Turning Gene Therapies Into Patient Reality: Large Scale Cost-Effective AAV Production

By Samuel Hernandez, PhD, Senior Vice President, Site Head - Texas Operations, FUJIFILM Biotechnologies

As gene therapies move beyond rare indications, manufacturing strategy has become a defining factor in whether these treatments can reach broader patient populations. Scaling adeno‑associated virus production introduces challenges that go far beyond making more material, touching cost per dose, process robustness, product quality, and regulatory readiness.

Large‑volume manufacturing amplifies variability in upstream operations such as transient transfection, where factors that are manageable at small scale can significantly impact productivity at 2,000 L. Downstream, purification strategies must balance yield, resin costs, and reuse without compromising capsid quality or potency. At the same time, the financial and patient impact of a failed commercial‑scale batch raises the stakes for process control and real‑time monitoring.

This SME Spotlight explores how integrated, end‑to‑end process design—supported by single‑use systems, scalable analytics, and quality‑by‑design principles—can help align scale, cost, and consistency. It offers practical insight into the levers that most influence commercial viability and why building quality and control into AAV processes early is critical for protecting supply, accelerating timelines, and enabling sustainable patient access.