Treating Niemann-Pick Type C with a Special Sugar Mixture
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.
Original patent title: “Hydroxypropyl beta-cyclodextrin compositions and methods”
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. Granted in 2026.
Coverage
What does this patent actually cover?
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.
The gap
What does this patent 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.
These exclusions are unique to PatentBrief — derived from the actual claim language, not patent-office boilerplate.
Key facts
What made this novel
The noveltynoveltyThe requirement that an invention be different from anything publicly known before its priority date.Read more → lies in the specific composition of hydroxypropyl beta-cyclodextrin mixtures, carefully formulated and qualified for direct administration into the central nervous system, to effectively address the unique challenges of treating Niemann-Pick disease Type C.
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
Pharmaceutical formulations for rare neurological diseases
Drugs delivered directly into the spinal fluid
Therapies for lysosomal storage disorders
Why it matters
The bigger picture
Niemann-Pick disease Type C is a rare, severe genetic disorder that causes cholesterol to build up in cells, leading to progressive neurological damage and often early death. Current treatments are limited, and delivering drugs directly to the brain or spinal fluid is crucial for many neurological conditions. This patent aims to provide a specific, safe, and effective formulation for this challenging delivery route and disease.
Filed
December 2, 2025
Granted
September 15, 2026
Market context
Who's building on this
Companies in this space
Pharmaceutical companies focused on rare neurological disorders and advanced drug delivery methods, particularly those researching treatments for lysosomal storage diseases, are likely to be interested in or building upon this type of technology. Companies like Sanofi, Takeda, and PTC Therapeutics have programs in rare disease therapies that could intersect with this area.
Market impact
This patent could enable the development of new, more effective therapies for Niemann-Pick disease Type C, a condition with significant unmet medical need. By focusing on specific administration routes, it could open new avenues for drug delivery to the central nervous system, potentially impacting the treatment landscape for other neurological disorders requiring direct brain or spinal fluid access.
Claim 1 — Plain English
What this patent covers
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.
The clever bit
The novelty lies in the specific composition of hydroxypropyl beta-cyclodextrin mixtures, carefully formulated and qualified for direct administration into the central nervous system, to effectively address the unique challenges of treating 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.
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). Treating Niemann-Pick Type C with a Special Sugar Mixture (U.S. Patent No. 12,734,191). U.S. Patent and Trademark Office. https://patentbrief.org/patent/us/12734191/hydroxypropyl-beta-cyclodextrin-compositions-and-methods-12734191
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 Treating Niemann-Pick Type C with a Special Sugar Mixture cover?
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.
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 rare, severe genetic disorder that causes cholesterol to build up in cells, leading to progressive neurological damage and often early death. Current treatments are limited, and delivering drugs directly to the brain or spinal fluid is crucial for many neurological conditions. This patent aims to provide a specific, safe, and effective formulation for this challenging delivery route and disease.
What does this patent NOT cover?
Does not cover beta-cyclodextrin mixtures that are not modified with hydroxypropyl groups, unless they are included as an optional component.
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