{
  "patent_number": "US 12734191",
  "country": "US",
  "title": "Treating Niemann-Pick Type C with a Special Sugar Mixture",
  "original_title": "Hydroxypropyl beta-cyclodextrin compositions and methods",
  "summary": "This patent describes specific mixtures of modified beta-cyclodextrin molecules designed for direct delivery to the brain to treat the rare genetic disorder Niemann-Pick disease Type C.",
  "what_it_does": "This patent focuses on specific mixtures of beta-cyclodextrin molecules, which are ring-shaped sugar molecules. These molecules are modified by adding 'hydroxypropyl' groups to one or more of their hydroxyl positions. The mixture can also include some unmodified beta-cyclodextrin. These special mixtures are intended to be used as an active ingredient in medicines. The patent also covers how to make these mixtures, how to test them to ensure they are safe for use, and how to use them in pharmaceutical compositions. Specifically, these compositions are designed for delivery directly into the spinal fluid (intrathecal) or brain ventricles (intracerebroventricular) to treat Niemann-Pick disease Type C.",
  "what_it_does_not_cover": [
    "Does not cover beta-cyclodextrin mixtures that are not modified with hydroxypropyl groups, unless they are included as an optional component.",
    "Does not cover using these compositions for diseases other than Niemann-Pick disease Type C.",
    "Does not cover drug delivery methods other than intrathecal or intracerebroventricular administration.",
    "Does not cover other types of cyclodextrin molecules, such as alpha-cyclodextrin or gamma-cyclodextrin.",
    "Does not cover compositions that do not include at least some hydroxypropyl beta-cyclodextrin molecules."
  ],
  "filed": "2025-12-02",
  "granted": "2026-09-15",
  "expires": null,
  "status": "active",
  "holder": null,
  "holder_url": null,
  "inventors": [],
  "times_cited": 0,
  "tags": [
    "pharmaceutical",
    "biotech",
    "neuroscience"
  ],
  "abstract": "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.",
  "url": "https://patentbrief.org/patent/us/12734191/hydroxypropyl-beta-cyclodextrin-compositions-and-methods-12734191",
  "markdown_url": "https://patentbrief.org/patent/us/12734191/hydroxypropyl-beta-cyclodextrin-compositions-and-methods-12734191/md",
  "google_patents_url": "https://patents.google.com/patent/US12734191",
  "relatedPatents": []
}