Treating Lysosomal Storage Disease Symptoms with Quinuclidine Compounds
This patent describes methods for using specific quinuclidine compounds, sometimes with enzyme replacement therapy, to treat symptoms like pain and skin issues in patients with lysosomal storage diseases such as Fabry disease.
Original patent title: “Methods for treating symptoms and disorders associated with lysosomal storage diseases”
This patent describes methods for using specific quinuclidine compounds, sometimes with enzyme replacement therapy, to treat symptoms like pain and skin issues in patients with lysosomal storage diseases such as Fabry disease. Granted in 2026.
Coverage
What does this patent actually cover?
This patent describes ways to treat or prevent specific symptoms and disorders linked to lysosomal storage diseases. It focuses on using 'quinuclidine compounds of formula (I)' for this purpose. The treatment can optionally be combined with enzyme replacement therapy. Examples of symptoms it aims to treat include pain, like abdominal pain, and skin problems such as angiokeratoma. The methods are intended for patients with diseases like Fabry disease.
The gap
What does this patent NOT cover?
- Does not cover treatments using compounds other than quinuclidine compounds of formula (I).
- Does not cover treatments for diseases that are not classified as lysosomal storage diseases.
- Does not cover treatments for symptoms unrelated to pain or dermatological disorders in these diseases.
- Does not cover treatments for lysosomal storage diseases that do not involve either quinuclidine compounds or enzyme replacement therapy.
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 identifying a specific class of chemical compounds, 'quinuclidine compounds of formula (I)', for treating particular symptoms of lysosomal storage diseases. This suggests a targeted approach to address specific manifestations like pain and skin issues, potentially offering a new therapeutic pathway.
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 treatments for rare genetic disorders
Drug therapies for Fabry disease
Enzyme replacement therapy adjuncts
Why it matters
The bigger picture
Lysosomal storage diseases are a group of rare, inherited metabolic disorders that can cause severe health problems, including chronic pain and organ damage. New therapeutic approaches, especially those targeting specific symptoms, can significantly improve the quality of life for affected patients. Fabry disease, for example, is a serious condition where effective symptom management is crucial.
Filed
February 3, 2020
Granted
September 15, 2026
Market context
Who's building on this
Companies in this space
Pharmaceutical companies specializing in rare diseases, such as Sanofi Genzyme, Takeda Pharmaceutical Company, and Amicus Therapeutics, are actively researching and developing treatments for lysosomal storage diseases. These companies often explore novel small molecule compounds and combination therapies to address unmet patient needs.
Market impact
This patent could contribute to the development of new therapeutic options for lysosomal storage diseases, a market with significant unmet medical needs. If successful, it could enable new product lines for pharmaceutical companies, offering improved symptom management for conditions like Fabry disease and potentially reducing the burden on patients and healthcare systems.
Claim 1 — Plain English
What this patent covers
This patent describes ways to treat or prevent specific symptoms and disorders linked to lysosomal storage diseases. It focuses on using 'quinuclidine compounds of formula (I)' for this purpose. The treatment can optionally be combined with enzyme replacement therapy. Examples of symptoms it aims to treat include pain, like abdominal pain, and skin problems such as angiokeratoma. The methods are intended for patients with diseases like Fabry disease.
The clever bit
The novelty lies in identifying a specific class of chemical compounds, 'quinuclidine compounds of formula (I)', for treating particular symptoms of lysosomal storage diseases. This suggests a targeted approach to address specific manifestations like pain and skin issues, potentially offering a new therapeutic pathway.
What it does not cover
- Does not cover treatments using compounds other than quinuclidine compounds of formula (I).
- Does not cover treatments for diseases that are not classified as lysosomal storage diseases.
- Does not cover treatments for symptoms unrelated to pain or dermatological disorders in these diseases.
- Does not cover treatments for lysosomal storage diseases that do not involve either quinuclidine compounds or enzyme replacement therapy.
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 · 13.3 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 Lysosomal Storage Disease Symptoms with Quinuclidine Compounds (U.S. Patent No. 12,734,160). U.S. Patent and Trademark Office. https://patentbrief.org/patent/us/12734160/methods-for-treating-symptoms-and-disorders-associated-with-lysosomal-storage
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Common Questions
Frequently Asked Questions
What does Treating Lysosomal Storage Disease Symptoms with Quinuclidine Compounds cover?
This patent describes methods for using specific quinuclidine compounds, sometimes with enzyme replacement therapy, to treat symptoms like pain and skin issues in patients with lysosomal storage diseases such as Fabry disease.
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?
Lysosomal storage diseases are a group of rare, inherited metabolic disorders that can cause severe health problems, including chronic pain and organ damage. New therapeutic approaches, especially those targeting specific symptoms, can significantly improve the quality of life for affected patients. Fabry disease, for example, is a serious condition where effective symptom management is crucial.
What does this patent NOT cover?
Does not cover treatments using compounds other than quinuclidine compounds of formula (I).
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