Evaluating Analytical Strategies to Quantify Capsid Titre: Towards a Platform-Method Approach to Accelerate AAV Drug Product Development

Traditional capsid titre methods rely on ELISA which commonly suffers from long turnaround times, low throughput, and large volume sample requirements. This limits the application of ELISA-based methods to routine analysis, thus requiring development of alternative high-throughput (HTP) capsid titre methods. We have performed a comprehensive assessment on currently available orthogonal capsid titre methods using multiple serotypes at concentration range relevant in IND-enabling preclinical and first-in-human (FIH) clinical studies.

Presented by Dr. Marilia Barros, PhD, Principal Scientist Biologics & Gene Therapy Drug Product Development at Regeneron

Dr. Barros is an expert in biologics and gene therapy drug product development with over 8 years of experience in the pharma and biotech industry, providing primary support for therapeutic areas in early through late phase development projects and spearheading the development of parenteral dosage forms to enable clinical and commercial use. She received her PhD in Physics from CMU and has worked at NIST Center for Neutron Research and Memorial Sloan Kettering Cancer Center. At Bristol Myers Squibb, she developed and led internal and external scientific collaborations to drive key innovation that impacted the progression of the sterile products portfolio and investigated the colloidal stability of biotherapeutics to support drug development and new strategies of drug product formulation. Currently, she works at Regeneron as a Principal Scientist developing strategies to identify and implement new technologies to advance the pipeline and serves as CMC drug product formulation development lead for Regeneron’s gene therapy programs.

Followed by Dr. Matt Ranaghan, Senior Applications Scientist, at Refeyn

Matt is a Senior Applications Scientist in the Market Development group at Refeyn. He has over 10 years of industry experience in small-molecule drug discovery and developing gene editing technologies. As a protein scientist, he has worked with various classes of proteins including GPCRs, enzymes, multimeric complexes and ribonucleoprotein complexes. Matt holds a PhD in Biochemistry from the University of Connecticut, where he studied the application of photoactive proteins in biophotonic devices.

Sponsored by Refeyn

Refeyn pioneers analytical instruments that put molecular mass measurement capabilities within easy reach for scientists. Refeyn’s unique products measure the mass of individual proteins, nucleic acids, complexes and viruses directly in solution – providing vital insights for scientific discovery, R&D and therapeutics production.

Our instruments feature mass photometry technology, which uses light to quantify the mass of single particles in solution without labels, and macro mass photometry technology, which uses light to characterize large viral vectors. Providing intuitive data in minutes, mass photometry technologies help scientists solve their research questions, optimize R&D processes and focus on innovation.