HYDROXYPROPYL BETA-CYCLODEXTRIN COMPOSITIONS AND METHODS

    公开(公告)号:US20240180957A1

    公开(公告)日:2024-06-06

    申请号:US18533373

    申请日:2023-12-08

    申请人: Mandos LLC

    IPC分类号: A61K31/724 A61K9/00 A61K9/08

    摘要: This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.