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Vectors are the unsung heroes of cell and gene therapy—molecular couriers that transport therapeutic genes into human cells to treat disease. But as Alan Griffith, Head of Global Operations at Vector Builder reveals, creating these sophisticated delivery vehicles is far more complex than most realize.
Drawing from over a decade of experience developing gene therapies before joining the CDMO sector, Griffith offers rare insight into what makes manufacturing these therapies so challenging. Vector design emerges as the critical yet underappreciated factor determining success or failure. "The fundamental sequence and vector composition itself is so fundamental to manufacturability," Griffith explains, noting how seemingly minor elements can dramatically impact production at scale. This knowledge has evolved painfully over the past decade, with clinical holds and manufacturing failures teaching hard lessons.
The relationship between therapy developers and contract manufacturers proves equally crucial. Rather than transactional exchanges, successful partnerships require deep collaboration and shared risk. "Working with a CDMO is like dancing with someone you don't know," Griffith observes, emphasizing how Vector Builder aims to understand diseases and vector compositions thoroughly rather than simply executing manufacturing orders.
Cost remains perhaps the greatest challenge facing cell and gene therapies. While small molecule drugs might be considered expensive at $10,000 per dose, that figure would be remarkably affordable for gene therapies. Strategies to address this include better vector design to reduce manufacturing failures, more targeted delivery requiring smaller doses, and challenging the "one-and-done" assumption that has proven more complex in reality as expression often diminishes over time.
Looking ahead, Griffith sees complementary roles for both viral vectors and emerging non-viral delivery systems like lipid nanoparticles and exosomes. He predicts viral vectors will continue to dominate certain applications for decades due to their unmatched effectiveness, while non-viral approaches may offer manufacturing advantages and potentially lower costs.

Cell and gene therapies are revolutionary treatments with vectors serving as "molecular couriers" that deliver genetic materials to target cells, but manufacturing these therapies presents significant challenges in scaling, cost, and regulatory compliance.
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