The use of gene therapy offers significant promise to treat, and even cure, disease. Viral vectors—primarily AAV—have become the most common method of delivering a gene therapy, and while there are many benefits to using these vectors, one major challenge is pre-existing immunity resulting from natural exposure to AAV.
Developing an assay to assess humoral immunogenicity has thus become a crucial step in the clinical development of gene therapies. With 2 primary approaches to assess immunogenicity, a neutralizing antibody assay (NAb) or a total antibody assay (TAb), a common question asked by gene therapy developers is “which assay is best?”
This webinar explores the differences and advantages of each assay type, discusses how assay type selection may impact overall regulatory strategy, and provides guidance on best practices for assay development.
Key points covered:
- Importance of immunogenicity assays in gene therapy development
- Key differences between NAb and TAb assays and considerations in selecting an assay type
- Regulatory insights surrounding gene therapy immunogenicity evaluations
- Important design considerations for creating immunogenicity assays
About the speakers:
Deb Phippard, PhD
Chief Scientific Officer, Precision for Medicine
Deb Phippard is a pharma industry veteran and expert at biomarker-driven clinical trial design and execution with more than 25 years of biotechnology and clinical translational research experience. She is a leader of biomarker and drug development programs for pharmaceutical and diagnostics companies, as well as the National Institutes of Health. She spearheaded the discovery of pharmacodynamic biomarkers and novel targets for inflammatory disease therapy. Deb gained her PhD in Developmental Biology from the Institute of Cancer Research, London.
Travis Harrison, PhD
Vice President, Bioassay Solutions, Precision for Medicine
Travis Harrison is an immunologist with 20 years’ experience in drug development including the design, validation, and management of immunoassays, cell-based assays, and diagnostics with an emphasis on immunogenicity and gene therapy. He has successfully developed and validated GLP and cGMP assays required for IND submission of more than 3 dozen vaccines, gene therapies, and protein therapeutics. At Precision for Medicine, he has managed development of companion diagnostics with an emphasis on gene therapies. Travis gained his PhD in Immunology from the Virginia Commonwealth University School of Medicine.