ARTICLE | Emerging Company Profile
AAVLife: Fixing Friedreich's heart
AAVLife developing gene therapy to treat Friedreich's ataxia cardiomyopathy
August 11, 2014 7:00 AM UTC
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...