AAVLife

Company profile

AAVLife company profile

AAVLife's lead indication is for Friedreich’s Ataxia Cardiomyopathy. Their program is based on the successful use of a gene-therapy approach in a mouse model developed to mimic the gene defect and cardiac symptoms of Friedreich’s Ataxia. The approach made use of an adeno-associated virus (AAV) to introduce a normal gene into cardiac tissue. The therapy restored cardiac function and reversed pathological heart enlargement in mice that had already progressed to heart failure. AAVLife has progressed research of this gene-therapy approach to inform decisions about dosing and route of administration to be used in a clinical trial. The company will be..... For more information, log in to dureach.com to view the full company profile.