Flexibility In Gene Therapy: Adapting To The Unique Gene Therapy Demands

Since the landmark approval of the first gene therapy (GT) in 1990 for severe combined immunodeficiency (SCID), the field has undergone a remarkable transformation. This progress can be attributed to a deeper understanding of virology and disease pathogenesis, enabling the development of safer and more effective viral vector-based GTs.

Viral vectors function as efficient delivery vehicles, introducing corrective genetic material to modify or replace defective genes within target cells. This approach has shown promise in treating a broad spectrum of illnesses, including rare diseases, various cancers (particularly those affecting the blood), and numerous other conditions.

Over the past two decades, advancements in viral vector design and manufacturing have yielded significant improvements in key parameters – efficacy, safety, and scalability. These breakthroughs stem from groundbreaking research in virology, encompassing areas like vector construction, novel serotype identification, cellular targeting and entry mechanisms, viral gene regulation, host-vector interactions, and the immune response to vectors. Continued research efforts hold the potential for even more potent GTs.

Download the E-Book to learn more about the burgeoning field of GT and keep abreast of innovations in this dynamic and rapidly evolving therapeutic space.