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Gene therapy: Bioanalytical support and strategies


About this Webinar

Gene and nucleic acid therapies have demonstrated patient benefit in specific areas of therapeutics to address unmet medical needs. An increasing number of gene- and nuclei acid-based products have been advanced in clinical development phases. Unlike other conventional drugs, a gene therapy medicinal product (GTMP) contains both vector and transgene. The pharmacokinetic behaviors of both GTMP and the expressed gene moieties are essential for understanding pharmacology and safety. In the case of virus-based gene therapies, pre-existing antibodies to the viral capsid due to prior infection/vaccination with related viruses may affect the safety and efficacy of the GTMP. Therefore, tests of the immune responses to the capsid as well as the transgene product need to be considered. Unlike most biotherapeutics, gene and nucleic acid products may have specific tissue/cell tropism and can enter cells. In addition, many products are administered directly into the diseased tissue/compartment; thus systemic exposure may not best describe the pharmacokinetics. Bioanalytical methods shall provide evidence to bridge systemic and tissue exposure and the method needs to be qualified or validated to ensure the data quality. All of those add complexity to the bioanalytical support for gene and nucleic acid therapies. This webinar will present the current practices in bioanalytical methods as well as pharmacokinetic /pharmacodynamical and immunogenicity evaluation for a number of gene and nucleic acid programs. The challenges, considerations, and regulatory aspects in this specific filed will be presented and discussed.

Learning Objective

  • Describe the current practices in method platform selection, method design and  optimization in support of Gene Therapy modalities
  • Provide an overview of method validation and relevant guidelines
  • Discuss challenges and opportunities in bioanalytical support of gene therapies


  • Robert Dodge, Ph.D.

    Rob Dodge has worked in the Pharmaceutical Industry for over 20 years in both CMC manufacturing and analytical release of Biologic therapeutics and regulated Bioanalytical testing of small molecules, protein drugs, biomarkers and novel molecular entities such as nucleic acids, cell therapy, and gene therapy products. Rob is the Director of Bioanalytical Sciences Development, leading a team of scientists who develop bioanalytical assays to support regulated bioanalysis for pharmacodynamics and immunogenicity assessments.  Rob is currently the Discovery Bioanalytical Global Lead at Novartis supporting assay development and bioanalytical testing using LC/MS/MS, LBA, qPCR, flow cytometry and cell based bioanalytical platforms for small molecules, biologics, nucleic acids and gene therapy therapeutics.

May 9, 2019
Thu 12:30 PM EDT

Duration 1H 30M

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