In this webinar, Dr. Philip Tiller reviews Metabolites in Safety Testing (MIST) and discusses the scientific foundation of our analytical approach. Finally Dr. Tiller shares a case study of our success.
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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 Newly released FDA draft guidance for Industry on human gene therapy products will also be discussed.
Biologics are complex large molecules derived from living organisms or manufactured from cells, and they have inherent structural heterogeneity. Indeed, proteins, given their chemistry, are the “most structurally complex and functionally sophisticated molecules known.” Thus, the analytical testing required to support Chemical, Manufacturing, and Controls (CMC) activities of biologics is highly sophisticated and is becoming more so as the modalities of biologics grow more complex (with antibody-drug conjugates, conjugate vaccines, PEGylated proteins, fusion proteins, gene therapies, and cell-based therapies).
Ensure comprehensive product analysis with Frontage’s team of experienced analytical scientists. We specialize in analytical method development, validation and transfer for product development and clinical trial materials (CTM) manufacturing support, as well as commercial product release and stability testing. Our services are designed to help sponsors throughout the drug development process in their effort to fully characterize drug substances, developmental formulations and commercial drug products.