Pharmacovigilance and Risk Management Strategies Conference

Advanced therapy medicinal products (ATMPs), which include gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer patients hope for various diseases for which there are limited or no therapeutic options. The development of ATMPs has sparked considerable interest and debate as there are many unknowns and uncertainties concerning the effects or responses these therapies may evoke, especially in the case of genetic manipulation. This session will examine the challenges associated with monitoring safety and the benefit-risk of these therapies, both short-term and long-term, and explore the complexities associated with determining the safety profile and benefit-risk across common delivery platforms such as gene therapy vectors and lipid nanoparticle delivery systems.