The Future of Non-Viral Gene Therapy: Accelerating Next-Generation Gene Therapies

Next-generation approaches to gene therapy are emerging as one of the most promising areas of drug development and disease treatment. For patients with rare genetic and hematologic diseases, the goal is to develop gene therapy and gene editing strategies with the potential to deliver functional cures. However, most gene therapy clinical trials use viral technologies like adeno-associated virus (AAV) for gene delivery, which may be transient in nature due to their episomal forms and have been associated with toxicities and safety concerns. To overcome these challenges the rapidly evolving science around gene therapy provides insight into new strategies to address a range of diseases using non-viral approaches. Such technologies entail integration directly into the genome, for better durability, or site-specificity, for enhanced safety profiles and consistent expression. This panel will explore the possibilities of non-viral gene therapy to solve some of the most pressing patient needs.