The Evolving Field of AAV-Based Gene Therapies

Site Selection for Advance Therapy Clinical Trials

Gene therapy is evolving pas­­t R&D phases with the emergence of new AAV-based gene therapies and a substantial increase in companies participating in gene therapy clinical work. TrialSite News summarizes recent progressions in the field that were reported in Genetic Engineering and Biotechnology News.

Each of the many emerging adeno-associated viral (AAV) vectors carry their own benefits and drawbacks. The main challenge concerns the delivery of the AAV, and researchers around the globe are working to improve this. In addition, natural AAVs are not targeted, possess little efficiency with human cell types, and can have interactions with the immune system. Those are three of the properties that need to be addressed when making the next generation of AAVs.

One of the companies at the forefront of gene therapy clinical work, Spark Therapeutics, takes a holistic approach to vector development and is working to improve the problematic interaction of AAVs and the immune system.

But there is another roadblock to gene therapy advancements: there is a large proportion of the population that carries antibodies against AAV—which is disqualifying for entrance into clinical trials...

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