BioCentury
ARTICLE | Targets & Mechanisms

Reversing (heart) failure in Friedreich's ataxia

April 24, 2014 7:00 AM UTC

Heart failure accounts for over half the deaths in patients with Friedreich's ataxia, but no therapies exist to treat this neurodegenerative disease or its associated cardiomyopathy. Now, a team of French researchers has shown that i.v. frataxin gene therapy could prevent or even reverse heart failure in a mouse model of Friedreich's ataxia.1

AAVLife S.A.S., a new company founded by several team members, has licensed the findings and will test dosing and safety of direct cardiac injection of the gene therapy in healthy pigs...