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Helene Puccio: Founder- Ronald Crystal: Founder
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 conferring with regulatory authorities about toxicity-study requirements and clinical-trial design. The goal is to commence a clinical trial in 2015 to evaluate gene therapy for heart disease associated with Friedreich’s Ataxia. An observational study will be initiated in 2014 as a run-up to treating patients in a Phase I/II study.
Friedreich’s Ataxia is a life-threatening disorder caused by a genetic mutation that results in progressive neurologic and cardiac symptoms typically first appearing in childhood or during the teenage years. Cardiac disease accounts for the majority of deaths due to Friedreich’s Ataxia, with death usually occuring in early adulthood.
They are passionate in their quest to serve patients and value their relationship with patient communities and the organizations that serve them.
