Mitochondrial Augmentation Therapy for Ocular Diseases
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Solution Overview
Problem
There is a long-felt need for effective and long-term therapies for ocular diseases that are not primarily caused by mitochondrial diseases or disorders.
Innovation Solution
The use of human stem cells enriched with healthy and functional mitochondria, administered to patients suffering from ocular-related diseases and disorders, to provide alleviation of symptoms and improve eye function through mitochondria augmentation therapy.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional supportive therapy is used for mitochondrial diseases, then patient survival is maintained, but no effective treatment breakthrough is achieved
Solution Approach 1:
The patent uses stem cells as an intermediary vehicle to deliver healthy mitochondria to affected tissues. The stem cells are first enriched with functional mitochondria ex vivo, then administered to patients where they serve as mediators to transfer healthy mitochondria to cells with mitochondrial dysfunction, thereby achieving therapeutic effects without directly injecting mitochondria into the body.
Solution Approach 2:
The patent performs preliminary enrichment of stem cells with functional mitochondria before administration. This ex vivo pre-treatment involves culturing stem cells with isolated mitochondria to load them with healthy mitochondrial populations, ensuring the therapeutic agent is pre-loaded and optimized before delivery to the patient, which enhances treatment effectiveness.
2Reliability
If stem cells are enriched with functional mitochondria ex vivo, then mitochondrial function is improved, but treatment complexity increases
Solution Approach 1:
The patent employs the natural ability of stem cells to take up and incorporate external mitochondria during culture. Rather than using complex transfection or injection methods to introduce mitochondria, the stem cells self-service by naturally internalizing the isolated functional mitochondria during ex vivo culture, simplifying the enrichment process while maintaining high mitochondrial function.
3Loss of time
If a single round of treatment is administered, then treatment time is reduced, but long-term therapy efficacy is uncertain
Solution Approach 1:
The patent achieves continuous therapeutic action through the self-renewal and differentiation capabilities of stem cells. The administered stem cells continuously produce functional mitochondria and can differentiate into various tissue types, providing ongoing mitochondrial support and regeneration rather than a single transient effect, thus ensuring long-term therapeutic durability.
Data Source
AI summary
The present invention provides stem cells enriched with healthy functional mitochondria, pharmaceutical compositions comprising these cells and methods of use thereof for treating ocular diseases, disorders and symptoms thereof where the disease may or may not be associated with acquired mitochondrial dysfunction.


