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

(530) 304-6810

​gino@ixchelpharma.com

Friedreich's ataxia

Ixchel holds exclusive intellectual property rights to Compositions of Matter on multiple Novel MMF prodrugs, and Methods of Use. 

© 2017 Ixchel Pharma

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.

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