Rare Disease: Operationalizing Gene Therapy Trials

Gene therapy holds promise for treating a wide range of diseases, from cancer and diabetes to rare genetic disorders. It has also sparked great interest because it offers the possibility of a cure, particularly for singlegene diseases such as sickle cell anemia or hemophilia. This potentially revolutionary treatment modality is still in its infancy, the science is stunningly complex, and the regulatory terrain is constantly evolving. In these early days of gene therapy, many sponsors are seeking guidance on how to navigate the challenges of bringing these treatments from bench to bedside.

In this white paper, we will explore the history of gene therapy trials, as well as the types of gene therapy vectors and delivery strategies. We will also discuss the regulatory and operational challenges associated with gene therapy trials, including start-up regulations, site selection, recruitment, and retention. Of note, this white paper focuses on somatic cell gene transfer, rather than germ cell gene therapy.