Safety & efficacy testing solutions for cellular therapies areas - Medical / Health Care
The key FDA’s and EMA’s biosafety concerns (1,2) are the potential for cell based tumorigenicity, immunogenicity, infectious disease, and or genetic modification. Cellular therapies are aimed at specific disease types and to proliferate and differentiate into the desired cell type without causing unnecessary and potentially dangerous proliferation into cancer cells or unwanted immunogenicity causing damage in surrounding tissue.
Overview Applications and Industries
Two key safety aspects of cellular therapy products include toxicity and efficacy and the establishment of the correct dose. Often this can not be achieved using an animal model. The need for new toxicological and immunogenicity assays using human tissue is clear.
- Using the Skimune® Platform to assess Safety and Specificity of Cellular Therapies.
- Do you need to test the safety and efficacy of your cellular products?
- Do you need to verify a lack of immunogenicity in your cellular product?
- Is your cellular product tissue specific?
- Do you want to assess bystander cytokine release due to your cellular product?
A significant challenge is the need to understand the immunotoxicity risks that may be associated with cellular therapy products.
Research by the founder and CEO of Alcyomics, has shown that Skimune® can assist with the assessment of immunogenicity and specificity of cellular products, including, those in use for combatting haematological disorders, viral infections and auto immune disease (3 and 4).
The Skimune® platform has been shown to demonstrate the specificity of T-cell clones developed for use in the treatment of leukaemia and the work was published in Nature Medicine in 2002 (3 and 4). More recently the same Skimune® platform was used to show the safety and efficacy of anti cytomegalovirus (CMV) T cells used in the treatment of CMV post haematopoietic stem cell transplantation.
Alcyomics, as a member of the Northern Alliance for Advanced Therapies Treatment Centre (NA-ATTC) has conducted safety assessments on two novel cellular products, an anti COVID T-Cell specific product and assessment of the safety of peptides for use in a tolerogenic dendritic cell therapy for the treatment of rheumatoid arthritis.
Through the partnerships created through the NA-ATTC a whitepaper has been produced on the regulations concerning the safety of cellular therapies.