Strategies to Accelerate Process Development from Preclinical to Manufacturing for Gene Therapy


Gene therapy is a fast-growing industry. Demand on its products such as pDNA, AAV, retrovirus, lentivirus, etc. has significantly increased during last 10 years while its production challenges remain unresolved. It is a complex process with a number of unoptimized and low yielding steps. A typical process development (PD) cycle from preclinical to late phase manufacturing (Mfg) takes 3 to 5 years. It is desired to rapidly advance to manufacturing for early clinical candidates using well established and high yielding processes. The following strategies were successfully employed by BIB to accelerate the process development activities: (1) A platform approach for early stage process development, (2) Efficient DoE application for process optimization and characterization, (3) Interaction improvement between PD and Mfg to streamline scale-up, tech transfer and manufacture support, (4) Development of a new PAT tools application to understand process design space and hence develop a control strategy. Case studies for BIB AAV and pDNA production platforms will be covered in this presentation.

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