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Compositions and methods for treating and preventing leber's hereditary optic neuropathy

An optic neuropathy, hereditary technology, applied in the field of compositions for the treatment and prevention of Leber hereditary optic neuropathy, capable of solving problems such as lack of resistance, permanent vision loss, etc.

Inactive Publication Date: 2021-06-01
IMBRIA PHARMA INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

So, despite our understanding of the molecular basis of LHON, we lack the tools to fight the disease, so many people continue to suffer permanent vision loss

Method used

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  • Compositions and methods for treating and preventing leber's hereditary optic neuropathy
  • Compositions and methods for treating and preventing leber's hereditary optic neuropathy
  • Compositions and methods for treating and preventing leber's hereditary optic neuropathy

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Experimental program
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Embodiment Construction

[0034] The present invention provides methods of treating and preventing Leber's hereditary optic neuropathy (LHON) by providing a composition comprising a TCA cycle intermediate, such as succinate, which has been modified to improve its dissolution sex. The compositions include compounds comprising TCA cycle intermediates conjugated to one or more amino acids, polyols, or both. This compound can be cleaved in vivo to release intermediates into the TCA cycle, resulting in succinate, which can enter the mitochondrial electron transport chain. As a result, mitochondrial respiration was less dependent on NADH dehydrogenase, an enzyme with reduced function in patients with LHON-causing mutations.

[0035] The TCA cycle looks like this:

[0036]

[0037] Another clinically important metabolic pathway is the mitochondrial electron transport chain. The electron transport chain uses a complex series of redox reactions to generate a proton gradient across the inner mitochondrial me...

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Abstract

Compositions and methods for treating and preventing Leber's hereditary optic neuropathy are provided.

Description

[0001] Cross References to Related Applications [0002] This application claims the benefit of and priority to U.S. Provisional Application No. 62 / 744,242, filed October 11, 2018, the contents of which are incorporated herein by reference. technical field [0003] The present application relates to compositions and methods for the treatment and prevention of Leber's hereditary optic neuropathy. Background technique [0004] Leber's hereditary optic neuropathy (LHON) is an inherited disorder in which degeneration of the retina leads to loss of vision. The onset of vision loss caused by LHON usually occurs in early adulthood and leads to vision of 20 / 200 or worse within a few months. Although the prevalence of LHON has not been extensively studied, the incidence of LHON in certain European populations is about 1 in 50,000, suggesting that hundreds of thousands of people may be affected worldwide. LHON is caused by any of several mitochondrial mutations that disrupt the fun...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K8/36A61P27/02
CPCA61P27/02A61K31/194A61K31/197A61K9/0048A61K47/542A61K47/54A61K31/225
Inventor A·D·莱文D·A·格罗斯
Owner IMBRIA PHARMA INC
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