Periodic Reporting for period 1 - CELLBOX (Innovative transport of “in culture” cell-based materials ensuring product safety, quality and function)
Periodo di rendicontazione: 2023-10-01 al 2024-09-30
Advanced Therapy Medicinal Products (ATMPs) have the potential to revolutionize medicine by offering personalized and targeted treatments, such as gene therapy, cell therapy, and tissue-engineered products. They promise to cure or significantly improve outcomes for previously untreatable conditions, reduce long-term healthcare costs, and enhance patients' quality of life by addressing the root causes of diseases at the cellular and genetic levels. Many of these advanced medicinal products cannot be cryopreserved like many other biological drugs but instead require tightly controlled environmental conditions to be kept functional. These constraints complicate and often prohibit transport of materials during manufacturing and also final product to the patients. In this project, we will develop a solution for global transport of ATMPs to fully unleash the potential of live cell-based products for the cell therapy and regenerative medicine markets, where access to increasingly complex and sensitive clinical-grade biological materials will be critical for treating patients throughout the EU and globally.
The Cellbox Go/AT will establish a new value chain and is a market-creating innovation. Advanced therapy medicinal product (ATMP) manufacturers face the challenge of producing highly specialized cells and tissues, but currently cannot ship them under adequate conditions because the appropriate transport vessels do not exist. For the first time, Cellbox Go/AT will enable the transport of biomaterials which cannot be frozen or should not be frozen due to their fragile nature. At present, no marketable solution exists for this need. Accordingly, there is an opportunity to disrupt the existing value chain and market for the established transports of ATMPs.
The Cellbox Go/AT will establish a new value chain and is a market-creating innovation. Advanced therapy medicinal product (ATMP) manufacturers face the challenge of producing highly specialized cells and tissues, but currently cannot ship them under adequate conditions because the appropriate transport vessels do not exist. For the first time, Cellbox Go/AT will enable the transport of biomaterials which cannot be frozen or should not be frozen due to their fragile nature. At present, no marketable solution exists for this need. Accordingly, there is an opportunity to disrupt the existing value chain and market for the established transports of ATMPs.
We have previously developed a self-sustaining incubator maintaining controlled environments (temperature and carbon dioxide concentration) for research-grade cell-based materials. Based on this platform, we started activities to expand the scope of applicability to clinical materials. This requires development of a regulatorily compliant software and data management, expanded atmospheric control for different oxygen concentrations, an expanded controlled temperature range, and enhanced packaging solutions compatible with international air transport regulations. Thus far, we have shown basic technical feasibility for all these new features in our prototypes regarding temperature variation, CO2 control, preconditioning gas mix, secondary packaging and user interface.
The main limitations of existing transport solutions for ATMPs are:
1. Cryopreservation for storage and transport has critical consequences: Most living organs or 3D tissue structures and many therapeutic cells are irreversibly deteriorated by freezing Cryoprotectants, used to prevent intracellular ice crystals formation, but which are cytotoxic at positive temperatures. Even for cell types that can be frozen and thawed, cryoinjury leads to loss of viability and function of the thawed cells (up to 60–70% loss).
2. Live cell shipment at lower temperatures for “cryo-sensitive” cells and tissues: At 18-25°C, in insulated boxes, but without active temperature control in this range, typical temperature excursions have serious detrimental effects on the tissues. At 4-8°C, in solutions that limit the stress imposed on cells at low temperature. Both solutions lead to damages due to lack of gas pressure control, altered pH of the medium and perturbation of the cell metabolic activity. These limitations pose significant hurdles for adoption for these transport technologies for clinical grade materials and products.
1. Cryopreservation for storage and transport has critical consequences: Most living organs or 3D tissue structures and many therapeutic cells are irreversibly deteriorated by freezing Cryoprotectants, used to prevent intracellular ice crystals formation, but which are cytotoxic at positive temperatures. Even for cell types that can be frozen and thawed, cryoinjury leads to loss of viability and function of the thawed cells (up to 60–70% loss).
2. Live cell shipment at lower temperatures for “cryo-sensitive” cells and tissues: At 18-25°C, in insulated boxes, but without active temperature control in this range, typical temperature excursions have serious detrimental effects on the tissues. At 4-8°C, in solutions that limit the stress imposed on cells at low temperature. Both solutions lead to damages due to lack of gas pressure control, altered pH of the medium and perturbation of the cell metabolic activity. These limitations pose significant hurdles for adoption for these transport technologies for clinical grade materials and products.