EA202192328A1 - Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления - Google Patents

Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления

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Publication number
EA202192328A1
EA202192328A1 EA202192328A EA202192328A EA202192328A1 EA 202192328 A1 EA202192328 A1 EA 202192328A1 EA 202192328 A EA202192328 A EA 202192328A EA 202192328 A EA202192328 A EA 202192328A EA 202192328 A1 EA202192328 A1 EA 202192328A1
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EA
Eurasian Patent Office
Prior art keywords
treatment
storage diseases
gemfibrosil
oleamide
lysosomal storage
Prior art date
Application number
EA202192328A
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English (en)
Inventor
Калипада Пахан
Original Assignee
Раш Юниверсити Медикал Сентер
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Application filed by Раш Юниверсити Медикал Сентер filed Critical Раш Юниверсити Медикал Сентер
Publication of EA202192328A1 publication Critical patent/EA202192328A1/ru

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    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • 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
    • A61K31/20—Carboxylic acids, e.g. valproic acid having a carboxyl group bound to a chain of seven or more carbon atoms, e.g. stearic, palmitic, arachidic acids
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    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Abstract

Здесь предложены способы лечения лизосомных болезней накопления, включающие введение генов, кодирующих лизосомальный фермент, и фармацевтического агента. Комбинирование генной терапии с фармацевтическими композициями посредством совместного введения не только дополнительно усиливает эффекты, оказываемые ими по отдельности, но также обеспечивает комплексный подход к лечению благодаря разным механизмам действия каждой отдельной композиции.
EA202192328A 2019-03-22 2020-03-20 Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления EA202192328A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962822310P 2019-03-22 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
EA202192328A1 true EA202192328A1 (ru) 2022-02-21

Family

ID=72609912

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192328A EA202192328A1 (ru) 2019-03-22 2020-03-20 Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления

Country Status (9)

Country Link
US (1) US20220152165A1 (ru)
EP (1) EP3941584A4 (ru)
JP (1) JP2022525888A (ru)
KR (1) KR20210143848A (ru)
CN (1) CN113811359A (ru)
AU (1) AU2020245415A1 (ru)
CA (1) CA3132379A1 (ru)
EA (1) EA202192328A1 (ru)
WO (1) WO2020197967A1 (ru)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK1513519T3 (da) * 2002-06-03 2009-06-02 Novartis Ag Anvendelse af substituerede cyanopyrrolidiner til behandling af hyperlipidæmi
US9750712B2 (en) * 2012-12-07 2017-09-05 Rush University Medical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US11045557B2 (en) * 2016-06-09 2021-06-29 Queen's University At Kingston Methods and gene therapy constructs for treating GM2 gangliosidoses
CN109475619A (zh) * 2016-06-13 2019-03-15 蓝鸟生物公司 神经元蜡样脂褐质沉积症的基因治疗
AU2017284198B2 (en) * 2016-06-13 2023-09-28 The University Of North Carolina At Chapel Hill Optimized CLN1 genes and expression cassettes and their use
JP2020505317A (ja) * 2017-01-20 2020-02-20 ユニバーシティ オブ ピッツバーグ − オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

Also Published As

Publication number Publication date
EP3941584A4 (en) 2022-11-30
CA3132379A1 (en) 2020-10-01
WO2020197967A1 (en) 2020-10-01
KR20210143848A (ko) 2021-11-29
AU2020245415A1 (en) 2021-09-16
EP3941584A1 (en) 2022-01-26
JP2022525888A (ja) 2022-05-20
CN113811359A (zh) 2021-12-17
US20220152165A1 (en) 2022-05-19

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