Abstract

Gene therapy medicinal products (GTMPs) represent a transformative medical innovation with the potential to cure previously untreatable genetic disorders. However, the journey from clinical development to commercial production is complex, particularly when it comes to meeting stringent regulatory chemistry, manufacturing and controls (CMC) requirements.

This article provides an in-depth exploration of the regulatory CMC landscape for gene therapies, emphasising the critical elements necessary to ensure quality, safety and efficacy throughout the product lifecycle. It highlights the evolving regulatory frameworks across key global markets, the unique challenges in scaling manufacturing processes and the importance of robust control strategies for critical quality attributes (CQAs).

From early-stage process development to commercial readiness, this article examines the delicate balance between innovation and compliance that will shape the future of gene therapy manufacturing. This comprehensive review aims to equip professionals with the knowledge to navigate the complex regulatory environment of gene therapy, facilitating successful transitions from clinical development to commercialisation.