Disease & Drug Development (Applications Theory)

Graduate

This course covers the theory of bioassay design, development, and validation—including high-throughput screening and PK/PD—alongside advances in cancer and infectious disease immunotherapies, mRNA therapeutics, and in vivo cell and gene engineering technologies for targeted therapeutic delivery. Bioassay Design, Development and Validation: Introduction to bioassays and statistical concepts for design, development, validation, and monitoring (including repeatability, precision, acceptance criteria, etc.). 1) High Throughput Screening (HTS) Instrumentation, Robotics/Automation 2) Pharmacokinetics/Pharmacodynamics (PK/PD) Advances in immunotherapies: current advances in immunotherapies to treat disease. 1) cancer (checkpoint inhibitors, CAR T-cell therapy, cytokines, immunomodulators, cancer vaccines, antibodies, oncolytic virus), 2) infectious diseases (T-cell engineering, activation of lymphocytes, antibody/ligand-based therapies). mRNA therapeutics: advances in formulation, targeted delivery, and immunogenicity of mRNA-based vaccines. 1) Applications in infectious diseases (COVID-19) 2) Applications in oncology 3) Applications in cardiology. d) In vivo Cell and Gene Engineering: latest developments in in vivo cell engineering and delivery of genetic cargos to targeted immune cells directly inside patients. 1) gene-editing technologies (mRNA, viral vectors, and nanoparticles) 2) Strategies to improve targeting, specificity, and delivery Offered Spring Term