AAV Based Gene Therapy for Rare Genetic Disorders: Strategies and Challenges

There are over 7000 rare diseases worldwide, and unfortunately, less than five percent have an FDA approved therapy. The lack of valid animal models remains one of the biggest hurdles for preclinical drug discovery for rare diseases. However, recent technology advances can enable the development of innovative rodent models to speed up the drug discovery process.

From a therapeutic perspective, gene therapy is the most promising method to rescue the rare genetic disease caused by loss of function mutations. Recombinant AAV strategies have become more and more popular due to their relative safety, highly efficient delivery, and broad range of serotypes choices. Yet, AAV methods have many drawbacks due to their size limitation, immunogenicity, and high cost. In this GEN webinar, we will review the challenges of utilizing AAV, as well as discuss in detail the recent progress and outstanding questions in AAV-based gene therapy.

A live Q&A session followed the presentations, offering you a chance to pose questions to our expert panelists.