Translating Cell and Gene Biopharmaceutical Products for Health and Market Impact. Product Scaling From Clinical to Marketplace: Lessons Learned and Future Outlook.

Cell and gene therapies have the potential to be curative for severe disease states such as cancer or incurable orphan genetic diseases. Despite the promise, there are only few such therapies available, although more are appearing in pharmaceutical pipelines. A major culprit limiting a fast translation from preclinical research to the clinic and the market is chemistry, manufacturing and control. The root cause is that most cell and gene therapies currently are personalized in form of ex vivo manipulated cells. This approach stands in sharp contrast to the population-based approach seen for small molecules and protein therapeutics. Therefore, it warrants a different approach to product manufacturing, testing, release, regulatory submissions, and product distribution. In this commentary, we highlight opportunities to solve these issues already in progress in industry and at academic institutions, but in addition call for expert contributions to a future cluster of articles in Journal of Pharmaceutical Sciences to illuminate additional solutions. Finally, we are also providing a perspective on future directions including expanding from current approaches of gene modification via viral vectors to for example gene editing, approaches that may lend themselves better toward allogenic and in vivo therapies and more typical chemistry, manufacturing and control approaches.