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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JP2023501897A5 (enExample
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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)

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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 (enExample) 2023-10-30

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

Families Citing this family (8)

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Publication number Priority date Publication date Assignee Title
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
MX2023014168A (es) * 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Marco de lectura abierto 72 del cromosoma humano 9 (c9orf72) composiciones de agente de arni y metodos de uso de este.
EP4427049A4 (en) * 2021-11-01 2025-08-20 Univ Florida POLY-GA PROTEINS IN ALZHEIMER'S DISEASE
TW202340467A (zh) * 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
WO2024073592A2 (en) * 2022-09-28 2024-04-04 Atalanta Therapeutics, Inc. Compositions and methods for treatment of neurological disorders
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
CN122374456A (zh) * 2023-10-20 2026-07-10 斯帕克医疗有限公司 靶向 α 突触核蛋白表达的抑制性核酸

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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Publication number Priority date Publication date Assignee Title
US10738326B2 (en) * 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
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
WO2013030588A1 (en) * 2011-08-31 2013-03-07 The University Of Manchester Method for diagnosing a neurodegenerative disease
WO2016168592A2 (en) * 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
AU2017234929B2 (en) * 2016-03-18 2024-05-02 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
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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EP4048794A1 (en) 2022-08-31
US20240067984A1 (en) 2024-02-29
EP4048794A4 (en) 2024-04-17
CN116134134A (zh) 2023-05-16
KR20230019063A (ko) 2023-02-07
CA3158518A1 (en) 2021-04-29
WO2021081236A1 (en) 2021-04-29
MX2022004771A (es) 2022-10-07
AU2020370291A1 (en) 2022-05-12
US20210147873A1 (en) 2021-05-20
IL292384A (en) 2022-06-01

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