Nonclinical Evaluation Studies of Cellular and Gene Therapy Products

Strategies, Models, and Regulatory Frameworks

Quanjun Wang, Qingli Wang, Xingchao Geng, Wang Quanjun, Wang Qingli, Geng Xingchao

• Publisher: Elsevier
• Year: 2026
• eBook ISBN:
  9780443486135
• Topic: Medicine
• Subtopic: Pharmacology

Nonclinical Evaluation Studies of Cellular and Gene Therapy Products: Strategies, Models, and Regulatory Frameworks systematically explores the nonclinical evaluation of Cell and Gene Therapies (CGT) products, emphasizing risk mitigation and innovation. The book provides comprehensive coverage, including 20+ chapters spanning preclinical toxicology, pharmacokinetics, and novel evaluation models. It is the first book to integrate organ-on-chip models into CGT evaluation, reflecting FDA/NMPA modernization initiatives. It includes practical guidance on addressing CRISPR off-target effects, immunogenicity, and long-term carcinogenicity risks. - Includes definitions, regulatory policies (US, EU, China), and quality control requirements - Provides in-depth coverage of humanized animal models, organ-on-chip systems, and 3D bioprinted tissues for toxicity screening - Covers evaluation strategies for mRNA drugs, exosome-based therapies, and red blood cell-mediated drug delivery - Includes case studies: FDA-approved CRISPR therapies, CAR-T safety assessments, and lessons from failed trials

Table of contents

• Title of Book
• Chapter 1. Overview of Cellular and Gene Therapy Products
• Chapter 2. Safety Risk Considerations of Cellular and Gene Therapy Products
• Chapter 3. Overview of Administrative Regulation for Cellular and Gene Therapy Products
• Chapter 4. Quality Control of Cellular and Gene Therapy Products
• Chapter 5. Nonclinical Toxicology Studies for Cellular and Gene Therapy Products
• Chapter 6. Research Progress on In Vivo Animal Models for the Evaluation of Cellular and Gene Therapy Products
• Chapter 7. Research Progress on In Vitro Organoid and Organ-on-a-Chip Models for the Evaluation of Cellular and Gene Therapy Products
• Chapter 8. Evaluation Research on Delivery Systems for Cellular and Gene Therapy Products
• Chapter 9. Immunogenicity Evaluation
• Chapter 10. Pharmacokinetic Research and Evaluation
• Chapter 11. Tumorigenicity/Oncogenicity Evaluation of Cellular and Gene Therapy Products
• Chapter 12. Integration Site Analysis and Off-target Analysis of Cellular and Gene Therapy Products
• Chapter 13. Nonclinical Evaluation of Stem Cell-Based Products
• Chapter 14. Nonclinical Evaluation of CAR-T Products
• Chapter 15. Nonclinical Evaluation Studies of Oncolytic Viruses
• Chapter 16. Nonclinical Evaluation of Gene-Editing Products
• Chapter 17. Nonclinical Evaluation Studies of Nucleic Acid Drugs
• Chapter 18. Nonclinical Evaluation Studies of Extracellular Vesicle-Based Drugs
• Chapter 19. Nonclinical Evaluation Studies of Red Cell Therapeutics Products
• Chapter 20. Nonclinical Research and Evaluation of Combination Therapies
• Chapter 21. Key Points of On-Site Inspection for Nonclinical Evaluation in Registration Applications
• Case Studies
• Index