ASGCT 2021 | NextGen & GenX AAV vectors for DMD gene therapy

Arun Srivastava, PhD, University of Florida, Gainesville, FL, explains that due to the high volume of muscle is the body, clinical trials investigating first-generation adeno-associated virus (AAV)-based gene therapies for muscular dystrophy require the use of high vector doses, resulting in the need for immune-suppression. In an effort to address this, next-generation (NextGen) AAVrh74 vectors and single-stranded generation X (GenX) AAV vectors have been developed. The encapsulation of the GenX genome containing the micro-dystrophin gene into NextGen AAVrh74 vector capsid, referred to as an optimized (Opt) AAVrh74 vector, is also being investigated and offers the potential to reduce vector doses while maintaining therapeutic efficacy for muscular dystrophy. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.