JP7637058B2 - Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス - Google Patents

Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス Download PDF

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JP7637058B2
JP7637058B2 JP2021549291A JP2021549291A JP7637058B2 JP 7637058 B2 JP7637058 B2 JP 7637058B2 JP 2021549291 A JP2021549291 A JP 2021549291A JP 2021549291 A JP2021549291 A JP 2021549291A JP 7637058 B2 JP7637058 B2 JP 7637058B2
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ヒンデラー，クリスチャン
ミラー，ニムロッド
ウイルソン，ジェームス・エム
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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JP2021549291A 2019-02-22 2020-02-21 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス Active JP7637058B2 (ja)

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JP2025022078A JP2025081439A (ja) 2019-02-22 2025-02-14 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス

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US201962809329P 2019-02-22 2019-02-22
US62/809,329 2019-02-22
US201962923812P 2019-10-21 2019-10-21
US62/923,812 2019-10-21
US202062969108P 2020-02-02 2020-02-02
US62/969,108 2020-02-02
PCT/US2020/019149 WO2020172490A1 (en) 2019-02-22 2020-02-21 Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration

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JPWO2020172490A5 JPWO2020172490A5 (enExample) 2023-02-17
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JP2025022078A Pending JP2025081439A (ja) 2019-02-22 2025-02-14 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス

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US (1) US12416017B2 (enExample)
EP (1) EP3927381A1 (enExample)
JP (2) JP7637058B2 (enExample)
KR (1) KR20210131370A (enExample)
CN (1) CN113710281A (enExample)
AU (1) AU2020225472A1 (enExample)
BR (1) BR112021015817A2 (enExample)
CA (1) CA3129672A1 (enExample)
CL (1) CL2021002172A1 (enExample)
CO (1) CO2021011040A2 (enExample)
IL (1) IL285654A (enExample)
MX (1) MX2021010134A (enExample)
PE (1) PE20211819A1 (enExample)
SG (1) SG11202108504YA (enExample)
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WO (1) WO2020172490A1 (enExample)

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CN115715327A (zh) * 2019-10-22 2023-02-24 应用遗传科技公司 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
EP4204014A1 (en) * 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
WO2022131322A1 (ja) * 2020-12-17 2022-06-23 国立大学法人京都大学 神経細胞賦活剤
EP4473123A2 (en) * 2022-02-01 2024-12-11 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
WO2024245120A1 (en) * 2023-05-26 2024-12-05 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating proteinopathies in central nervous system
AU2024311750A1 (en) * 2023-06-21 2025-12-18 Eli Lilly And Company Compounds for the delivery of granulin across the blood brain barrier
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SG11202108504YA (en) 2021-09-29
PE20211819A1 (es) 2021-09-14
MX2021010134A (es) 2021-09-23
KR20210131370A (ko) 2021-11-02
BR112021015817A2 (pt) 2021-10-13
IL285654A (en) 2021-09-30
JP2025081439A (ja) 2025-05-27
CO2021011040A2 (es) 2021-09-09
AU2020225472A1 (en) 2021-08-26
US12416017B2 (en) 2025-09-16
US20220136008A1 (en) 2022-05-05
JP2022523766A (ja) 2022-04-26
TW202045730A (zh) 2020-12-16
WO2020172490A1 (en) 2020-08-27
CL2021002172A1 (es) 2022-02-11
CA3129672A1 (en) 2020-08-27
CN113710281A (zh) 2021-11-26
EP3927381A1 (en) 2021-12-29

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