Strategies To Accelerate Process Development From Preclinical To Manufacturing For Gene Therapy

By Dogan Ornek, XingchengChen, Hassan Sakhtah, Chih-Cheng Chen, Nida S. Zubairy, Tao Xiang, Jared Kaufman, Linda Ma, Acelya Babali, Chi-Ming Yu, W. Roy Lin

A typical gene therapy process development cycle, preclinical through late-phase manufacturing, runs three to five years. A team at Boston Institute of Biotechnology, led by Dogan Ornek, describes how it shortened that path with a platform built for early-stage work and a new PAT application that maps design space and supports a control strategy. Efficient DoE and closer coordination between development and manufacturing round out the approach. Two case studies put it to the test: an AAV media toolbox that raised titer 50-fold over a client's original process, and a pDNA platform covering fermentation through recovery and purification.

Study the data and process workflows to see how the pieces fit.