The manufacturing process for voretigene neparvovec has undergone significant development from the initial process used for Phase 1 clinical material to the final commercial process. Key improvements included:
- Scale-up from cell culture flasks to stirred-tank bioreactors to meet commercial demand.
- Optimization of the purification scheme, including the introduction of an additional ion-exchange chromatography step, to improve the removal of process- and product-related impurities, particularly empty capsids.
- Implementation of a more robust and scalable Tangential Flow Filtration (TFF) system for the final concentration and formulation step.