COMPOSITION FOR TREATMENT, PREVENTION, OR IMPROVEMENT OF DISEASE OR DISORDER DUE TO INTRACELLULAR CHOLESTEROL TRAFFICKING DISORDER

WIPO Patent Application WO/2024/090558

A1

Provided is a composition for treatment and the like of a disease and the like due to an intracellular cholesterol trafficking disorder which is effective as an NPC therapeutic agent, said composition being safer than conventional 2-hydroxypropyl-β-cyclodextrin while providing an equivalent or greater therapeutic effect.　This composition for treatment, prevention, or improvement of a disease or a disorder due to an intracellular cholesterol trafficking disorder contains 2-hydroxypropyl-β-cyclodextrin in which a 2-hydroxypropyl group is substituted for an OH group of β-cyclodextrin, wherein the average degree of substitution of the 2-hydroxypropyl group is 13-25.

ISHITSUKA YOUICHI (JP)
ISHII AKIRA (JP)
KONDO YUKI (JP)
ORITA YORIHISA (JP)
ISHIKURA KANDAI (JP)
YANAGIHARA KAZUNORI (JP)
TAKAGI HIROKI (JP)

PCT/JP2023/038897

May 02, 2024

October 27, 2023

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UNIV KUMAMOTO NAT UNIV CORP (JP)
JAPAN MAIZE PROD (JP)

A61K31/724; A61P3/06

WO2015083736A1	2015-06-11
WO2023156966A1	2023-08-24

JP2018517046A	2018-06-28
JP2021529243A	2021-10-28

YAMADA, YUSEI ET AL.: "Fine-tuned cholesterol solubilizer, mono-6-0-cc-D-maltosyl-,I- cyclodextrin, ameliorates experimental Niemann-Pick disease type C without hearing loss", BIOMEDICINE & PHARMACOTHERAPY, vol. 155, 16 September 2022 (2022-09-16), pages 1 - 12, XP087201528, DOI: 10.1016/j.biopha.2022.113698

DELOITTE TOHMATSU IP FIRM (JP)

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