AAVLife is a gene therapy company focused on developing treatments for rare diseases with great unmet medical need.

AAVLife is a gene therapy company focused on developing treatments for rare diseases with great unmet medical need.

AAVLife's lead indication is for Friedreich’s Ataxia Cardiomyopathy

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 -- An Effective Gene Therapy In An Animal Model

Friedreich's Ataxia -- An Effective Gene Therapy In An Animal Model. Science 2.0, By News Staff | April 6th 2014

The transfer, via a viral vector, of a normal copy of the gene deficient in patients, allowed to fully and very rapidly cure the heart disease in mice.

As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

AAVLife aimed at advancing gene therapy for cardiomyopathy in Friedreich’s ataxia to clinic

AAVLife aimed at advancing gene therapy for cardiomyopathy in Friedreich’s ataxia to clinic. International collaboration advances gene therapy for Friedreich’s ataxia

DOWNINGTOWN, Pa., April 6, 2014 /PRNewswire-USNewswire/ — The Friedreich’s Ataxia Research Alliance (FARA) is pleased to recognize the ground-breaking gene-therapy research of FARA-funded scientist Dr. Hélène Puccio.

FARA Press Release:
International collaboration advances gene therapy for Friedreich’s ataxia; AAVLife aimed at advancing gene therapy for cardiomyopathy in Friedreich's ataxia to clinic

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia. Morgane Perdomini, Brahim Belbellaa, Laurent Monassier, Laurence Reutenauer, Nadia Messaddeq, Nathalie Cartier, Ronald G Crystal, Patrick Aubourg & Hélène Puccio; Nature Medicine (2014) doi:10.1038/nm.3510

KEYWORDS: Cardiac failure, conditional mouse model, adeno-associated virus rh10 vector, establish the preclinical proof of concept, FRDA cardiomyopathy.