The Evolving Landscape of Cell and Gene Therapy—Opportunities, Challenges and the Road Ahead

Dear Colleagues,

Cell and gene therapies (CGT) have emerged as powerful modalities for treating conditions once considered intractable. With over 22 approved therapies to date and hundreds more in development, the field is undergoing rapid and transformative growth. Significant strides have been made in improving targeted delivery and reducing off-target effects, allowing for greater precision and safety in clinical applications.

Cell therapies—such as CAR-T cells—restore or enhance cellular functions by introducing viable, functional cells into patients. Gene therapies, in contrast, aim to correct or silence defective genes through direct modification of genetic material. Together, these approaches have expanded therapeutic possibilities for rare genetic disorders, cancers, and neurodegenerative diseases—particularly in patients who fail to respond to standard treatments or do not reach therapeutic targets.

This Special Issue focuses on the recent advances in CGT, with particular attention to addressing unmet medical needs over the past decade. It also highlights the critical role of in vitro and in vivo models in optimizing therapeutic strategies and the emerging potential to target previously inaccessible tissues and molecular pathways.

As we enter a new era of personalized and precision medicine, it is imperative to critically evaluate both the promise and limitations of CGT. This issue aims to foster dialogue and innovation toward more effective, scalable, and equitable therapeutic solutions.

Dr. Rai Ajit K. Srivastava
Guest Editor

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