Andrew Zydney, Ph.D.
Bayard D. Kunkle Chair and Professor of Chemical Engineering, The Pennsylvania State University
Sponsor: Department of Energy (DOE) Energy Frontier Research Center (EFRC) Materials for Water and Energy Systems (M-WET)
Title: New Opportunities for Membrane Technology in the Production of Next Generation Gene Therapies
Abstract
Membrane systems are used extensively for the purification of a wide range of important biological products. This presentation will focus on new / potential applications in the production of next generation gene therapy products, including both small interfering RNA (siRNA) and messenger RNA (mRNA) for vaccines. Although ultrafiltration can be used for the concentration of siRNA, existing membranes / modules are unable to achieve the high concentrations (>150 mg/mL) needed for final formulations. We have demonstrated the potential of using negatively-charged ultrafiltration membranes in this application, with more than a 3-fold increase in the maximum achievable concentration. There is growing interest in the development of continuous processes for mRNA manufacture, and we have shown that membranes can play a major role in these processes for both concentration (by single pass tangential flow filtration) and purification (by high performance countercurrent membrane purification). Sterile filtration can also be a challenge for mRNA formulations using lipid nanoparticles (LNP) due to the unique properties of the LNP. This talk highlights how new advances in membrane technology can address many of the critical issues involved in the production of these next generation biopharmaceuticals.