CN109475646A - 用于crispr/cas成分的脂质纳米颗粒制剂 - Google Patents

用于crispr/cas成分的脂质纳米颗粒制剂 Download PDF

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Publication number
CN109475646A
CN109475646A CN201780032950.4A CN201780032950A CN109475646A CN 109475646 A CN109475646 A CN 109475646A CN 201780032950 A CN201780032950 A CN 201780032950A CN 109475646 A CN109475646 A CN 109475646A
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composition
lnp
lipid
guide rna
nucleic acid
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CN201780032950.4A
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Chinese (zh)
Inventor
D·V·莫里西
M·C·帕特尔
J·D·芬恩
A·M·R·史密斯
L·J·肖
C·东布罗夫斯基
R·R·沙哈
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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Priority to CN202311619829.5A priority Critical patent/CN117731805A/zh
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
CN201780032950.4A 2016-03-30 2017-03-30 用于crispr/cas成分的脂质纳米颗粒制剂 Pending CN109475646A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202311619829.5A CN117731805A (zh) 2016-03-30 2017-03-30 用于crispr/cas成分的脂质纳米颗粒制剂

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662315602P 2016-03-30 2016-03-30
US62/315,602 2016-03-30
US201662375776P 2016-08-16 2016-08-16
US62/375,776 2016-08-16
US201662433228P 2016-12-12 2016-12-12
US62/433,228 2016-12-12
US201762468300P 2017-03-07 2017-03-07
US62/468,300 2017-03-07
PCT/US2017/024973 WO2017173054A1 (en) 2016-03-30 2017-03-30 Lipid nanoparticle formulations for crispr/cas components

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CN202311619829.5A Division CN117731805A (zh) 2016-03-30 2017-03-30 用于crispr/cas成分的脂质纳米颗粒制剂

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US (2) US20190136231A1 (https=)
EP (2) EP4585272A3 (https=)
JP (3) JP7245651B2 (https=)
KR (1) KR102617874B1 (https=)
CN (2) CN109475646A (https=)
AU (2) AU2017244143B2 (https=)
BR (1) BR112018069795A2 (https=)
CA (1) CA3018978A1 (https=)
CO (1) CO2018011554A2 (https=)
DK (1) DK3436077T3 (https=)
ES (1) ES3030658T3 (https=)
FI (1) FI3436077T3 (https=)
HR (1) HRP20250670T1 (https=)
HU (1) HUE071550T2 (https=)
LT (1) LT3436077T (https=)
MX (1) MX2024008101A (https=)
PL (1) PL3436077T3 (https=)
PT (1) PT3436077T (https=)
RS (1) RS66865B1 (https=)
SI (1) SI3436077T1 (https=)
SM (1) SMT202500211T1 (https=)
TW (1) TWI773666B (https=)
WO (1) WO2017173054A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
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CN114026237A (zh) * 2019-02-13 2022-02-08 比姆医疗股份有限公司 用于治疗1a型糖原贮积病的组成物和方法

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