I-Corps: Translation Potential of a Continuous Manufacturing Process for Viral Vectors

This I-Corps project is based on the potential commercialization of a more effective method for manufacturing a viral gene therapy. The technology could also be applied to the manufacture of viral vaccines. This technology seeks to accelerate the manufacturing rate and reduce the costs associated with gene therapies for the treatment of life-threatening diseases, like melanoma, sickle cell anemia, and muscular dystrophy. More specifically, this continuous purification technology could reduce capital manufacturing costs by 90% and operating costs by 50%, improving the competitiveness of the U.S. biotherapeutic market. By bringing this solution to market, this technological advancement could increase the accessibility and affordability of gene therapies for patients with both common and rare diseases, reducing overall healthcare costs in the U.S. while improving patient quality of life and life expectancy. This I-Corps project utilizes experiential learning coupled with a first-hand investigation of the industry ecosystem to assess the translation potential of a continuous manufacturing process for viral gene therapy. The technology may reduce the costs of these biotherapeutics. Viral vector processing still uses batch processes, which are time consuming and expensive. This new solution is a continuous manufacturing process based on liquid-liquid extraction, which offers robust recovery (between 47-86%) for a diverse set of viral products, including adeno-associated virus (AAV) serotypes 2 and 9, herpesvirus, and lentivirus. This technology includes a large range of enveloped and non-enveloped viruses that can be purified with the same system. The continuous manufacturing approach has the potential to become a platform process for all viral particles, meaning that very little change in conditions is needed to purify a new viral particle. The ability to purify a wide range of viral particles with minimal adjustments makes the system highly scalable and adaptable, reducing the time and cost needed to develop purification processes for new viral therapies, vaccines, ande/or gene therapies. This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.