KR20210143848A - 리소좀 축적 장애를 위한 비강 유전자 전달 및 경구 신남산, 올레아미드 또는 젬피브로질의 병용 - Google Patents

리소좀 축적 장애를 위한 비강 유전자 전달 및 경구 신남산, 올레아미드 또는 젬피브로질의 병용 Download PDF

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KR20210143848A
KR20210143848A KR1020217034175A KR20217034175A KR20210143848A KR 20210143848 A KR20210143848 A KR 20210143848A KR 1020217034175 A KR1020217034175 A KR 1020217034175A KR 20217034175 A KR20217034175 A KR 20217034175A KR 20210143848 A KR20210143848 A KR 20210143848A
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composition
disease
gene
administered
lysosomal storage
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칼리파다 파한
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러쉬 유니버시티 메디컬 센터
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    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61K31/192—Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid 
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    • A61K31/19—Carboxylic acids, e.g. valproic acid
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KR1020217034175A 2019-03-22 2020-03-20 리소좀 축적 장애를 위한 비강 유전자 전달 및 경구 신남산, 올레아미드 또는 젬피브로질의 병용 Pending KR20210143848A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962822310P 2019-03-22 2019-03-22
US62/822,310 2019-03-22
PCT/US2020/023768 WO2020197967A1 (en) 2019-03-22 2020-03-20 Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders

Publications (1)

Publication Number Publication Date
KR20210143848A true KR20210143848A (ko) 2021-11-29

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KR1020217034175A Pending KR20210143848A (ko) 2019-03-22 2020-03-20 리소좀 축적 장애를 위한 비강 유전자 전달 및 경구 신남산, 올레아미드 또는 젬피브로질의 병용

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Country Link
US (1) US20220152165A1 (https=)
EP (1) EP3941584A4 (https=)
JP (1) JP2022525888A (https=)
KR (1) KR20210143848A (https=)
CN (1) CN113811359A (https=)
AU (1) AU2020245415B2 (https=)
CA (1) CA3132379A1 (https=)
EA (1) EA202192328A1 (https=)
WO (1) WO2020197967A1 (https=)

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CA3121785A1 (en) * 2018-12-05 2020-06-11 Washington University Methods of detecting, preventing, reversing, and treating neurological diseases
CN116723870A (zh) * 2021-01-20 2023-09-08 拉什大学医学中心 球形细胞脑白质营养不良症或克拉伯病的改良治疗
JP2026504932A (ja) * 2023-01-18 2026-02-10 ラッシュ・ユニバーシティ・メディカル・センター Ｃｌｎ３関連若年型神経セロイドリポフスチン症の治療のための経口ゲムフィブロジル

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CN100402026C (zh) * 2002-06-03 2008-07-16 诺瓦提斯公司 取代的氰基吡咯烷和含有它们的组合制剂用于治疗高脂血症和相关疾病的用途
WO2008049058A2 (en) * 2006-10-18 2008-04-24 Cornell Research Foundation, Inc. Cln2 treatment of alzheimer's disease
WO2012177997A1 (en) * 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2016081365A1 (en) * 2014-11-19 2016-05-26 Rush University Medical Center Compositions and methods for treating lysosomal disorders
CN107249646B (zh) * 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
HK1256341A1 (zh) * 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
US20180236105A1 (en) * 2015-10-23 2018-08-23 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
US11045557B2 (en) * 2016-06-09 2021-06-29 Queen's University At Kingston Methods and gene therapy constructs for treating GM2 gangliosidoses
US20220323612A1 (en) * 2016-06-13 2022-10-13 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
RU2764919C2 (ru) * 2016-06-13 2022-01-24 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены и экспрессионные кассеты cln1, и их применение
BR112019010210A2 (pt) * 2016-12-29 2019-09-03 Rush University Medical Center melhora na atividade locomotora e aumento da longevidade de indivíduos com lipofuscinose ceroide neuronal infantil tardia por gemfibrozil
US12599636B2 (en) * 2017-01-20 2026-04-14 University of Pittsburgh—of the Commonwealth System of Higher Education Treatment of Krabbe disease with umbilical cord blood transplantion (UCBT) and increased galactocerebrosidase (GALC) expression
US12454701B2 (en) * 2018-12-05 2025-10-28 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
MX2021009250A (es) * 2019-02-01 2021-11-04 Spark Therapeutics Inc Metodos de tratamiento por vectores aav para la lipofuscinosis ceroidea neuronal infantil tardia tipo 2.

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WO2020197967A1 (en) 2020-10-01
JP2022525888A (ja) 2022-05-20
CA3132379A1 (en) 2020-10-01
US20220152165A1 (en) 2022-05-19
EA202192328A1 (ru) 2022-02-21
AU2020245415B2 (en) 2025-06-26
CN113811359A (zh) 2021-12-17
EP3941584A1 (en) 2022-01-26
AU2020245415A1 (en) 2021-09-16
EP3941584A4 (en) 2022-11-30

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