long-lasting frataxin / Fratagene - LARVOL DELTA

3D Heart models suggest restoring frataxin could treat cardiac dysfunction in Friedreich’s ataxia (Friedreich's Ataxia News) - "Normalizing the cardiac levels of frataxin via gene therapy or an alternative strategy could be an effective treatment for cardiac symptoms in patients with Friedreich’s ataxia (FA), as suggested by three-dimensional (3D) heart tissues made in the lab, a study suggests...The data further showed that the higher the production of frataxin, the greater the contractility. Using viral vectors to restore protein and RNA levels of frataxin also normalized cardiac cell contractility in comparison with the controls."

Preclinical

Naturally occurring mutation in ISCU2 protein may bypass effects of frataxin loss, early study suggests (Friedreich's Ataxia News) - "A naturally occurring variant of ISCU2 protein can have the same molecular role as frataxin, an experimental study has found...This suggested that ISCU2 was speeding up the clusters’ production at a different step of the pathway compared to frataxin. Further analysis revealed that, contrary to frataxin, ISCU2 variant was accelerating not the production of Fe-S clusters but rather their transfer to proteins."

Preclinical