CN119487200A - 用于基因治疗方法的组织特异性基因外安全港的鉴定 - Google Patents

用于基因治疗方法的组织特异性基因外安全港的鉴定 Download PDF

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Publication number
CN119487200A
CN119487200A CN202380047942.2A CN202380047942A CN119487200A CN 119487200 A CN119487200 A CN 119487200A CN 202380047942 A CN202380047942 A CN 202380047942A CN 119487200 A CN119487200 A CN 119487200A
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genomic
nucleic acid
seq
safe harbor
coordinates
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Chinese (zh)
Inventor
西罗·博内蒂
国春·龚
J·何
J·刘
格雷格·沃肖
埃里克·乔
布莱恩·扎姆布罗维兹
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • A61K48/0025—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 '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
    • A61K48/0041—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 '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 polymeric
    • A—HUMAN NECESSITIES
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    • 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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    • C12N15/1034—Isolating an individual clone by screening libraries
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • A—HUMAN NECESSITIES
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    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202380047942.2A 2022-04-29 2023-04-28 用于基因治疗方法的组织特异性基因外安全港的鉴定 Pending CN119487200A (zh)

Applications Claiming Priority (3)

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US202263336663P 2022-04-29 2022-04-29
US63/336,663 2022-04-29
PCT/US2023/066343 WO2023212677A2 (en) 2022-04-29 2023-04-28 Identification of tissue-specific extragenic safe harbors for gene therapy approaches

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US (1) US20250332287A1 (enExample)
EP (1) EP4514981A2 (enExample)
JP (1) JP2025514304A (enExample)
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US12668774B2 (en) 2023-10-12 2026-06-30 Btl Healthcare Technologies A.S. Product, system and method of cell cultivation

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WO2023212677A2 (en) 2023-11-02
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