Conserving Drug Product To Treat More Patients: De-Risking Cell And Gene Therapies Early In Process Development For Late-Stage Success And Savings

By Scott Sznyter and Tatiana Nanda, Center for Breakthrough Medicines (CBM)

Important early studies a company may choose to postpone during process development are stability studies, both for process intermediates and the final product itself. Companies, worried about expending too much material early on, will push back against this testing. However, adopting a risk-based approach that incorporates testing at the stages where there is the highest risk to the process is key to getting the future formulation right.

An early, formal risk assessment that includes likely failure modes can help companies form a holistic view of the precautions to take to avoid outright failure. Partnering with a contract development and manufacturing organization (CDMO) with the experience and expertise to navigate this evaluation will help companies better understand what early studies are integral to the success of their project, as well as what efficiencies can help them achieve the agility to surmount cost constraints. While the rigors of early process development in cell and gene therapy can appear burdensome for nascent companies, prioritizing certain early studies can mean the difference between success and failure for these highly sensitive drug products. Early investment in drug product characterization to understand its capacity for scale-up and patient administration is integral to maximizing its potential reach.