Course

Unit 1

Unit 1

Introduction to Cell Therapy and Regulatory Landscape, Overview of cell therapy and regenerative medicine, Historical perspective and evolution of regulations, Types of cell-based products (e.g., somatic, stem, genetically modified), Need for regulation in cell therapy, Key stakeholders: regulators, developers, clinicians, patients.

Unit 2

Unit 2

Regulatory Authorities and Global Guidelines, Role and structure of regulatory bodies: FDA (USA), EMA (EU), CDSCO (India), PMDA (Japan), WHO and ICH guidelines, Classification and regulation of Advanced Therapy Medicinal Products (ATMPs), Global harmonization and differences in regulatory pathways.

Unit 3

Unit 3

Approval Pathways and Compliance Requirements, Investigational New Drug (IND) and Clinical Trial Applications (CTA), Preclinical requirements: GLP compliance, GMP standards in cell manufacturing, Clinical trial phases and Good Clinical Practice (GCP), Post-marketing surveillance and pharmacovigilance.

Unit 4

Unit 4

Ethical and Legal Considerations in Cell Therapy, Ethical concerns: donor consent, genetic manipulation, stem cell sourcing, Legal issues: ownership of biological material, intellectual property rights, Bioethics committees and Institutional Review Boards (IRBs), Cases of malpractice and regulatory response.

Unit 5

Unit 5

Risk Assessment and Quality Control, Risk classification of cell-based therapies, Safety testing: sterility, endotoxins, tumorigenicity, Product characterization and potency assays, Quality Assurance (QA) and Quality Control (QC) systems, Batch release and lot-to-lot consistency.

Unit 6

Unit 6

Case Studies and Regulatory Challenges in Clinical Translation, Case studies of approved cell therapies (CAR-T, stem cell therapies), Regulatory setbacks and lessons learned, Challenges in scaling up and commercialization, Real-world strategies for regulatory navigation, Emerging trends and future regulatory considerations.

(45 classes)

Preamble

Regulatory Aspects of Cell Therapy is a specialized course designed to familiarize students with the legal and regulatory frameworks governing cell-based therapies. The course will explore compliance standards, approval pathways, ethical considerations, and global regulatory guidelines essential for the clinical translation and commercialization of cell therapies. Through case studies and discussions, students will learn about the challenges and strategies in navigating the regulatory landscape of cell therapy.

Course Outcome:

CO1: Explain the evolution and importance of regulatory frameworks in cell therapy, including key stakeholders and categories of cell-based products.

CO2: Compare global regulatory authorities and guidelines, including FDA, EMA, CDSCO, and ICH, and their roles in classifying and regulating ATMPs.

CO3: Describe the approval pathways, preclinical and clinical compliance requirements, and post-marketing responsibilities for cell therapies.

CO4: Analyze ethical and legal considerations such as informed consent, intellectual property, and oversight by IRBs and bioethics committees.

CO5: Evaluate risk management strategies, including safety testing, quality control, and assurance systems essential for clinical-grade products.

CO6: Interpret real-world regulatory challenges and case studies to develop strategic approaches for successful clinical translation and commercialization.

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

Galli, M. C. (Ed.). (2023). Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective. Springer.

Reference

National Institutes of Health (NIH). (2024). Regulatory Knowledge Guide for Cell and Gene Therapies.