Toward a new framework for the development of individualized therapies

Abstract

The concept of personalized medicine—selecting the appropriate previously developed treatment off the shelf to address an individual’s condition—now has been around for decades. As the era of molecular medicine matures, however, it is becoming increasingly apparent that individualized therapeutics developed for one or a few individuals will become central to addressing the unique molecular defects present in hundreds to thousands of genetic or acquired diseases through the application of any one of several different technologies. The recent appreciation of the potential for bespoke therapeutics to address serious unmet medical needs has highlighted the need for a shift to an end-to-end approach enabling the provision of individualized therapeutic products, most notably in the areas of directly administered vectored gene therapy, genetically modified cellular therapies, and antisense oligonucleotides (ASOs) [ 1 , 2 ]. Foundational to enabling an individualized approach to product development will be the development of a regulatory framework that supports such development in a way that is fundamentally different in certain respects from the approach that has been taken in the past for products aimed for the treatment of tens or hundreds of thousands of individuals.

Topics & Concepts