{
  "patent_number": "US 12734190",
  "country": "US",
  "title": "New HPBCD Mixtures for Treating Niemann-Pick Disease Type C",
  "original_title": "Hydroxypropyl beta-cyclodextrin compositions and methods",
  "summary": "This patent describes specific mixtures of a modified sugar molecule, hydroxypropyl beta-cyclodextrin, 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 defining specific mixtures of a modified sugar called hydroxypropyl beta-cyclodextrin (HPBCD). These mixtures consist of beta-cyclodextrin molecules that have hydroxypropyl groups attached at one or more hydroxyl positions, and they can optionally include some unmodified beta-cyclodextrin molecules. The disclosure also covers methods for creating these precise mixtures and procedures for 'qualifying' them, which means testing them to ensure they are suitable for pharmaceutical use. These specialized HPBCD mixtures are intended for use in pharmaceutical compositions that can be administered directly into the brain or spinal fluid, specifically through intrathecal or intracerebroventricular routes. The ultimate purpose of these compositions is to treat Niemann-Pick disease Type C.",
  "what_it_does_not_cover": [
    "Does not cover other types of cyclodextrins (e.g., alpha-cyclodextrin, gamma-cyclodextrin) or other chemical modifications besides hydroxypropyl groups.",
    "Does not cover HPBCD compositions that are not specifically defined as 'mixtures' of substituted and optionally unsubstituted beta-cyclodextrin molecules.",
    "Does not cover HPBCD used for routes of administration other than intrathecal or intracerebroventricular, such as oral or intravenous delivery.",
    "Does not cover the use of HPBCD for treating diseases other than Niemann-Pick disease Type C.",
    "Does not cover methods of making or qualifying HPBCD that do not result in the specific mixtures described."
  ],
  "filed": "2025-12-02",
  "granted": "2026-09-15",
  "expires": null,
  "status": "active",
  "holder": null,
  "holder_url": null,
  "inventors": [],
  "times_cited": 0,
  "tags": [
    "pharmaceutical",
    "biotech",
    "rare_diseases",
    "neurology",
    "drug_delivery"
  ],
  "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/12734190/hydroxypropyl-beta-cyclodextrin-compositions-and-methods",
  "markdown_url": "https://patentbrief.org/patent/us/12734190/hydroxypropyl-beta-cyclodextrin-compositions-and-methods/md",
  "google_patents_url": "https://patents.google.com/patent/US12734190",
  "relatedPatents": []
}