Panel discussion: challenges and advances in AAV therapy bioanalysis

What will you learn?

• The bioanalytical methods used to assess biodistribution, transgene expression and immunogenicity for AAV therapies
• Strategies to ensure compliance with evolving regulatory guidelines
• Major challenges in the field, including the evaluation of immunogenicity and safety profiles of AAV therapies
• Key technologies and advances that will shape the future of AAV therapies

Who may this interest?

• Biotech and pharma professionals involved in gene therapy development
• Preclinical and clinical researchers focused on gene therapy safety
• Bioanalytical scientists seeking innovative approaches to assess AAV therapies
• Regulatory and quality assurance teams in biotech aiming to gain insights into clinical trial preparation
• Drug developers working on translating gene therapies from lab to clinical applications

Speakers

Catherine Spickler

Associate Director of Immunology (MA, USA)

Charles River Laboratories

Catherine started her career in the pharmaceutical industry over 25 years ago, focusing on early drug discovery. She started her path at Charles River (MA, USA) 5 years ago as a preclinical safety Immunology Scientist specializing in molecular biology. She rapidly evolved to being one of the Immunology department Associate Directors and helped build what is now the Molecular Biology team at the Charles River Laval site in Canada. As a subject matter expert in molecular biology, she drives the scientific harmonization effort across the Charles River global organization.

Yanmei Lu

Vice President of Biomarker and Bioanalytical Sciences

Sangamo Therapeutics (CA, USA)

Dr Yanmei Lu is currently the Vice President of Biomarker and Bioanalytical Sciences at Sangamo Therapeutics. The functions of her group include biomarker strategy, bioanalysis and companion diagnostic development to support gene and cell therapy programs at both preclinical and clinical stages. Prior to joining Sangamo, Dr Lu acquired extensive experience at Genentech (CA, USA) in developing PK, biomarker, immunogenicity and functional assays for antibody screening, candidate selection and in vivo studies to evaluate the PK/PD, efficacy and safety of large molecule therapeutics in disease areas such as neuroscience, immunology, oncology and ophthalmology. Dr Lu holds a PhD in Biochemistry and Molecular Biology from the University of South Florida (FL, USA) and has published over 50 peer-reviewed articles and book chapters. She is also the editor for the book ‘Drug Development for Gene Therapy: Translational Biomarkers, Bioanalysis, and Companion Diagnostics’.

Tong-Yuan Yang

Senior Director of Bioanalytical Sciences

Johnson & Johnson Innovative Medicine (PA, USA)

Tong-Yuan Yang is a Senior Director in the Bioanalytical Sciences group at Johnson & Johnson Innovative Medicine (JJIM), where he leads the development and validation of bioanalytical assays and their implementation in non-clinical and clinical study bioanalysis to support JJIM’s pipelines. Before joining JJIM, Tong had worked at Merck Research Laboratories (NJ, USA) focusing on drug discovery and product development. Tong obtained his PhD in Molecular Virology from Pennsylvania State University (PA, USA), where he also currently holds an adjunct professorship in the Department of Pharmacology.

Hildegund CJ Ertl

Independent Consultant (Casablanca, Morocco)

Ertl joined The Wistar Institute (PA, USA) as an associate professor in 1987. She earned her medical degree from the University of Göttingen (Germany). Her scientific journey began during medical school with research training at the Max Planck Institute of Experimental Medicine (Göttingen, Germany). Following research fellowships at the Australian National University (Canberra, Australia) and the University of Minnesota (MN, USA), she joined the faculty at Harvard University (MA, USA). In 1996, Ertl was promoted to full professor at Wistar and also holds professorships at the University of Pennsylvania School of Medicine and The Children’s Hospital of Philadelphia (both PA, USA). Ertl’s research in the Ertl Laboratory at Wistar has developed innovative vaccine technologies, focusing on a preventative rabies vaccine while leveraging their expertise with adenovirus-based platforms to create vaccines for SARS-CoV-2, HIV, HPV, and hepatitis B.