Over the past several years, gene therapies and cell-based immunotherapies for cancer have become a reality, with several products approved by US and EU regulatory agencies and numerous clinical trials ongoing. Gene-editing approaches leveraging programmable nucleases, such as CRISPR-Cas9, have invigorated work in this area, as evidenced by a bolus of high-impact papers and a flood of biotechnology companies seeking to bring edited therapeutics to the market. The potential of targeted gene editing to personalize stem-cell-based therapies for degenerative disease and regenerative medicine is likewise the subject of intense preclinical interest. This meeting will bring together academic and industry scientists and clinicians working on developing and implementing gene therapy and cell-based therapy for a wide variety of conditions.
Topics will include in vivo gene therapy with CRISPR and other platforms, ex vivo genetic engineering of immune cells and stem cells for translational applications, development of methods for delivering genes and editing machinery to desired target cells, and strategies to circumvent barriers such as host immune reactions against introduced vectors, proteins, and cells.