AAVLife: Fixing Friedreich's heart
AAVLife developing gene therapy to treat Friedreich's ataxia cardiomyopathy
AAVLife S.A.S. is developing an adeno-associated viral vector-based gene therapy that could become the first disease-modifying treatment for Friedreich's ataxia-associated cardiomyopathy. The therapy has the potential to decrease the risk of cardiac events and improve patient survival.
Friedreich's ataxia is a rare hereditary progressive neurodegenerative condition caused by mutations in the gene encoding frataxin (FXN; FRDA) that result in decreased expression of the mitochondrial protein.
FXN deficiency leads to degeneration of the nervous system, resulting in progressive loss of motor function, scoliosis, and impaired vision, hearing and