Monoclonal antibodies and next generation molecules such as antibody-drug
conjugates (ADC) are being developed and moved into early phase clinical testing.
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Industry Best Practices for their Detection and Characterization
Gene therapy holds the promise to transform medicine and creates options for patients who are living with incurable diseases. The industry has been booming in the last 20 years. Before a gene therapy product can be used in humans, the product must be tested for safety and effectiveness.
For in vivo genome modification, the most used gene delivery method is by Adeno-associated virus (AAV) vectors. For AAV-based gene therapy products, a full scale of testing methods have been established to ensure the identity (CE-SDS, LC-MS, Q-PCR/D-PCR), potency (A260/A280, HPLC, TCID50 and Infectivity assay), purity (Host cell DNA/Protein, residual plasmid DNA, Residual BSA and Benzonase by ELISAs, residual cesium, RCA, AUC for Empty : Full ratio), and safety (BioBurden, Endotoxin, Sterility, Mycoplasma, etc.).
For in vitro genome modification, Lenti viral vectors are used to genetically modify the patient’s own T cells to express a chimeric antigen receptor (CAR) designed to recognize and bind to a target antigen on tumor cells to eliminate the tumor cells. For Lenti viral gene therapy products, genomic titer and p24 ELISA are widely used for the calculation of viral titer. In addition to the mentioned assays, RCL, VSVg, and SV40 tests are often used as a safety test for Lenti viral products.
This webinar focuses on the introduction of three critical test methods: AUC, Infectivity and RCL assays. Newly released FDA draft guidance for Industry on human gene therapy products will also be discussed.
This webinar will provide a general overview of the regulatory guidelines required for developmental and reproductive toxicity (DART) testing. In order to initiate clinical trials for women of child-bearing potential (WOCBP), the FDA requires initiation of preclinical DART studies using mammalian research models. These regulated studies provide important information regarding the effects of drug exposures prior to and during parental mating, as well as evaluating maternal and fetal changes during gestation, and identifying any alterations in the development of the offspring following birth.