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.
Original patent title: “Hydroxypropyl beta-cyclodextrin compositions and methods”
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. Granted in 2026.
Coverage
What does this patent actually cover?
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.
The gap
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.
- 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.
These exclusions are unique to PatentBrief — derived from the actual claim language, not patent-office boilerplate.
Key facts
What made this novel
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.
Schematic visualization of the patent's claim structure. Hand-drawn diagrams in progress for each landmark patent.
Where you've seen this
Real-world examples
VTS-270 (HPBCD for Niemann-Pick Type C)
Investigational therapies for Niemann-Pick disease Type C
Drugs requiring intrathecal administration for neurological conditions
Why it matters
The bigger picture
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.
Filed
December 2, 2025
Granted
September 15, 2026
Market context
Who's building on this
Companies in this space
Companies focused on rare neurological disorders, such as Mallinckrodt (which acquired Vtesse, developers of VTS-270 for NPC), are actively researching and developing therapies for Niemann-Pick disease Type C. Other biotech firms specializing in drug delivery to the central nervous system would also be interested in this area, given the challenges of delivering therapeutics to the brain.
Market impact
As a recently filed patent with a future grant dategrant dateThe day the USPTO officially granted the patent. Enforceability typically starts here.Read more →, its direct market impact is yet to be realized. However, if successfully developed and commercialized, this patent could establish new standards for the precise formulation and quality control of hydroxypropyl beta-cyclodextrin for treating Niemann-Pick disease Type C. This could potentially enable new drug products or improve existing ones, offering better treatment options for patients with this severe neurological condition by ensuring safer and more effective central nervous system delivery.
Claim 1 — Plain English
What this patent covers
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.
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.
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.
Patent timeline
Application submitted to the patent office
Patent officially issued
PatentBrief Score
Impact Score
Early stage
Citation count
0/40
No citations yet
Claim breadth
0/20
Narrow claimsclaimsThe numbered statements at the end of a patent that legally define what the inventor owns.Read more →
Recency
20/20
Granted within 5 years
Assignee scale
0/20
Independent or smaller assigneeassigneeThe entity that owns the patent — usually the inventor's employer or a company.Read more →
PatentBrief Impact Score — based on citation count, claim breadth, recency, and assignee scale. Not a legal assessment.
Heuristic Value Estimate
What this patent might be worth
$36K – $115K
Midpoint $72K · 19.2 yr remaining · industry ×3.0
Heuristic only — blends forward/backward citation counts, claim scope, time remaining, litigation history, and CPC-derived industry baseline. Real valuations need a professional appraisal.
Claim text not yet imported for this patent
Concepts involved
Cite this patent
(2026). New HPBCD Mixtures for Treating Niemann-Pick Disease Type C (U.S. Patent No. 12,734,190). U.S. Patent and Trademark Office. https://patentbrief.org/patent/us/12734190/hydroxypropyl-beta-cyclodextrin-compositions-and-methods
Auto-generated from the patent record. Double-check author order and the issue date against the official USPTO document before submitting.
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Common Questions
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.
Patent monitoring