Development of Gene Therapies from an Academic Perspective

Gene therapy has significantly transformed how we approach treating inherited diseases. By manipulating the specific genes responsible for a disease condition, this technology has evolved in medicine as a potential avenue to treat various diseases. The success of gene therapies in treating rare monogenic disorders is encouraging their development for common conditions with single gene etiologies. Interestingly, gene therapy has also altered how academia moves therapeutic research across the translational spectrum. The role of academia in the development of orphan drugs has been reviewed extensively (Coles and Cloyd, 2012; van den Berg et al., 2021; Rosenberg et al., 2023). The drug development industry has historically moved away from academia due to high costs, time-consuming regulatory oversight, and the bureaucracy in general logistics of conducting studies with academia. However, when it comes to orphan drugs, academia is increasingly involved in discovery and early development. In contrast, gene therapy has led to considerable involvement of academic researchers beyond early-stage research, which includes disease biology, target identification, and the discovery of new molecular entities. From an academic perspective, the development of gene therapies involves a long and complex process that requires interdisciplinary collaboration and a deep understanding of genetics, molecular biology, biochemistry, and clinical medicine, and a commitment to safety, efficacy, and ethical considerations especially since there is the potential for genetic modification.