Full Description
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.
Contents
1. Overview of cell and gene therapy products
2. Safety risks of cell and gene therapy products
3. Regulatory frameworks for cell and gene therapy products
4. Quality control of cell and gene therapy products
5. Nonclinical toxicology studies
6. Advances in in vivo animal models for evaluation
7. Organoid and organ-on-chip models for evaluation
8. Evaluation of delivery systems
9. Immunogenicity assessment
10. Pharmacokinetics studies and evaluation
11. Tumorigenicity/oncogenicity assessment
12. Integration site analysis and off-target assessment
13. Nonclinical evaluation of stem cell products
14. Nonclinical evaluation of car-t cell products
15. Nonclinical evaluation of oncolytic viruses
16. Nonclinical evaluation of gene editing products
17. Nonclinical evaluation of nucleic acid drugs
18. Nonclinical evaluation of extracellular vesicle therapies
19. Nonclinical evaluation of red cell therapeutics
20. Nonclinical evaluation of combination therapies
21. On-site GLP inspection for registration Abbreviations
