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

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