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A Targeted Solution for Mitochondrial Diseases: Hope and Innovation for Rare Genetic Disorders

Introduction

Mitochondrial diseases represent some of the most challenging medical conditions, affecting patients on a cellular level and causing severe, often life-altering symptoms. Current treatment options are limited, leaving patients and healthcare providers without effective long-term solutions. Our innovative treatment offers a breakthrough in addressing mitochondrial dysfunction, providing a targeted approach that brings real hope to individuals and families affected by these disorders.

The Problem

Mitochondrial diseases occur when mitochondria, the “powerhouses” of the cell, fail to function properly. These disorders can manifest in a wide range of symptoms, from muscle weakness and neurological problems to multi-system organ failure. Due to the complexity of mitochondrial function and the genetic variability of these diseases, traditional treatments have been largely ineffective in providing lasting relief or addressing the root cause of the problem.

The Solution

Our patented treatment represents a groundbreaking advancement in mitochondrial disease therapy. By specifically targeting dysfunctional mitochondria, this therapy works at the cellular level to improve mitochondrial performance, reduce symptoms, and slow disease progression. It goes beyond symptom management, aiming to address the underlying dysfunction in the mitochondria itself.

Why This Technology Stands Out

  1. Precision Treatment: The therapy is designed to directly target the mitochondria, providing a focused approach that enhances its ability to generate energy and maintain cellular health. This precision sets it apart from generalized treatments that fail to address the core issue.
  2. Wide-Ranging Impact: Mitochondrial diseases affect multiple systems in the body. By improving mitochondrial function, this treatment has the potential to alleviate symptoms across a broad range of organ systems, improving quality of life for patients with complex, multi-system disorders.
  3. Innovative Mechanism of Action: This treatment utilizes novel mechanisms to enhance mitochondrial function, opening up new pathways for future research and combination therapies.

The Opportunity

Licensing this technology offers pharmaceutical companies and research institutions a unique opportunity to lead in a largely underserved market. With a growing understanding of mitochondrial diseases and the need for effective treatments, this innovation positions your company to be at the forefront of genetic and mitochondrial therapy.

This is more than a treatment—it’s a transformative approach that brings real hope to a patient population in desperate need of solutions.

Please see terms and conditions
The present application relates to methods of treating a subject with a disease or disorder associated with a mitochondrial deficit, comprising: determining at least one of a type or degree of the mitochondrial deficit in the subject; and administering an amount of a butyrate compound to the subject based at least partially on the determining step so as to promote Adenosine triphosphate (ATP) production in the subject as well as to methods of screening for compounds that restore cellular mitochondrial activity.

We claim:

1. A method of treating a subject with a disease or disorder associated with a mitochondrial deficit, comprising:

determining at least one of a type or degree of said mitochondrial deficit in said subject; and
administering an effective amount of a butyrate compound to said subject based at least partially on said determining so as to promote Adenosine triphosphate (ATP) production in said subject;
wherein said butyrate compound is selected from the group of butyrate compounds consisting of sodium butyrate, 1,3-Di(butanoyloxy)propan-2-yl butanoate and pivaloyloxym ethyl butyrate;
wherein said disease or disorder is Mitochondrial Encephalopathy with Lactic Acidosis and Stroke-like episodes (MELAS), Myoclonic Epilepsy with Ragged-Red Fibers (MERFF), or a mitochondrial respiratory disorder with a deficit in ATP levels due to a genetic mutation in a mitochondrial or nuclear genome.
2. The method of claim 1, wherein said administering an amount of said butyrate compound results in synergizing a mitochondrial-nuclear crosstalk by promoting nuclear reprogramming and mitochondrial metabolic reprogramming.
3. The method of claim 2, wherein said administering an amount of said butyrate compound results in an increase in mitochondrial mass and biogenesis.
4. The method of claim 2, wherein said administering an amount of said butyrate compound results in increased mitochondrial membrane potential and bioenergetics.
5. The method of claim 1, wherein said administering an amount of said butyrate compound results in a decrease of diseased mitochondria.
6. The method of claim 1, wherein said butyrate compound is administered in an amount of about 150 mg/Kg/day to about 500 mg/Kg/day.
7. The method of claim 1, wherein said butyrate compound is administered for at least 3 days.
8. The method of claim 7, further comprising observing a resting period following administration of said butyrate compound and prior to administration of a subsequent dose of said butyrate compound.
9. The method of claim 1, wherein said butyrate compound is administered orally, intravenously, or by injection.
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Title

Treatment for mitochondrial diseases

Inventor(s)

Anne Chiaramello, Martine Uittenbogaard

Assignee(s)

George Washington University

Patent #

10272056

Patent Date

April 30, 2019

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