About this Research Topic
Cellular therapies targeting the immune system are fast becoming a mainstay of medical treatment regimens for many diseases. One long-term goal for cellular therapies is to develop allogeneic, “off-the-shelf,” products which can be produced in large quantities to enable treatment of hundreds or even thousands of patients from each lot of cells. Manufacturing cellular therapies at sizeable scales while maintaining control over variables impacting efficacy is not a trivial matter. Late-stage clinical trials involving hundreds or thousands of patients can require the manufacture of multiple lots, each derived from different seed stocks of primary cells. Demonstrating that the potency of each manufactured batch of cells is comparable is critical for obtaining regulatory approval.
Establishing potency assays can be problematic for complex cellular therapies given that, even if the mechanism of action is clear, the exact factor(s) contributing to efficacy might not be definitively known. To ensure comparability between manufactured lots, FDA guidance calls for a matrix of potency assays that orthogonally define cell function in a particular disease indication. These assays must be robust and quantitative, demonstrating accuracy and repeatability in good manufacturing practice (GMP) compliant testing facilities.
This Research Topic strives to present a diverse range of approaches to developing potency assays for immunomodulatory cellular therapies that are directly translatable to incorporating into a cellular therapy manufacturing environment. We invite contributions from leaders in the field who are actively involved in commercial development of immunomodulatory cellular therapies.
We encourage the submissions of Original Research, Review, Mini-Review, Perspective, Method, and Opinion articles centered on immunomodulatory cellular therapies or products derived therefrom, as well as novel tools or instrumentation to accelerate potency assay development.
Examples of applicable topics include:
• Development of novel immunological disease-relevant potency factors as scalable manufacturing platforms
• Biopotency and surrogate assays to validate therapeutic efficacy of cellular therapies
• Strategies for method validation prior to implementation in a GMP testing facility
• New instrumentation to facilitate potency factor discovery or quantitative assessment
• Regulatory perspectives
Examples of relevant cellular therapies, either isolated from human specimens or induced from pluripotent cells, include:
• CAR-T cell therapies
• CAR-engineered NK cells, macrophages and Tregs
• Myeloid-derived suppressor cells (MDSC)
• Tolerogenic dendritic cells (tolDCs)
• Culture-expanded Tregs
• Tumor-infiltrating lymphocytes (TILs)
• Mesenchymal stem/stromal cells (MSCs)
• Extracellular vesicles (EVs)
Dr. Brian Johnstone is Vice President of Research at Ossium Health, which is involved in developing stem cell-based therapies to improve treatments for patients suffering from hematologic diseases, organ transplant rejection, and orthopedic trauma. The other topic editors declare no competing interests with regard to the topic theme.
Establishing potency assays can be problematic for complex cellular therapies given that, even if the mechanism of action is clear, the exact factor(s) contributing to efficacy might not be definitively known. To ensure comparability between manufactured lots, FDA guidance calls for a matrix of potency assays that orthogonally define cell function in a particular disease indication. These assays must be robust and quantitative, demonstrating accuracy and repeatability in good manufacturing practice (GMP) compliant testing facilities.
This Research Topic strives to present a diverse range of approaches to developing potency assays for immunomodulatory cellular therapies that are directly translatable to incorporating into a cellular therapy manufacturing environment. We invite contributions from leaders in the field who are actively involved in commercial development of immunomodulatory cellular therapies.
We encourage the submissions of Original Research, Review, Mini-Review, Perspective, Method, and Opinion articles centered on immunomodulatory cellular therapies or products derived therefrom, as well as novel tools or instrumentation to accelerate potency assay development.
Examples of applicable topics include:
• Development of novel immunological disease-relevant potency factors as scalable manufacturing platforms
• Biopotency and surrogate assays to validate therapeutic efficacy of cellular therapies
• Strategies for method validation prior to implementation in a GMP testing facility
• New instrumentation to facilitate potency factor discovery or quantitative assessment
• Regulatory perspectives
Examples of relevant cellular therapies, either isolated from human specimens or induced from pluripotent cells, include:
• CAR-T cell therapies
• CAR-engineered NK cells, macrophages and Tregs
• Myeloid-derived suppressor cells (MDSC)
• Tolerogenic dendritic cells (tolDCs)
• Culture-expanded Tregs
• Tumor-infiltrating lymphocytes (TILs)
• Mesenchymal stem/stromal cells (MSCs)
• Extracellular vesicles (EVs)
Dr. Brian Johnstone is Vice President of Research at Ossium Health, which is involved in developing stem cell-based therapies to improve treatments for patients suffering from hematologic diseases, organ transplant rejection, and orthopedic trauma. The other topic editors declare no competing interests with regard to the topic theme.
Keywords: cellular immunotherapies, immunomodulatory, potency assays, therapeutic efficacy, cellular therapy manufacturing
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
