Streamlining Gene Therapy with AAVs

Gene therapies are enabling a rapid expansion in the scope of treatable disease.  Adeno-associated viruses (AAVs) are particularly advantageous delivery vehicles for introducing functional genes to specific tissues or cells due to their simplicity, safety, and efficacy. A successful AAV discovery and development pipeline covers basic vector design, in vitro assays, in vivo biodistribution, immunogenicity, efficacy, and safety studies.

In this white paper, critical considerations for a successful AAV discovery and development pipeline are discussed. New technologies and design factors that increase both speed and safety are highlighted, including advanced self-silencing adenovirus technology which enables the safe, efficient creation of AAVs without contaminating adenoviruses.

White Paper_Streamlining Gene Therapy with AAVs

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