Periodic Reporting for period 1 - PAT4CGT (Automated online monitoring & control to improve processes and decision making in cell and gene therapy manufacturing)
Reporting period: 2022-10-01 to 2023-09-30
Highly personalised advanced therapy medicinal products (ATMP) such as cell & gene therapies (CGT) represent a breakthrough in medicine, with the promise to become one-off cures for a wide range of conditions. However, making CGT products widely available to patients proved challenging so far. Existing CGT manufacturing processes involve multiple manual steps that need to be performed in high grade clean rooms by skilled professionals who have limited tools to monitor progress over time. This leads to two major problems: unpredictable outcome and high cost – a single dose can cost anywhere from €250k-€2m due, in part, to the labor-intensive nature of the process.
PAT4CGT consortium seeks to overcome these obstacles by developing a solution that will support fully automated CGT manufacturing processes. To achieve the objectives of the project, partners are developing a self-contained analytical instrument to enable non-invasive real-time in-line sensing of critical process parameters (CPPs). Coupled with process automation, this solution will monitor the overall status of the cell culture, obliterating the need for manual sampling steps. The consortium is pooling the expertise of partners to define these CPPs and better understand their role, so that optimal conditions can be found, and process parameters adjusted. The collected data is used to build digital twin models and prediction algorithms that will allow process control and increase product quality. The resulting tool will help CGT developers identify key relationships between CPPs, as potential predictors of cell quality and therapeutic outcome. Ultimately, the project will enable the biopharma industry to scale CGT production and help patients to access these innovative therapies.
PAT4CGT consortium seeks to overcome these obstacles by developing a solution that will support fully automated CGT manufacturing processes. To achieve the objectives of the project, partners are developing a self-contained analytical instrument to enable non-invasive real-time in-line sensing of critical process parameters (CPPs). Coupled with process automation, this solution will monitor the overall status of the cell culture, obliterating the need for manual sampling steps. The consortium is pooling the expertise of partners to define these CPPs and better understand their role, so that optimal conditions can be found, and process parameters adjusted. The collected data is used to build digital twin models and prediction algorithms that will allow process control and increase product quality. The resulting tool will help CGT developers identify key relationships between CPPs, as potential predictors of cell quality and therapeutic outcome. Ultimately, the project will enable the biopharma industry to scale CGT production and help patients to access these innovative therapies.
During the first 12 months of the PAT4CGT project, partners have successfully implemented the research programme and tasks planned for that period as stipulated in the Grant Agreement. This has allowed the consortium to progress towards the overall goal of increasing reproducibility and quality of CGT products manufacturing, in addition, the consortium moved closer to the goal of widespread adoption of the technology through flexibility of integration into various production systems. The project advanced from TRL1/TRL 2 to reach TRL3 at the end of September 2023.
The consortium has identified three aspects that need to be addressed in order to ensure a successful market entry:
1) Quality: Our end-users will require high quality devices and single use consumables to use in a good manufacturing practices (GMP) compliant environment. We perform tests (biocompatibility, verification and validation) to guarantee that our product meets GMP requirements and specifications;
2) Credibility and visibility: Users will adopt our solution if they see an important improvement in performance compared to existing solutions. To get traction, we need to show high quality validation, both internal and external, and to present robust data;
3) Risk averse industry: Cell therapy developers avoid cumulating asset clinical risk and CMC technology risk. They would rather use suboptimal but proven tools for manufacturing. We need to identify and engage with early adopter customers focused on niche applications as they will likely see enabling value in adopting our technology. This is being facilitated through our engagement with the industry at conferences, and by the portfolio activities within the Pathfinder sub-portfolio.
1) Quality: Our end-users will require high quality devices and single use consumables to use in a good manufacturing practices (GMP) compliant environment. We perform tests (biocompatibility, verification and validation) to guarantee that our product meets GMP requirements and specifications;
2) Credibility and visibility: Users will adopt our solution if they see an important improvement in performance compared to existing solutions. To get traction, we need to show high quality validation, both internal and external, and to present robust data;
3) Risk averse industry: Cell therapy developers avoid cumulating asset clinical risk and CMC technology risk. They would rather use suboptimal but proven tools for manufacturing. We need to identify and engage with early adopter customers focused on niche applications as they will likely see enabling value in adopting our technology. This is being facilitated through our engagement with the industry at conferences, and by the portfolio activities within the Pathfinder sub-portfolio.