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183 Avenue de Choisy, Paris, 75013, FRA
- AAVLife is a gene therapy company focused on developing treatments for rare diseases with great unmet medical need. Our lead indication is for the cardiomyopathy associated with Friedreich’s Ataxia. Our 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.
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