Translational Readiness in Gene and Cell Therapy: Biomarkers, Safety, and Monitoring

Gene and cell therapies are reshaping the treatment landscape for inherited, degenerative, and acquired disorders. Yet successful translation depends on more than proof of concept. Progress is slowed by challenges in vector and product characterisation, target validation, biomarker development, biodistribution assessment, and safety profiling, alongside the need for robust frameworks to monitor long-term therapeutic durability. These challenges are especially acute in complex disease settings where treatment response, biological heterogeneity, and extended follow-up must all be accounted for. At the same time, advances in bioengineering, analytical tools, and translational model systems are creating new opportunities to strengthen therapy readiness ahead of broad clinical implementation. A focused discussion of these enabling strategies is needed to bridge the gap between laboratory innovation and effective therapeutic application.This Research Topic brings together studies that advance the preclinical and translational development of gene and cell therapies through biological validation, engineering innovation, and clinically relevant monitoring. We welcome contributions addressing how therapeutic candidates can be better designed, evaluated, and prepared for clinical use, including work on vector optimisation, potency assessment, target engagement, immune response characterisation, and treatment-monitoring platforms. Particular interest will be given to studies that link mechanistic insight with practical development needs to improve translational decision-making. Submissions may focus on inherited disorders, degenerative disease, regenerative applications, or other areas where gene- and cell-based interventions are being advanced, provided the work presents a clear bioengineering or biotechnology contribution relevant to therapy readiness or product performance.We welcome original research, systematic reviews, methods, and perspective articles on the bioengineering and biotechnology of gene and cell therapy development. Suitable submissions include studies on vector design and delivery, preclinical efficacy and safety, biomarker and endpoint validation, immunogenicity, biosafety, manufacturing or quality considerations, translational animal or organoid models, and monitoring technologies supporting treatment evaluation. Manuscripts should present a clear biological, translational, or technological contribution. Purely clinical, epidemiological, or statistical studies lacking a strong bioengineering/biotechnology component are not suitable, and clinical trials should not be submitted. Computational analyses should include appropriate experimental or biological validation where required. Topic editors: Vahid Mansouri, Jun Liu Source: https://www.frontiersin.org/research-topics/83224/translational-readiness-in-gene-and-cell-therapy-biomarkers-safety-and-monitoring