Cell and gene therapy development faces numerous challenges, but industry experts are highlighting key strategies to streamline the process and reduce associated risks. According to Hugh Murray, Senior Scientist at MilliporeSigma’s Upstream Process Development team, success in this field requires more than just “a gene and a dream.”
At MilliporeSigma’s 202,000 square-foot Carlsbad facility, the CDMO team has implemented three crucial approaches to optimize viral vector-based therapy manufacturing. These strategies combine scalable equipment, predictive modeling, and automated systems to enhance efficiency while minimizing uncertainties.
The first essential element is maintaining consistency throughout the development and manufacturing process. Murray emphasizes the importance of backward planning from the desired outcome, ensuring each step remains repeatable and scalable. The facility’s unique setup, where pilot scale operations directly connect to process development, enables seamless transitions between development stages. This integration of development and manufacturing teams under one roof facilitates knowledge sharing and smooth progression from early-stage work through clinical and commercial production.
Equipment consistency plays a vital role in this approach. The facility maintains identical instruments, from basic pH meters to sophisticated bioreactors, in both development and manufacturing areas. This mirroring strategy helps prevent unexpected complications during scale-up and accelerates technology transfer processes.
The second key strategy involves leveraging predictive modeling to anticipate and address potential challenges before they materialize. Using design of experiments (DOE) methodology, the team develops comprehensive bench-scale models that guide critical decision-making before investing in large-scale production. These models help identify potential problem areas or “danger zones” in the process, allowing for preemptive solutions and tight control parameters.
High-throughput viral characterization capabilities represent the third crucial element. The facility employs advanced automated robotics and statistical approaches to accelerate testing and characterization processes. This automation enables a single scientist to perform work that traditionally required five or more personnel, significantly reducing development timelines while maintaining high standards of replication and accuracy.
Early and frequent testing ensures precise understanding of the product throughout development. As Dayne Callum, Senior Scientist in Downstream Process Development at MilliporeSigma, notes, this comprehensive approach provides clients with well-characterized quality attributes and minimizes surprises during scale-up and manufacturing transfer.
The facility’s automated workflows and robust testing capabilities allow for rapid screening of multiple process iterations
simultaneously. This efficiency enables deeper understanding of process variance while maintaining high throughput, ultimately supporting faster progression to clinical trials.
The integration of these three strategies – consistent processes and equipment, predictive modeling, and advanced characterization capabilities – creates a comprehensive approach to de-risking cell and gene therapy manufacturing. This systematic methodology helps companies navigate the complex path from concept to clinical implementation with greater confidence and efficiency.
Murray’s team emphasizes the importance of understanding target product quality profiles from the outset and working backward to establish appropriate development pathways. This approach, combined with automated systems and statistical analysis, provides a robust framework for successful therapy development.
For cell and gene therapy innovators, partnering with experienced CDMO providers like MilliporeSigma can significantly reduce uncertainty in the development process. The company’s integrated approach allows therapy developers to focus on advancing their treatments while relying on established manufacturing expertise and infrastructure.