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Elsevier

Nonclinical Evaluation Studies of Cellular and Gene Therapy Products

Strategies, Models, and Regulatory Frameworks

  • 1 Edición - 1 de octubre de 2026
  • Última edición
  • Editores: Quanjun Wang, Qingli Wang, Xingchao Geng
  • Idioma: Inglés

Nonclinical Evaluation Studies of Cellular and Gene Therapy Products: Strategies, Models, and Regulatory Frameworks systematically explores the nonclinical evaluation of Cell a… Leer más

Descripción

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.

Puntos claves

  • 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

De interès para

Industry professionals: Nonclinical researchers, toxicologists, and CMC teams in biopharma Clinical Researchers: Investigators designing first-in-human trials for gene-edited therapies

Índice

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

Detalles del producto

  • Edición: 1
  • Última edición
  • Publicado: 1 de octubre de 2026
  • Idioma: Inglés

Sobre los editores

QW

Quanjun Wang

Dr Quanjun Wang is CEO and Chief Scientist of the Safe Medical Research Institute and Vice President of the Suzhou Institute of Biomedical Engineering and Technology, Chinese Academy of Sciences. He is a Distinguished Professor at the Hefei Institutes of Physical Science, Chinese Academy of Sciences; Adjunct Doctoral Supervisor, School of Pharmacy, East China Normal University

Dr Wang has over 20 years of experience in innovative drug R&D and nonclinical safety evaluation. He has published 140+ peer-reviewed papers and authored/co-authored eight monographs in toxicology and regulatory science. He golds 20+ patents in drug evaluation methodologies
Afiliaciones y experiencia
Vice President, Suzhou Institute of Biomedical Engineering and Technology, Chinese Academy of Sciences, China

QW

Qingli Wang

Dr Qingli Wang is Chief Pharmacist and Senior Reviewer at the Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA). Dr. Wang joined the CDE in 2002 and has since specialized in the evaluation of drug safety studies. Currently serving as Director of the Department of Pharmacology and Toxicology at the CDE, he leads technical reviews for pharmacological and toxicological studies of drugs and spearheads the development of China’s technical guidance system for non-clinical drug research.

Concurrently, he holds the position of Chair of the Drug Toxicology Committee at the Chinese Pharmacological Society
Afiliaciones y experiencia
Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA), China

XG

Xingchao Geng

Professor Xingchao Geng a Research Professor and Director of the National Center for Safety Evaluation of Drugs at the National Institutes for Food and Drug Control (NIFDC), China. Dr. Geng serves concurrently as: Chair of the Drug Safety Evaluation Research Committee, Chinese Pharmaceutical Association, National GLP Inspector and National Expert for New Drug Evaluation. With over 20 years of experience in drug safety evaluation research, he has directed preclinical safety assessments for more than 200 domestic and international new drug candidates
Afiliaciones y experiencia
National Center for Safety Evaluation of Drugs at the National Institutes for Food and Drug Control (NIFDC), China