LX2006 with positive results

Lexeo's LX2006 gene therapy is very promising. It uses an adeno-associated viral (AAV) vector to deliver a functional copy of the *FXN* gene (which encodes frataxin) directly into myocardial cells. The goal is to increase frataxin protein expression, restoring impaired mitochondrial function in patients with Friedreich's ataxia (AF). The refinement of this technique represents a significant advance toward a new therapeutic option for patients with AF-associated cardiomyopathy.

Here is a post by Amália Maranhão, Director of Abahe (Brazilian Association of Hereditary and Acquired Ataxias)

“Lexeo Therapeutics has positive preliminary data on the efficacy of the LX2006 gene therapy for Friedreich's Ataxia heart disease

Lexeo Therapeutics today announced positive interim data from the Phase 1/2 study of its gene addition therapy for AF cardiomyopathy.

In both the Lexeo-sponsored Phase 1/2 SUNRISE-FA clinical trial and the Weill Cornell Medicine investigator-initiated Phase 1A trial, treatment with LX2006 was associated with clinically meaningful improvements in cardiac biomarkers and functional measures, and increased frataxin protein expression was observed in all participants with cardiac biopsies.

Among the six participants with abnormal baseline left ventricular mass index (LVMI, a measure of the size and thickness of the heart's left ventricle), five had a greater than 10% improvement in LVMI at or before the 12-month visit.

Lexeo has achieved alignment with the FDA on key endpoints related to the planned LX2006 study, and this study is expected to occur in early 2026.

To read the full press release, visit: https://ow.ly/Wy9r50Vw3NL

Source: Amália Oliveira (Abahe)