CP-FXN, Friedreich Ataxia

Friedreich ataxia (FRDA) is an autosomal recessive neurodegenerative disorder characterized by progressive gait and limb ataxia with associated limb muscle weakness, absent lower limb reflexes, extensor plantar responses, dysarthria, and decreased vibratory sense and proprioception. Onset is usually in the first or second decade before the end of puberty. It is one of the most common forms of autosomal recessive ataxia, occurring in about 1 in 50,000 individuals. FRDA is caused by a deficiency of the frataxin (FXN) protein, which changes cardiac muscle that leads to hypertrophic cardiomyopathy, or heart muscle disease. Around 75 percent of people with FRDA develop heart abnormalities leading to early death.

There is no fundamental treatment for FRDA other than drugs that temporarily relieve symptoms. Therefore, the only key treatment is the direct transfer of this mitochondrial protein into brain neurons and cardiac muscle cells.

In the CV-14 project, CP-FXN, a cell-permeable FXN, has been developed for FRDA treatment by applying TSDT platform, our original technology. Joint development of a new drug for FRDA is successfully in progress with a global pharmaceutical company. Until now, studies have shown that it is possible to transfer a large protein to the heart and brain, which has been considered to be impossible so far, as well as proven the therapeutic efficacy that it can save lives from an incurable illness in disease animal models. At 13 weeks of age (around 25 years in terms of human age), the treatment group showed 80% higher survival rate than the placebo group, which means that patients' lives can be saved and extended for years to decades (Figure 1).

ICP-FXN is a innovative new drug that can prolong life and save lives from death. It can give FRDA patients new expectation and an opportunity to change their lives from despair to hope.

Figure 1. CP-FXN increases the survival rate of FRDA disease animal model