With an emerging gene therapy asset in the pipeline for a rare disorder, the client wanted to better understand why some gene therapies are more successful than others in the rare disease space


To investigate, we used secondary market research spanning viral vector research articles, clinical case studies, plus an examination of current supply chain and manufacturing challenges


We produced a robust report delineating the reasons for current successes and challenges of ex vivo and in vivo gene therapies, giving a range of future considerations and key cross-functional strategic recommendations