Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics, discusses the Rare Pediatric Disease designation and Orphan Drug designation granted to LX2006 for the treatment of cardiomyopathy associated with Friedreich’s ataxia.

Friedreich’s ataxia is a degenerative neuromuscular disorder caused by mutations in the FXN gene which reduces the production of frataxin, a protein important for proper mitochondrial functions. The onset of symptoms varies greatly among patients but generally occurs between the ages of 5 to 18 years. Some of the more common symptoms include ataxia, fatigue, scoliosis, diabetes mellitus, and various heart conditions, including hypertrophic cardiomyopathy and arrhythmias. Currently, there is no approved treatment for Friedreich’s ataxia. 

LX2006 is an IV-administered, adeno-associated virus (AAV)-mediated gene therapy encoding the human frataxin gene. The designations granted to LX2006 cover cardiac disease and broader symptoms associated with FA.

As Mr. Townsend explains, Lexeo has been partnered with the Friedreich’s Ataxia Research Alliance (FARA) and will continue this partnership throughout the clinical trial process. Lexeo is planning on submitting an Investigational New Drug Application (IND) this year with the hope of an approval by the end of the year. Assuming the IND is approved, a phase 1/2 clinical trial of LX2006 is likely to begin recruiting by early 2022.

To learn more about Friedreich’s ataxia and other rare neurological disorders, visit