Course

Unit 1

Unit 1:

Fundamentals of Gene and Cell Therapy, History and evolution of gene and cell therapy, Overview of genetic diseases and cellular dysfunctions, Distinction between gene therapy and cell therapy, Key therapeutic goals: gene addition, editing, silencing, and cell replacement, In vivo vs ex vivo strategies

Unit 2

Unit 2:

Gene Therapy Vectors, Viral vectors: lentivirus, retrovirus, adenovirus, AAV, Non-viral vectors: liposomes, nanoparticles, electroporation, Design considerations for safe and effective vectors, Vector production and quality control, Case examples of vector-based therapies

Unit 3

Unit 3:

Delivery Mechanisms and Targeting Strategies, Systemic vs local delivery routes, Tissue-specific targeting and promoter design, Barriers to delivery (immune response, degradation, off-target effects), Enhancing transduction/transfection efficiency, Case studies on delivery innovations

Unit 4

Unit 4:

Cell Therapy: Types and Applications, Autologous vs allogeneic cell therapies, Stem cells in therapy: embryonic, adult, iPSCs, CAR-T cells and immune cell engineering, Regenerative medicine and tissue engineering, Clinical applications: hematologic cancers, neurodegenerative diseases.

Unit 5

Unit 5:

Clinical Trials and Approved Therapies, Overview of clinical trial phases for advanced therapies, Approved therapies (e.g., Luxturna, Zolgensma, Kymriah), Challenges in efficacy, safety, and scalability, Analysis of breakthrough studies and trial designs, Personalized vs off-the-shelf approaches

Unit 6

Unit 6:

Ethical, Regulatory, and Commercial Considerations, Regulatory frameworks: FDA, EMA, CDSCO perspectives, Ethics of genetic modification and germline editing, Manufacturing, quality control, and GMP in ATMPs, Market access, pricing, and patient access, Future trends and multidisciplinary collaboration

(45 classes)

Preamble:

Designed to provide a foundational understanding of cell and gene therapy, this course covers key topics such as vector design, delivery mechanisms, therapeutic strategies, and regulatory considerations. Through lectures and analysis of current case studies, students will explore the latest advancements and challenges in emerging medical treatments, equipping them with the knowledge to contribute to this rapidly evolving field.

Course Outcomes:

CO1: Describe the foundational concepts and therapeutic strategies of cell and gene therapy, including in vivo and ex vivo approaches.

CO2: Explain the design, types, and safety considerations of viral and non-viral vectors used in gene delivery.

CO3: Analyze various delivery methods and targeting strategies, and evaluate challenges in effective gene transfer.

CO4: Distinguish between different cell therapy types and discuss their clinical applications in disease treatment.

CO5: Interpret clinical trial data and approved cell and gene therapy products, assessing challenges in efficacy and safety.

CO6: Identify key ethical, regulatory, and commercial considerations involved in the development and deployment of advanced 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

Kantoff, P. W. (Ed.). (2010). Introduction to Gene and Cell Therapy. Wiley-Blackwell.

Reference

Nichols, C. D. (Ed.). (2003). An Introduction to Molecular Medicine and Gene Therapy. Wiley-Liss.