Personalized Gene Therapy Is Here. The Next Challenge Is Manufacturing.

By Tony Duong, Senior Drug Delivery Scientist, Battelle Memorial Institute

The successful rapid development of a personalized CRISPR therapy for a seven-month-old patient signals a turning point in medicine, shifting the manufacturing focus from volume-driven "scale-up" to process-driven "scale-in". This new paradigm replaces centralized mass production with a flexible, decentralized ecosystem capable of creating unique, high-quality treatments at the point of care.

Achieving this requires optimizing four critical pillars: accelerating custom DNA template synthesis through enzymatic ligation, streamlining RNA production with stable circular RNA modalities, utilizing tissue-specific delivery vehicles like polymer-based nanoparticles, and validating efficacy via patient-representative cell models. As regulatory frameworks evolve toward approving these standardized platform processes rather than individual products, the industry must prioritize automated, in-line quality control to ensure safety at the speed patients require. Learn how these innovations are making personalized gene therapy a scalable reality.