Cell and Gene Therapy from Preclinical Program to IND

Abstract

The evolution of drug development from small molecules and proteins to the next generation of cell and gene therapy (CGT) provide tremendous promise for patients. CGT products involve complex and diverse chemistry and biological mechanisms aided by rapidly evolving scientific research. The intrinsic material composition and putative MOA(s) of CGT products differ from conventional drugs. Therefore, the traditional, standardized approaches for a preclinical program may not be appropriate for evaluating the safety of CGT products. This position paper is intended to briefly introduce the current thinking of FDA on the preclinical program development of CGT products, and to provide Aleon’s position on the path from preclinical program to IND application for CGT products...