Innovative regulatory pathways for cell and gene products have accelerated product development and approval allowing patients in need to access transformative therapies. However, the shortened development timeline has exerted significant pressure to accelerated CMC development.
One of the biggest challenges is to develop, characterize, and validate the manufacturing process under these compressed clinical development timelines while ensuring comparability between process versions and maintaining the link to clinical data. In the past few years, several cell and gene therapy products have overcome this challenge and successfully launched new CGTPs. This session will provide the challenges industry is facing and approaches to speed up the CMC development. Perspectives from regulators will help set the expectations of CMC acceleration and pathways to facilitate innovation.
Specifically, the session will demonstrate how risk assessments are leveraged to guide process development activities for these products to ensure that the CGTP can be manufactured commercially and meet the CQAs established during registration trials.