Leigh Syndrome is a usually lethal inherited orphan neurodegenerative and myodegenerative disease of infancy, caused by reductions in single mitochondrial proteins. IXC-109 dose-dependently increases gene expression of multiple mitochondrial proteins, and alleviates lethality and increases mitochondrial functions in the animal model of Leigh Syndrome.

Gino Cortopassi

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Friedreich's ataxia

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Leigh Syndrome

Friedreich's is the most common inherited recessive ataxia in the USA and Europe. Friedreich's is caused by the reduction of a single mitochondrial protein, frataxin, which leads to a lethal cardiomyopathy. Ixchel's IXC-109 dose-dependently increases frataxin protein, and uniquely rescues cardiac pathophysiology and lethality in animal models of Friedreich's. There is no approved therapy for Friedreich's in US or Europe, hence there is a large unmet need.