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Drug Product Manufacture Challenges and Opportunities for Gene Therapy Products

Jun 29, 2022 12:08pm ‐ Jun 29, 2022 12:30pm

Credits: None available.


Drug Product manufacture of gene therapy products is typically challenged by small batch sizes with limited ambient stability which requires expedient processing and measures to minimize product losses. Several case studies will highlight problems that occurred during manufacturing processes and how issues could be resolved by use of different components, procedural controls, or currently available technology. Other cases will be discussed where future technology may help to solve issues.

While fill/finish technology has been developed to the needs of gene therapy by conserving product solution, the whole design of the single use flow path needs to be evaluated to minimize loss of product solution in the manufacturing process. Due to cold storage requirements, preferred materials for vials are ready-to-use non-glass components. Recent developments provide glass formulations suitable for cold storage applications. Maybe the availability of those new formulations will allow use of bulk glass vials for small batch sizes?

Due to small batch sizes, not a lot of time remains during a fill to detect and correct equipment setup issues. Due to flexibility to support needs of clients, the equipment needs to frequently be changed over between different vial formats. How can a meaningful setup process be implemented that minimizes issues during the run? Some gene therapy products contain intrinsic proteinaceous particles. How can the visual inspection process be meaningful, and could future technologies help us distinguish between intrinsic and extrinsic particles? Sterile filtration of drug substance solution is a completely manual process. It requires creation of multiple flow paths and use of different instruments and connections.

Despite theoretical and practical operator training, operators oftentimes lack confidence in performing this step. Could VR technology be helpful by providing operators with reference information while the tasks are being executed?


  • Dena A. Flamm, Business Development Manager, OPTIMA Pharma
  • Lars Waldmann, PhD, Staff Engineer, Viral Vector Services, Thermo Fisher Scientific


Credits: None available.

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