Pharmaceutical Applications of CRISPR Technology
Abstract
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and its associated Cas proteins represent a transformative genome-editing platform derived from prokaryotic adaptive immune systems. This technology enables precise, programmable modifications to virtually any genomic locus, making it a powerful tool across biomedical research and pharmaceutical sciences. This review examines the current and emerging pharmaceutical applications of CRISPR technology, with particular emphasis on gene therapy for monogenic disorders, cancer immunotherapy, infectious disease treatment, drug discovery, and precision medicine strategies. Key applications discussed include CRISPR-mediated correction of pathogenic mutations in hemoglobinopathies, development of CAR-T cell therapies, high-throughput functional genomic screening for drug target identification, and generation of relevant disease models. CRISPR systems offer unparalleled advantages including versatility, scalability, low cost, and ease of design compared to earlier genome-editing tools such as zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). However, significant challenges remain, including off-target editing, efficient in vivo delivery, immunogenicity, and complex regulatory landscapes. Ethical concerns surrounding germline editing further complicate clinical translation. Despite these hurdles, early clinical trials have yielded promising results, particularly for sickle cell disease and beta-thalassemia. The continued development of base editing, prime editing, and CRISPR-based diagnostics expands the therapeutic repertoire. Overall, CRISPR technology holds substantial promise for revolutionizing pharmaceutical drug development and personalized medicine in the coming decade.
How to Cite This Article
Dr. Olivia J Wilson, Liam A Clarke, Dr. Mia R Bennett (2025). Pharmaceutical Applications of CRISPR Technology . International Journal of Pharma Insight Studies (IJPIS), 2(2), 22-27.