# New HPBCD Mixtures for Treating Niemann-Pick Disease Type C

> 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.

- **Patent:** US 12734190
- **Original title:** Hydroxypropyl beta-cyclodextrin compositions and methods
- **Granted:** 2026
- **Status:** Active
- **Times cited:** 0
- **Field:** pharmaceutical, biotech, rare_diseases, neurology, drug_delivery

## 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.

## The clever bit

The clever aspect lies in defining specific mixtures of hydroxypropyl beta-cyclodextrin molecules, including their substitution patterns, and then providing rigorous methods to qualify these mixtures for safe and effective direct delivery into the brain or spinal fluid. This level of precision and quality control is essential for a drug administered to such a sensitive and vital area of the body.

## Real-world examples

1. VTS-270 (HPBCD for Niemann-Pick Type C)
2. Investigational therapies for Niemann-Pick disease Type C
3. Drugs requiring intrathecal administration for neurological conditions

## Why it matters

Niemann-Pick disease Type C is a severe and rare genetic disorder where cholesterol builds up in cells, particularly affecting the brain and causing progressive neurological damage. Effective treatments are scarce, and delivering drugs directly to the central nervous system is a major challenge. This patent is significant because it aims to provide precisely defined and qualified HPBCD formulations for direct brain delivery, which is crucial for effectively addressing the neurological symptoms of NPC. Developing such specific compositions and administration methods could lead to improved therapeutic options for patients facing this devastating disease.

## Frequently asked questions

### What does New HPBCD Mixtures for Treating Niemann-Pick Disease Type C cover?

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.

### When does this patent expire?

This patent is expected to expire on September 15, 2046, when the invention enters the public domain.

### What problem does this patent solve?

Niemann-Pick disease Type C is a severe and rare genetic disorder where cholesterol builds up in cells, particularly affecting the brain and causing progressive neurological damage. Effective treatments are scarce, and delivering drugs directly to the central nervous system is a major challenge. This patent is significant because it aims to provide precisely defined and qualified HPBCD formulations for direct brain delivery, which is crucial for effectively addressing the neurological symptoms of NPC. Developing such specific compositions and administration methods could lead to improved…

### What does this patent NOT cover?

Does not cover other types of cyclodextrins (e.g., alpha-cyclodextrin, gamma-cyclodextrin) or other chemical modifications besides hydroxypropyl groups.

**Full plain-English explainer:** https://patentbrief.org/patent/us/12734190/hydroxypropyl-beta-cyclodextrin-compositions-and-methods

**Original patent:** https://patents.google.com/patent/US12734190

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_Source: PatentBrief — https://patentbrief.org. Patent facts are from public records; the plain-English explanation is PatentBrief's._