Analysis and quality control of novel mixed cell population for therapeutic development
Lead Participant:
VIDEREGEN LIMITED
Abstract
This project will focus on developing a critical quality control (QC) method that will allow manufacturers to test and ensure the quality of an immune therapy (called BioThymus).
The BioThymus therapy is designed to treat a life-threatening disease in infants. Children born without a thymus gland (called complete DiGeorge Syndrome or cDGS) have a poorly functioning immune system, and without treatment may die within 3 years.
The research that lead to the BioThymus was invented in the UK at University College London and the Francis Crick Institute, and is now being developed by Videregen to treat children with cDGS and in the future it could be used to treat autoimmune diseases.
The BioThymus combines mixed cell populations (from stem cells) within a scaffold made from thymus tissue. This provides a copy of a thymus gland structure, which is capable of regenerating the cells of the immune system.
Videregen (project lead), will focus on developing the industrial manufacture process compliant with UK and International regulations and standards. The BioThymus's unique cellular aspects are key to its safety and efficacy, but because of the uniqueness of the therapy there are no off-the-shelf methods or kits to measure and quantify the cells used. A standardised, robust and accurate QC test method is required to ensure final product quality, safety and efficacy control.
Videregen will collaborate in the project with the cell metrology lab of the National Measurement Laboratory (NML). NML have the necessary expertise and facilities to help address the cell measurement problem, by understanding how the multiple cell properties/attributes in the BioThymus can be harnessed and streamlined into a reproducible QC method that Videregen can incorporate into their manufacture process.
BioThymus manufacture for preclinical studies and transition to clinical trials cannot be achieved without reliable QC methods that quantify and identify the cells in the product, which links to product effectiveness. QC methods to define BioThymus's cellular aspects is a critical activity/milestone for therapy development.
* Method will be used as a QC measure and final release test.
* Method will allow further research to understand relationships between thymic cell phenotype and their function to educate T-cells in-vivo, allowing manufacturing optimisation and therapy effectiveness.
* Method will provide regulatory bodies, clinicians and investors a high degree of confidence to adopt or invest in the technology.
The BioThymus therapy is designed to treat a life-threatening disease in infants. Children born without a thymus gland (called complete DiGeorge Syndrome or cDGS) have a poorly functioning immune system, and without treatment may die within 3 years.
The research that lead to the BioThymus was invented in the UK at University College London and the Francis Crick Institute, and is now being developed by Videregen to treat children with cDGS and in the future it could be used to treat autoimmune diseases.
The BioThymus combines mixed cell populations (from stem cells) within a scaffold made from thymus tissue. This provides a copy of a thymus gland structure, which is capable of regenerating the cells of the immune system.
Videregen (project lead), will focus on developing the industrial manufacture process compliant with UK and International regulations and standards. The BioThymus's unique cellular aspects are key to its safety and efficacy, but because of the uniqueness of the therapy there are no off-the-shelf methods or kits to measure and quantify the cells used. A standardised, robust and accurate QC test method is required to ensure final product quality, safety and efficacy control.
Videregen will collaborate in the project with the cell metrology lab of the National Measurement Laboratory (NML). NML have the necessary expertise and facilities to help address the cell measurement problem, by understanding how the multiple cell properties/attributes in the BioThymus can be harnessed and streamlined into a reproducible QC method that Videregen can incorporate into their manufacture process.
BioThymus manufacture for preclinical studies and transition to clinical trials cannot be achieved without reliable QC methods that quantify and identify the cells in the product, which links to product effectiveness. QC methods to define BioThymus's cellular aspects is a critical activity/milestone for therapy development.
* Method will be used as a QC measure and final release test.
* Method will allow further research to understand relationships between thymic cell phenotype and their function to educate T-cells in-vivo, allowing manufacturing optimisation and therapy effectiveness.
* Method will provide regulatory bodies, clinicians and investors a high degree of confidence to adopt or invest in the technology.
Lead Participant | Project Cost | Grant Offer |
|---|---|---|
| VIDEREGEN LIMITED | £27,480 | £ 16,488 |
Participant |
||
| LGC LIMITED | £60,300 |
People |
ORCID iD |
| Paul Armitage (Project Manager) |