Key considerations in formulation development for gene therapy.

Abstract

Gene therapy has emerged as an important area of research leading to the success of multiple product approvals in the clinic. The number of clinical trials for this class of therapeutics is expected to grow over the next decade. Gene therapies are complex and heterogeneous products that employ different types of vectors and are susceptible to degradation. The product development process for commercially viable gene-based pharmaceuticals remains challenging. This review discusses these challenges, covering the strategies used to ensure drug stability and to develop formulations involving viral or non-viral vectors, as well as accelerated regulatory approval pathways for gene therapy products.