CN110214005A - 用于基因治疗的结构和方法 - Google Patents

用于基因治疗的结构和方法 Download PDF

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Publication number
CN110214005A
CN110214005A CN201780083137.XA CN201780083137A CN110214005A CN 110214005 A CN110214005 A CN 110214005A CN 201780083137 A CN201780083137 A CN 201780083137A CN 110214005 A CN110214005 A CN 110214005A
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liposome
alkyl
deuterium
cases
polymer
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Pending
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CN201780083137.XA
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English (en)
Chinese (zh)
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马布西吉·艾哈迈德
蒂莫西·保罗·戴
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Particella Inc
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Dnalite Therapeutics Inc
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Publication of CN110214005A publication Critical patent/CN110214005A/zh
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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
    • A61K48/0008—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
    • 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
    • A61K48/0033—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 non-polymeric
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    • A61K31/63—Compounds containing para-N-benzenesulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonyl hydrazide
    • A61K31/635—Compounds containing para-N-benzenesulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonyl hydrazide having a heterocyclic ring, e.g. sulfadiazine
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    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
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    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5138—Organic macromolecular compounds; Dendrimers obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyvinyl pyrrolidone, poly(meth)acrylates
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    • 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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Peptides Or Proteins (AREA)
CN201780083137.XA 2016-11-11 2017-11-10 用于基因治疗的结构和方法 Pending CN110214005A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662421103P 2016-11-11 2016-11-11
US62/421,103 2016-11-11
US201762485493P 2017-04-14 2017-04-14
US62/485,493 2017-04-14
PCT/US2017/061111 WO2018089799A1 (en) 2016-11-11 2017-11-10 Structures and methods for gene therapy

Publications (1)

Publication Number Publication Date
CN110214005A true CN110214005A (zh) 2019-09-06

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CN201780083137.XA Pending CN110214005A (zh) 2016-11-11 2017-11-10 用于基因治疗的结构和方法

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US (1) US20190351071A1 (https=)
EP (1) EP3538075A4 (https=)
JP (1) JP2020500218A (https=)
CN (1) CN110214005A (https=)
WO (1) WO2018089799A1 (https=)

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WO2023015901A1 (zh) * 2021-08-13 2023-02-16 广州谷森制药有限公司 新型阳离子脂质化合物
CN116113438A (zh) * 2020-09-22 2023-05-12 Bp基因有限公司 含新cd47结合剂和多核苷酸的癌症治疗用脂质体复合体
CN118351957A (zh) * 2024-06-18 2024-07-16 北京剂泰医药科技有限公司 可电离脂质分子的头尾识别方法、脂质双层膜的分子模拟方法、系统及计算机程序产品

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CN111712468A (zh) 2017-12-22 2020-09-25 北卡罗莱纳州立大学 聚合物荧光团、包含其的组合物及制备和使用其的方法
EP3914232A1 (en) * 2019-01-25 2021-12-01 F. Hoffmann-La Roche AG Lipid vesicle for oral drug delivery
BR112022002365A2 (pt) 2019-08-09 2022-04-26 Nutcracker Therapeutics Inc Métodos e aparelhos para fabricar e para remover material de uma composição terapêutica
US20230023615A1 (en) * 2019-12-13 2023-01-26 Dnalite Therapeutics, Inc. Compositions and methods for biological delivery vehicles
WO2021134023A2 (en) * 2019-12-24 2021-07-01 Ajk Pharmaceutical Llc Compositions and methods for nucleic acid delivery
US20230143984A1 (en) * 2020-03-20 2023-05-11 Heidelberg University Colloidal carrier systems for transfer of agents to a desired site of action
WO2024168347A1 (en) * 2023-02-10 2024-08-15 Genprex, Inc. Treatment of cancer using combined tusc2/tusc4 gene therapy

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Publication number Priority date Publication date Assignee Title
CN116113438A (zh) * 2020-09-22 2023-05-12 Bp基因有限公司 含新cd47结合剂和多核苷酸的癌症治疗用脂质体复合体
WO2023015901A1 (zh) * 2021-08-13 2023-02-16 广州谷森制药有限公司 新型阳离子脂质化合物
CN118351957A (zh) * 2024-06-18 2024-07-16 北京剂泰医药科技有限公司 可电离脂质分子的头尾识别方法、脂质双层膜的分子模拟方法、系统及计算机程序产品
CN118351957B (zh) * 2024-06-18 2024-09-06 北京剂泰医药科技有限公司 可电离脂质分子的头尾识别方法、脂质双层膜的分子模拟方法、系统及计算机程序产品

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