Development of Cell and Gene Therapies to Treat, Prevent, and Potentially Cure Diseases

C.E. Credits: P.A.C.E. CE Florida CE
Speaker

Abstract

Cell therapy is broadly defined as the transfer of viable pluripotent, multipotent, and unipotent cells from either an allogeneic or autologous source after storage or manipulation. The use of manipulated advanced cellular therapy (ACT) products to treat patients, especially with advanced cancers, immune or degenerative conditions, is increasing as the benefits are realised, now with 25 FDA-approved ACTs and over 10,000 clinical trials. Though the potential for impact is high, challenges remain; variability in reported patient responses can result from unpredictable heterogeneity of infused ACTs, highlighting the unmet need for robust and reproducible biomanufacturing. Fundamental work done by my group using pluripotent, multipotent, and maturing unipotent cells has shown that metabolic signatures change well before genotype and phenotype indicating that what will happen to cells ultimately is governed by cellular metabolic fitness and culture conditions to which they are exposed, creating a pathway towards manipulating cellular heterogeneity and function through metabolism. The overall aim of this talk is to discuss how to characterise and direct cellular heterogeneity through the understanding and control of metabolism throughout the bioprocess using combined in vitro-in silico approaches in order to deliver personalised ACTs through Metabolism-Driven Precision Biomanufacturing for improved clinical outcomes.


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