Submitted by Tim MacLachlan and Joy Cavagnaro, Chairpersons of the upcoming 2016 Continuing Education (CE) course, "Unique Approaches to Safety Assessment of Gene, Cell, and Nucleic Acid-Based Therapies"
This 2016 SOT Annual Meeting CE Course will address the regulatory, scientific, and strategic inputs required to support the safe use of such advanced therapies in initial clinical trials and beyond and will feature speakers from both industry and the US Food and Drug Administration (FDA).
The platforms used for therapeutic treatment of disease have been greatly expanding over the last decade beyond the standard small molecule approaches and the now widespread use of proteins and monoclonal antibodies. The prospect of gene therapy began several decades ago with the promise that misfunctioning genes could be simply replaced, but was stunted in its growth with several notable safety events in the clinic. Now gene therapy is making a furious comeback, with several industry and academic groups employing various technologies and racing to catch up.
Cell therapy has experienced similar peaks and valleys in interest, with stem cells touted as a platform able to replace entire damaged organ systems. Multiple variants of what one would call a cell therapy now are in development ranging from treatment with fully differentiated somatic cells to naïve cells able to grow and differentiate in vivo. A combination of gene and cell therapy approaches is used in the widely popular T cell immunotherapy approaches for cancer treatment where cells are modified ex vivo to target tumors after reintroduction to the patient. Considering the potency of T cells, it is not surprising that safety concerns have limited their target profile.
Finally, the concept of knocking down expression of gene expression has gained significant momentum with the introduction of therapeutic RNA interference and most recently with gene editing via a variety of methods. All of these “advanced therapy” platforms require very unique approaches outside of the standards defined by internationally accepted guidance for preclinical safety assessment.
To view a listing of all 2016 CE courses, visit the SOT 2016 Annual Meeting website.
To register, also visit SOT 2016 Annual Meeting website.