Overcoming the Challenges in Bioanalysis of Oligonucleotide-based Therapies and Relevance to the Clinical Development Plan. “Gene therapy provides the hope of actual cures. These therapies still have many challenges to overcome before they will become widely developed therapeutic options.” This interview features Frontage's Chief Business Officer, Hugh Davis, and Vice President of Biologics Services, Weiping Shao.
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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 Newly released FDA draft guidance for Industry on human gene therapy products will also be discussed.
Gene therapy has become a one-time treatment method for a complete cure by fixing genomic errors which altered the protein functions of normal cells. The high titer virus packaging and purity are essential for the success of clinical gene therapy.
In this presentation, we report to establish a facility to manufacture GLP-grade viruses and to develop the relevant QC assays at Frontage. Our efforts will facilitate the application of this modern cutting-edge technology in the clinical arena.
Speaker: Dr. Rejean Wang, Gene therapy group leader of CMC, Frontage Laboratories, Inc.
Dr. Wang is a gene therapy group leader of CMC at Frontage Laboratories, Inc. He has extensive experience in the gene therapy field, including 16 years at the University of Florida and 3 years in the vaccine center of NCI, NIH as a post-doctor and an assistant scientist. He made important contributions to the development of rare disease mice models, such as Alph-1 antitrypsin deficiency model for gene therapy purposes. He also contributed to the development of AAV capsid mutants for lung targeting infection and adenovirus-mediated HIV vaccine. He has published 18 peer-reviewed scientific papers.
In this infographic, we talk about key trends in cellular and gene therapies.