Abstract

Gene therapy holds tremendous potential for treating genetic disorders and acquired diseases by delivering therapeutic genes into target cells. The success of gene therapy largely depends on the development of effective delivery systems, known as vectors, which can transport genetic material safely and efficiently. This article explores the biopharmaceutical considerations critical to the design and optimization of gene therapy delivery systems. It discusses the types of vectors—viral and non-viral—used in gene therapy, highlighting their mechanisms of action and biopharmaceutical challenges such as targeting specificity, stability, immunogenicity, pharmacokinetics, and biodistribution. Future directions in vector design and technological advancements are also addressed, underscoring the ongoing efforts to overcome existing barriers and enhance the clinical applicability of gene therapy.