CN110546262B - 用于治疗血红蛋白病的组合物和方法 - Google Patents

用于治疗血红蛋白病的组合物和方法 Download PDF

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CN110546262B
CN110546262B CN201880020201.4A CN201880020201A CN110546262B CN 110546262 B CN110546262 B CN 110546262B CN 201880020201 A CN201880020201 A CN 201880020201A CN 110546262 B CN110546262 B CN 110546262B
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cell
seq
cells
grna molecule
grna
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CN110546262A (zh
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C·S·米克宁
C·施密特
J·斯尼德
S·C·史蒂文森
杨毅
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Novartis AG
Intellia Therapeutics Inc
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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
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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CN201880020201.4A 2017-02-06 2018-02-05 用于治疗血红蛋白病的组合物和方法 Active CN110546262B (zh)

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US201762455464P 2017-02-06 2017-02-06
US62/455,464 2017-02-06
PCT/IB2018/050712 WO2018142364A1 (en) 2017-02-06 2018-02-05 Compositions and methods for the treatment of hemoglobinopathies

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US (2) US11466271B2 (https=)
EP (1) EP3577223A1 (https=)
JP (3) JP2020505934A (https=)
KR (2) KR102630217B1 (https=)
CN (2) CN110546262B (https=)
AR (1) AR110962A1 (https=)
AU (3) AU2018215726B2 (https=)
CA (1) CA3052275A1 (https=)
EA (1) EA201991862A1 (https=)
IL (2) IL311222A (https=)
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PH (1) PH12019501812A1 (https=)
SA (1) SA519402400B1 (https=)
SG (1) SG11201907056XA (https=)
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