Periodic Reporting for period 2 - Facer (The First Cell Culture Platform that Enables Affordable Cell Therapies at a Large Scale)
Reporting period: 2020-01-01 to 2020-12-31
Cell therapy has shown promising results for treating and curing a large range of diseases, from cancer to diabetes. The principles of cell therapy are easy to describe: cells (possibly modified) are injected into patients. Cell therapy is now a reality in Europe: in August 2018 the first two treatments for patients with blood cancer were approved; more of them are about to successfully conclude the approval path. What hinders the large-scale uptake of cell therapy is its costs: culturing enough cells of high quality requires specialised personnel and infrastructure, which only a few centres can afford. The market asks for a scalable cell culture platform that can produce large quantities of high-quality cells at much lower costs than done nowadays. To address this need, we have developed the Facer – the first fully-autonomous cell culture platform. The Facer can manufacture billions of high-quality cells starting from the small quantity (about 500,000 cells) of a tissue biopsy without the need of human intervention, thanks to a unique patented iterative process.
Our product is at TRL 7; its performances, in terms of quality and costs, are superior to any competitor commercially available. We have developed the Facer over the course of 5 years thanks to cash investments of over €3 million. The objectives in the Phase 2 project are: 1) to finalise the product and perform a validation with external partners, 2) finalise the regulatory path, and 3) find a distributor and a launching customer. We will initially tackle the European market to then expand globally once that the local regulatory barriers are overcome. Our innovation can enable affordable cell therapies at a large scale and change the life of millions of patients who suffer from diseases that have been deemed 'incurable' so far.
Our product is at TRL 7; its performances, in terms of quality and costs, are superior to any competitor commercially available. We have developed the Facer over the course of 5 years thanks to cash investments of over €3 million. The objectives in the Phase 2 project are: 1) to finalise the product and perform a validation with external partners, 2) finalise the regulatory path, and 3) find a distributor and a launching customer. We will initially tackle the European market to then expand globally once that the local regulatory barriers are overcome. Our innovation can enable affordable cell therapies at a large scale and change the life of millions of patients who suffer from diseases that have been deemed 'incurable' so far.
The objective of Aglaris was to transform the Facer 1.0 to an optimized industrialized equipment (Facer 2.0) and to validate it. To do so, the specific aims were: (1) to manufacture some Aglaris Facer 2.0 units, (2) to verify and calibrate them, (3) to industrialize the single-use cartridge, and (4) to externally validate the technology through a beta-testing program. During the project, the final engineering of the Facer 2.0 has been completed. Aglaris has defined a manufacturing and verification plan for the units that has concluded with the verified units ready for beta-testing. In parallel, the single-use cartridge specific for T cells, where the actual cell expansion takes place, has been also industrialized providing a prototype nearly ready for commercialization. Not only the technical aspects have been taken into account, but also the industrial design has been carefully studied to create a solution that really meets the needs of the end users and provides competitive advantages to other bioreactors in the market.
Regarding the software, a new user interface has been created to improve the user experience with the platform. It has been designed with the feedback obtained from key opinion leaders to ensure its correct use and maximise its functionality. The FACER´s interface provides a step-by-step guide through the process of installation for the user to follow when setting up the single-use cartridge. The user must login in the platform at different stages to allow traceability and follow the on-screen steps in order to correctly place the cartridge in the oven module. The different routines for the culture have been adapted to make them flexible and allow the Facer to work with different cell cultures.
Aglaris has advanced in the regulatory path and it is maintaining and improving its QMS with the intention to be closer to ISO9001 certification. We have created additional documentation required for the certified QMS, such as general documents (scope, objectives) and SOPs.
Due to the recent Brexit, we had to evaluate the effect of the new UK regulation on our products. During the project, we have been preparing documentation for the CE marking (and now also UKCA marking). The requirements have been taken into account when manufacturing both products. The cartridge, which is in contact with the therapeutic product, needs to meet additional biocompatibility and safety requirements. As part of the cartridge industrialization, each component has been carefully selected and internally tested, when required. Furthermore, the sterilization strategy has been defined and tested. Finally, Aglaris has a very strong position on IP, as it has 5 patents already filed on the Facer system.
During the project, Aglaris has been re-evaluating the way the distribution of the Facer will be conducted. Additionally, Aglaris has been planning the ramp-up phase, considering the resources required in terms of personnel, investment, manufacturing, etc.
We have done an extensive dissemination of the Aglaris Facer project within major stakeholders: distributors and end users (point of care, CMO, biopharmaceutical companies). When relationships have been already established, Aglaris has made private demonstrations of the technology either on-site or remotely during the pandemic.
Regarding the software, a new user interface has been created to improve the user experience with the platform. It has been designed with the feedback obtained from key opinion leaders to ensure its correct use and maximise its functionality. The FACER´s interface provides a step-by-step guide through the process of installation for the user to follow when setting up the single-use cartridge. The user must login in the platform at different stages to allow traceability and follow the on-screen steps in order to correctly place the cartridge in the oven module. The different routines for the culture have been adapted to make them flexible and allow the Facer to work with different cell cultures.
Aglaris has advanced in the regulatory path and it is maintaining and improving its QMS with the intention to be closer to ISO9001 certification. We have created additional documentation required for the certified QMS, such as general documents (scope, objectives) and SOPs.
Due to the recent Brexit, we had to evaluate the effect of the new UK regulation on our products. During the project, we have been preparing documentation for the CE marking (and now also UKCA marking). The requirements have been taken into account when manufacturing both products. The cartridge, which is in contact with the therapeutic product, needs to meet additional biocompatibility and safety requirements. As part of the cartridge industrialization, each component has been carefully selected and internally tested, when required. Furthermore, the sterilization strategy has been defined and tested. Finally, Aglaris has a very strong position on IP, as it has 5 patents already filed on the Facer system.
During the project, Aglaris has been re-evaluating the way the distribution of the Facer will be conducted. Additionally, Aglaris has been planning the ramp-up phase, considering the resources required in terms of personnel, investment, manufacturing, etc.
We have done an extensive dissemination of the Aglaris Facer project within major stakeholders: distributors and end users (point of care, CMO, biopharmaceutical companies). When relationships have been already established, Aglaris has made private demonstrations of the technology either on-site or remotely during the pandemic.
Nowadays, state-of-the-art methods to culture cells for therapeutic purposes require specialised infrastructure and personnel. As a consequence, cell therapy is too expensive (treatments cost at least €20,000 per patient) and not widely available. There is a huge market demand for a scalable cell culture platform that can produce large quantities of high-quality cells at much lower costs. Even more so now that the first cell therapy protocols have been approved in Europe.
The FACER project aims to fully develop and bring to the market the Facer, the first fully-autonomous cell culture platform tailored to the needs of cell therapy. In the Facer, cell culture happens iteratively in a closed environment to avoid contamination. The process is monitored with single-use sterile sensors and autonomously adjusted in real time – thanks to advanced algorithms developed in house –in order to ensure the optimal conditions for a homogeneous high-quality cell growth. Thanks to the iterative process, the Facer can manufacture billions of high-quality cells starting directly from the small quantity of a tissue biopsy without the need of human intervention. The production process is automatically documented, and it is in compliance with the GMP standards (Good Manufacturing Practice) required for cell therapy.
The Facer has clear advantages over the current solutions in the market in terms of: 1) cost, 2) quality, 3) scalability, and 4) versatility (i.e. possibility to work with different cells). The cost-effective high-quality manufacturing of cells and their derivatives will have a positive impact in the society since it will contribute to make cell therapies available to more patients and to widen the offer of therapeutic solutions. Fully defined cellular products can be used to restore the structure and function of damaged tissues and organs and to develop cell-based cellular therapies for the treatment of cancer and hematological disorders, autoimmune and other inflammatory diseases and genetic disorders.
The FACER project aims to fully develop and bring to the market the Facer, the first fully-autonomous cell culture platform tailored to the needs of cell therapy. In the Facer, cell culture happens iteratively in a closed environment to avoid contamination. The process is monitored with single-use sterile sensors and autonomously adjusted in real time – thanks to advanced algorithms developed in house –in order to ensure the optimal conditions for a homogeneous high-quality cell growth. Thanks to the iterative process, the Facer can manufacture billions of high-quality cells starting directly from the small quantity of a tissue biopsy without the need of human intervention. The production process is automatically documented, and it is in compliance with the GMP standards (Good Manufacturing Practice) required for cell therapy.
The Facer has clear advantages over the current solutions in the market in terms of: 1) cost, 2) quality, 3) scalability, and 4) versatility (i.e. possibility to work with different cells). The cost-effective high-quality manufacturing of cells and their derivatives will have a positive impact in the society since it will contribute to make cell therapies available to more patients and to widen the offer of therapeutic solutions. Fully defined cellular products can be used to restore the structure and function of damaged tissues and organs and to develop cell-based cellular therapies for the treatment of cancer and hematological disorders, autoimmune and other inflammatory diseases and genetic disorders.