Project Call 9.1

Project: Continuous and Automated Downstream Purification of AAV
Lead: Michigan Technological University
Participants: Cytiva, EMD Millipore Corporation, Janssen, Landmark Bio, and PAK BioSolutions

This project will advance process control and analytical technologies for Michigan Technological University’s aqueous two‑phase system (ATPS), which is used for continuous purification of viral vectors in gene therapy. The work aims to boost viral vector productivity and yield while reducing cost, labor, and analytical complexity.

Project: Leveraging Multidimensional Partner Data to Develop Predictive Digital Twins for Integrated Upstream Processes
Lead: University of Delaware
Participants: Sanofi and Waters Technologies Corporation

This project will develop and validate an integrated Digital Twin for CHO cell culture process that links process conditions and high-dimensional data to productivity and glycosylation Critical Quality Attributes (CQAs), using datasets from Waters. The goal is to advance digital twins as a predictive, scalable tool that speeds up upstream process development, improves robustness and product quality, and reduces experimental burden.

Project: Upstream Enrichment and Bioreactor Scale-Up for High Genome-Filled rAAV Capsids
Lead: Massachusetts Institute of Technology
Participants: EMD Millipore Corporation, Landmark Bio, and Virica Biotech

This project targets the high levels of empty capsids in recombinant adeno-associated viral vectors (rAAV), a major challenge in gene therapy manufacturing. This proposal integrates Decoupled Replication Initiated Vector Encapsulation (DRIVE) with model-based process control to create a production platform for high titer, high quality rAAV. By reducing empty capsids, the approach can streamline downstream purification, reduce time and cost, and improve the overall quality of gene therapy products.

Project: Continuous Fiber-Optic Probe for Detection of Antibodies and FC Fusions
Lead: University of Houston
Participants: Amgen, Boehringer-Ingelheim, Bristol-Myers Squibb, Genentech, and Mettler-Toledo Process Analytics

This project will advance the industrial readiness of an in-line or at-line fiber-optic probe capable of measuring monoclonal antibodies and Fc-fusion protein concentration in real time. This continuous PAT technology is designed to eliminate the delays, complexity, and costs associated with current offline methods.

Project: Intelligent Affinity: Bayesian Optimization of Full AAV Purification Using Convective Affinity Adsorbents
Lead: North Carolina State University
Participants: ChromaGenix, EMD Millipore Corporation, Genentech, Refeyn, University of North Carolina, Chapel Hill, and Waters Technologies Corporation

This project aims to fix two major challenges in making gene therapy products: slow, unreliable tools for separating full AAV particles and process development methods that require extensive trial and error. To address this, the team will develop improved purification materials that can better capture full AAVs, along with machine-learning software that identifies optimal process conditions. These tools will speed up development, improve product quality, and reduce costs.