JP2023501897A - Ｃ９ｏｒｆ７２関連疾患の治療のための三重機能アデノ随伴ウイルス（ａａｖ）ベクター - Google Patents

Ｃ９ｏｒｆ７２関連疾患の治療のための三重機能アデノ随伴ウイルス（ａａｖ）ベクター Download PDF

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JP2023501897A
JP2023501897A JP2022523436A JP2022523436A JP2023501897A JP 2023501897 A JP2023501897 A JP 2023501897A JP 2022523436 A JP2022523436 A JP 2022523436A JP 2022523436 A JP2022523436 A JP 2022523436A JP 2023501897 A JP2023501897 A JP 2023501897A
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c9orf72
nucleic acid
vector
sequence
aav
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ズー，ペイシン
ワン，シジア
ペノック，スティーヴン
シャーマン，マーク
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アプライド ジェネティック テクノロジーズ コーポレイション
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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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JP2022523436A 2019-10-22 2020-10-22 Ｃ９ｏｒｆ７２関連疾患の治療のための三重機能アデノ随伴ウイルス（ａａｖ）ベクター Pending JP2023501897A (ja)

Applications Claiming Priority (3)

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US201962924351P 2019-10-22 2019-10-22
US62/924,351 2019-10-22
PCT/US2020/056905 WO2021081236A1 (en) 2019-10-22 2020-10-22 Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases

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JP2023501897A true JP2023501897A (ja) 2023-01-20
JP2023501897A5 JP2023501897A5 (https=) 2023-10-30

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JP2022523436A Pending JP2023501897A (ja) 2019-10-22 2020-10-22 Ｃ９ｏｒｆ７２関連疾患の治療のための三重機能アデノ随伴ウイルス（ａａｖ）ベクター

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US (2) US20210147873A1 (https=)
EP (1) EP4048794A4 (https=)
JP (1) JP2023501897A (https=)
KR (1) KR20230019063A (https=)
CN (1) CN116134134A (https=)
AU (1) AU2020370291A1 (https=)
CA (1) CA3158518A1 (https=)
IL (1) IL292384A (https=)
MX (1) MX2022004771A (https=)
WO (1) WO2021081236A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3947700A4 (en) 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
BR112023006305A2 (pt) 2020-10-09 2023-05-09 Tenaya Therapeutics Inc Métodos e composições de terapia gênica com placofilina-2
EP4347822A2 (en) * 2021-06-04 2024-04-10 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
EP4427049A4 (en) * 2021-11-01 2025-08-20 Univ Florida POLY-GA PROTEINS IN ALZHEIMER'S DISEASE
WO2023133574A1 (en) * 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
CN120035669A (zh) * 2022-09-28 2025-05-23 阿塔兰塔治疗公司 用于治疗神经系统病症的组合物和方法
CN121057823A (zh) * 2023-04-10 2025-12-02 耶鲁大学 体内修饰神经元以治疗和/或预防肌萎缩性侧索硬化(als)的方法
WO2025085713A1 (en) * 2023-10-20 2025-04-24 Spark Therapeutics, Inc. Inhibitory nucleic acid targeting alpha synuclein expression

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019084068A1 (en) * 2017-10-23 2019-05-02 Prevail Therapeutics, Inc. GENE THERAPIES FOR NEURODEGENERATIVE DISEASE

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WO2012057363A1 (ja) * 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
US9096671B2 (en) * 2011-06-29 2015-08-04 Consejo Superior De Investigaciones Cientificas (Csic) LRP1 as key receptor for the transfer of sterified cholesterol from very-low-density lipoproteins (VLDL) to ischaemic cardiac muscle
US9896729B2 (en) * 2011-08-31 2018-02-20 The University Of Manchester Method for diagnosing a neurodegenerative disease
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019084068A1 (en) * 2017-10-23 2019-05-02 Prevail Therapeutics, Inc. GENE THERAPIES FOR NEURODEGENERATIVE DISEASE

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IL292384A (en) 2022-06-01
EP4048794A4 (en) 2024-04-17
WO2021081236A1 (en) 2021-04-29
MX2022004771A (es) 2022-10-07
AU2020370291A1 (en) 2022-05-12
US20210147873A1 (en) 2021-05-20
EP4048794A1 (en) 2022-08-31
US20240067984A1 (en) 2024-02-29
CN116134134A (zh) 2023-05-16
KR20230019063A (ko) 2023-02-07
CA3158518A1 (en) 2021-04-29

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