Gene therapy is RSRT’s most advanced of the four curative approaches and is their lead program. The concept behind gene therapy is simple: delivery healthy copies of the MECP2 gene to compensate for the mutated ones. Over the past three years the results of RSRT’s Gene Therapy Consortium have exceeded expectations. The magnitude of improvement in the mouse models of Rett Syndrome is much greater than that of any drug in development and suggests that significant benefit may be achieved in people.

Based on these results the biotech company, AveXis, has committed to advancing a gene therapy treatment into clinical trials. The first human clinical trial in gene therapy is on track to begin this year; a second-generation gene therapy is being developed to ensure maximum impact on lives; potentially curative approaches in MECP2 reactivation, RNA editing and transsplicing, and protein replacement are making promising strides.

A single gene therapy treatment should be a ONE-TIME FIX


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