CN113613633A - 用于cns递送的融合剂脂质体组合物 - Google Patents

用于cns递送的融合剂脂质体组合物 Download PDF

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Publication number
CN113613633A
CN113613633A CN201980088528.XA CN201980088528A CN113613633A CN 113613633 A CN113613633 A CN 113613633A CN 201980088528 A CN201980088528 A CN 201980088528A CN 113613633 A CN113613633 A CN 113613633A
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cell
fusogenic
liposome
fusogenic liposome
target cell
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CN201980088528.XA
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Chinese (zh)
Inventor
G.A.冯马尔特扎恩
J.R.鲁本斯
J.V.沙
A.鲁佐马蒂亚斯
F.普奇
J.M.米尔维德
M.T.米
N.F.戈登
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Flagship Pioneering Innovations V Inc
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Flagship Pioneering Innovations V Inc
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    • 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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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • 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
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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
    • A61K48/0033—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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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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CN201980088528.XA 2018-11-14 2019-11-14 用于cns递送的融合剂脂质体组合物 Pending CN113613633A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862767358P 2018-11-14 2018-11-14
US62/767,358 2018-11-14
US201962900064P 2019-09-13 2019-09-13
US62/900,064 2019-09-13
PCT/US2019/061424 WO2020102499A2 (en) 2018-11-14 2019-11-14 Fusosome compositions for cns delivery

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Publication Number Publication Date
CN113613633A true CN113613633A (zh) 2021-11-05

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CN201980088528.XA Pending CN113613633A (zh) 2018-11-14 2019-11-14 用于cns递送的融合剂脂质体组合物

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US (1) US20220008557A1 (https=)
EP (1) EP3880179A2 (https=)
JP (2) JP7676305B2 (https=)
KR (1) KR20210133948A (https=)
CN (1) CN113613633A (https=)
AU (1) AU2019378881A1 (https=)
CA (1) CA3120082A1 (https=)
IL (1) IL283179A (https=)
SG (1) SG11202105079QA (https=)
WO (1) WO2020102499A2 (https=)

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