Cell and gene therapies are much more complex to develop, manufacture, and review than other biologics and small-molecule drugs, posing distinct and often unique challenges for getting potentially life-saving therapies to patients. The development of these therapies has resulted in many bespoke solutions that each require their own development program and regulatory review.
On November 1, 2023, the Alliance for Regenerative Medicine (ARM) and the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) co-sponsored a Scientific Exchange between CGT developers, FDA staff, and other key stakeholders, which focused on identifying reusable elements of CGT technologies that could be leveraged to improve the time and resource efficiency. This whitepaper provides the framework for the building blocks discussion during the session’s interactions, discusses the viability of specific building block proposals in 3 technology areas (LNP, iPSC, and AAV), and explores potential regulatory paths toward establishing reusable technologies.
