Gene-edited cell therapies represent a rapidly growing area for advanced therapeutic development, but manufacturing-related challenges remain to commercialize such complex therapies.
One such challenge is the gene delivery process. Current delivery technologies rely on mechanisms that may adversely impact the quality of the modified cells, ultimately leading to reduced clinical efficacy. The landscape for next-generation gene-modified cell therapies could be dramatically changed if a gene-delivery technology was available that had negligible impact on cell quality.
In this White Paper, Avectas demonstrates how a proprietary platform, Solupore®, offers an automated, reproducible transfection system for editing primary immune cells suitable for a clinical manufacturing process.
The Solupore process for delivery, when compared with standard electroporation, results in healthier, less apoptotic cells, which retain a larger population of stem-cell memory phenotype. The production of healthier edited cells will potentially improve in-vivo cell function in various clinical applications where the modified cells may be more effective as ‘programmable’ assailants against a target antigen. The White Paper results suggest that cells transfected by Solupore are strong candidates for multiple clinical applications.
In summary, the essential features gleaned from the investigation included:
- Superior cell health & phenotype – improved capacity for replication, young memory phenotype and reduced cell death compared to electroporation, leading to enhanced cell function in relevant models.
- Versatility – non-viral delivery of RNA, DNA, or protein cargo to a range of cell types, including T cells, NK cells, and iPSCs
- Flexible, complex editing
- Ease of adoption – automated, closed system available now with full GMP support by the 2nd half of 2023
The investigation determined that the Solupore platform can deliver complex cargos while maintaining cell health, phenotype and function. In doing so, the platform demonstrates an effective solution to manufacturing challenges associated with gene-edited cell therapies.
This White Paper has been published by Phacilitate on behalf of Avectas.
