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eChalk Talk: Assays Supporting Gene Therapy: Biodistribition and Immunology

Description

To gain a better understanding of or to confirm design of how a gene therapy agent will travel throughout the body to the on and off target organs, a biodistribution study should be conducted. Additionally, this study will inform on the persistence of the gene therapy and to an extent the potential for delayed adverse events. PCR is the tool of choice for determination of viral vector biodistribution and transgene expression. There are a number of critical factors that will be discussed herein that go into developing and validating a suitable qPCR assay for biodistribution and an appropriate RT-qPCR assay to evaluate transgene expression.

Additionally, we will briefly explore the impact of the immune response on a gene therapy agent and how it can impact study design and endpoints. Considerations include screening for neutralizing antibodies, using immune suppression protocols and evaluating immunogenicity. The talk will focus in more detail on the utility of the ELISpot assay to monitor for cellular mediated immune activity in response to the gene therapy vector/transgene.

Contributors

  • Edward (Ted) Barrett, Ph.D.

    Ted Barrett, Ph.D., is Senior Director, Pharmacology at Lovelace Biomedical. Dr. Barrett has spent most of his career investigating the effects of small molecule, biologic, nucleic acid and medical device technologies in various animal models of disease. His programs aim to translate nonclinical efficacy, pharmacokinetic, and pharmacodynamic data obtained by from species ranging from rodents to nonhuman primates to the human clinical paradigm. Dr. Barrett received his doctorate in toxicology from the University of Rochester and joined Lovelace in 1999.

  • Brian Gullick, Ph.D.

    Brian Gullick is the Associate Director of Bioanalytical Operations at Lovelace Biomedical.  Dr. Gullick is a scientific department leader with over 15 years of GLP and GMP regulated experience in Quality Control, Process Development, Bioanalysis, and Pharmacokinetic applications. Bryan earned a BS in Chemistry and a minor in Biology from Erskine College, and went on to achieve his Ph.D. in Chemistry from the University of Alabama.

May 5, 2021
Wed 11:15 AM EDT

Duration 0H 30M

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