Clinical Strategy and Development of CRISPR-Directed Gene Editing for Solid Tumors

The breakthrough gene editing technology, CRISPR/Cas, has moved rapidly from molecular analyses to clinical development and application, potentiating the well-known bench to bedside paradigm of biomolecular therapeutic development. Our laboratory has been advancing a CRISPR-directed gene editing platform technology as an augmentative therapeutic strategy for squamous cell carcinoma of the lung with follow-on applications to esophageal and head and neck cancer. In this talk, I will describe our rationale for developing this approach, what we learned from our failures in process including how we rerouted our scientific direction. I will also talk about how our choice of target (s) was influenced by unmet medical needs, by availability of an appropriate delivery systems and by input from frontline clinicians involved in cancer clinical trials. I will also describe our success through cell based and animal models and the challenges that we still face, both scientific and practical as we advance this protocol through FDA review panels. The talk will essentially be a case study describing the journey from bench to bedside of a remarkable novel technology that might eventually become a mainstay in the treatment of solid tumors.