Originally Aired: March 31, 2021
Time: 8:00 am PT, 11:00 am ET, 17:00 CET
Summing up the ground-breaking work that researchers in the cell and gene therapy space are producing is easy if one follows the notion that “the most reliable way to predict the future is to create it.” In recent years, investigators have worked tirelessly to solve many of the fundamental challenges associated with these therapeutic modalities. Yet, innovation and success are an evolving process that address challenges during the drug discovery and development stages.
In this GEN webinar, our distinguished presenters will provide perspectives on how the gene therapy field ought to evolve. From the early vector systems, which often represented replicating viruses, to the recent sophisticated engineering of targeted virus driven cell and gene therapies that have demonstrated clinical benefits and gained FDA approval for multiple diseases. Moreover, we will also hear how reliance on pre-clinical murine models to predict the clinical efficacy of AAV serotype vectors in humans is not adequate. In addition, an example of the utility for one specific AAV serotype vector, not efficacious in murine models will be discussed, leading up to a Phase I/II clinical trial in humans.
A live Q&A session followed the presentations, offering you a chance to pose questions to our expert panelists.
Produced with support from: