De-risking AAV gene therapy: how early development and CMC decisions shape manufacturing and regulatory outcomes

Dr Clément has 25+ years of experience in the field of Gene Therapy, with industry leading expertise in adeno-associated virus (AAV) viral vectors manufacturing. At the Powell Gene Therapy Center of the University of Florida, Gainesville, she led AAV process development and manufacturing for R&D POC, IND-enabling Toxicology and IND studies. During this time, she oversaw the manufacturing, release testing and stability campaigns of several AAV INDs. Her research focused on developing scalable GMP-ready platforms using the HSV and the transfection systems in suspension. Dr Clément joined Resilience, a large CDMO, as the Director of the PD for Viral Vaccines and Gene Therapy, where she oversaw multiple industry-sponsored manufacturing programs. She joined Siren Biotechnology in January 2022, a clinical stage company focused on delivering a cure to cancer, where she leads manufacturing and CMC operations.

Patrick G Starremans, PhD is the Executive Director, Analytical Sciences at Solid Biosciences. Dr Starremans received his PhD in Medical Sciences and MSc in Medical Biology from the Radboud University in Nijmegen, the Netherlands, and an undergraduate degree in Biochemistry from Zuyd University of Applied Sciences.

He continued his post-doctoral training at Harvard University Medical School from 2003 to 2007 doing research on several rare genetic diseases. Following his move into Biopharma, he spent the next 10+ years completing tenures in discovery pharmacology & translational sciences at Genzyme, Sanofi, Evotec and Mitobridge, focused on molecular/biochemical and cell-based assay development in various genetic and metabolic disease areas.

In early 2018 he transitioned to the fast-growing field of Gene Therapy leveraging his assay development background and pivoting to CMC-Analytical Development when joining Voyager Therapeutics, where over the course of 3 years he progressed to lead analytical development for their CNS portfolio. In 2021 he joined Resilience US, an emerging CDMO, and built their analytical capabilities and team from the ground up as head of gene therapy analytical development. Finally, in 2024, he joined Solid Biosciences where he oversees the Analytical Development, AS&T and QC functions supporting their Neuromuscular and Cardiac disease portfolios

Christina Fuentes is a Principal at Dark Horse Consulting Group where she provides technical expertise in Cell and Gene Therapy to help clients address unique challenges in the field. Her specialties include supporting clients utilizing engineered nucleases for gene modification as well as non-viral and viral in vivo therapies. In her time at Dark Horse Consulting Group, Christina has supported over 100 clients ranging from regulatory strategy and authorship to long-term embedded CMC support. Dr. Fuentes co-authored the first externally prepared proposed draft guidance on “Testing of Adeno-Associated Viral (AAV) Vector-Based Human Gene Therapy Products for Empty Capsid During Product Manufacture” which was formally submitted to the FDA for consideration on May 15, 2022. In 2023, Christina co-authored a white paper titled “Beyond Empty and Full: Understanding Heterogeneity in rAAV Products and Impurities.” This document details the importance of product characterization and heterogeneity in product-related impurities to improve product understanding and inform manufacturing strategy.

Jacob Smith is Head of Technical Development & CMC Strategy at Viralgen, having previously served as the company’s Head of Manufacturing. Jacob came to Viralgen in 2023 from AskBio, where he served as VP of Process Development and Manufacturing for six years, and where he led the development of the Pro10™ manufacturing system. His industry experience includes roles at Pfizer, Bamboo Therapeutics, and UNC Chapel Hill Gene Therapy Center & Vector Core. He has extensive experience in molecular biology and virology, with special expertise in natural product isolation and identification, DNA sequencing and analysis, and AAV vector design and production, as well as manufacturing process development, implementation, and technology transfer.