A1 (en) 2020-04-09 2021-10-14 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
TW202233232A (en) 2020-11-06 2022-09-01 法商賽諾菲公司 Lipid nanoparticles for delivering mrna vaccines
CN113509542A (en) * 2021-04-20 2021-10-19 嘉晨西海(杭州)生物技术有限公司 Medicine for expressing interleukin 12 and aiming at tumor based on mRNA and preparation method thereof
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CA3132191A1 (en) * 2021-09-28 2023-03-28 Providence Therapeutics Holdings Inc. Compositions and methods for the prevention and/or treatment of covid-19
CA3236924A1 (en) * 2021-11-05 2023-05-11 Sanofi Respiratory syncytial virus rna vaccine
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WO2023165582A1 (en) * 2022-03-04 2023-09-07 益杰立科(上海)生物科技有限公司 Targeted delivery system and method
KR20230145791A (en) 2022-04-11 2023-10-18 연세대학교 원주산학협력단 Cationic lipid nanoparticles for mRNA vaccine
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
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