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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...