Course

Unit 1

Unit 1

Fundamentals of Immune Cell Engineering, Overview of adoptive cell therapies (ACTs), Immune cell types used in therapy: T cells, NK cells, macrophages, Introduction to gene editing tools (CRISPR, TALENs, ZFNs), Vectors and delivery systems (viral and non-viral methods), Safety and specificity in immune cell engineering

Unit 2

Unit 2

CAR-T Cell Design and Mechanism of Action, Structural components of CARs (scFv, hinge, transmembrane, signaling domains), Generations of CARs and their evolution, Antigen selection and tumor targeting, Signal transduction pathways in CAR-T activation, Persistence, trafficking, and function of CAR-T cells in vivo

Unit 3

Unit 3:

CAR-T Cell Manufacturing and Quality Control, CAR-T production workflow: cell collection, transduction, expansion, Good Manufacturing Practices (GMP) and facility requirements, Quality assurance and release criteria, In-process testing: viability, sterility, identity, and potency, Cryopreservation and product stability

Unit 4

Unit 4

Clinical Applications and Trials of CAR-T Therapy, Approved CAR-T therapies (e.g., Kymriah, Yescarta, Tecartus), Indications: hematologic malignancies, emerging solid tumor strategies, Clinical trial design and endpoints for cell therapies, Efficacy assessment and patient monitoring, Adverse effects: cytokine release syndrome (CRS), neurotoxicity, on-target off-tumour effects.

Unit 5

Unit 5

Emerging Strategies in Immune Cell Engineering, Next-generation CARs: dual CARs, logic-gated CARs, armoured CAR-Ts, Universal and allogeneic CAR-T approaches, NK cell-based and macrophage-based CAR therapies, TCR-T therapy vs CAR-T, Synthetic biology in immune cell programming

Unit 6

Unit 6

Regulatory, Ethical, and Translational Aspects, Regulatory pathways for cell and gene therapies (FDA, EMA, CDSCO), Ethical considerations: gene editing, accessibility, informed consent, Challenges in commercialization and global access, Case studies in approval, pricing, and reimbursement, Future outlook and barriers to widespread clinical translation.

(45 classes)

Preamble

CAR-T and Immune Cell Engineering is an advanced course focusing on the principles and applications of engineering immune cells, particularly Chimeric Antigen Receptor T-cell (CAR-T) therapy. The course will cover the design, development, and therapeutic potential of engineered immune cells in targeting cancers and other diseases. Through lectures, case studies, and practical sessions, students will gain insights into the cutting-edge techniques and challenges in immune cell engineering.

Course Outcome:

CO1: Describe the fundamental concepts of immune cell engineering and the technologies used to modify immune cells for therapeutic purposes.

CO2: Explain the structure, function, and mechanism of action of CAR-T cells and analyze how design elements influence therapeutic outcomes.

CO3: Demonstrate an understanding of CAR-T manufacturing processes, GMP standards, and quality control measures necessary for clinical application.

CO4: Evaluate clinical applications, trial designs, and safety concerns associated with CAR-T therapies in hematologic and solid tumors.

CO5: Analyze emerging strategies in immune cell engineering, including advanced CAR constructs and alternative immune cell therapies.

CO6: Discuss regulatory frameworks, ethical issues, and translational challenges involved in the commercialization and global accessibility of CAR-T therapies.

Program outcome

PO1: Bioscience Knowledge

PO2: Problem Analysis

PO3: Design/Development of Solutions

PO4: Conduct Investigations of complex problems

PO5: Modern tools usage

PO6: Bioscientist and Society

PO7: Environment and Sustainability

PO8: Ethics

PO9: Individual & Team work

PO10: Communication

PO11: Project management & Finance

PO12: Lifelong learning

0 – No affinity; 1 – low affinity; 2 – Medium affinity; 3 – High affinity

Program-specific outcome

PSO 1 – Emerging technologies in cell therapy

PSO 2 – Biomolecules in Medicine

PSO 3 – Molecular dysregulation in diseases

PSO 4 – Molecular technology in diagnosis and therapy

PSO 5 – Applying lab discoveries to clinical practice

PSO 6 – Advanced techniques in cell culture, gene editing, and bioprocessing

PSO 7 – Statistical methods to interpret and validate diagnostic results

PSO 8 – Integrate cell therapy into personalized medicine

PSO 9 – GMP and regulatory practices in cell therapy production

PSO 10 – Bioinformatics and biological data use

0 – No affinity; 1 – low affinity; 2 – Medium affinity; 3 – High affinity

Textbook

Kröger, N., Gribben, J. G., Chabannon, C., Yakoub-Agha, I., & Einsele, H. (Eds.). (2022). The EBMT/EHA CAR-T Cell Handbook (1st ed.). Springer.

Reference book

Cornell University. (n.d.). Engineering Materials for Immunomodulation. Cornell eCommons.