Target Audience: Investigators & TDC Director's

This session will discuss two new genetic therapies guidance documents for industry: 1) pre-clinical and 2) proof of concept and early trial outcomes. As more genetic therapies approach the clinic, we must partner with industry and identify the guiding principles for building a sufficient pre-clinical package as well as considerations for selecting study populations, clinical trial designs and selecting appropriate clinical outcome measures. Session leaders will facilitate a robust discussion on what we know and what gaps remain as we prepare and enroll these exciting new genetic therapy trials.