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Preliminary OPTIMIZE IN VIVO GENE THERAPY WHAT IS IN VIVO GENE THERAPY AND HOW DOES IT DIFFER FROM EX VIVO GENE THERAPY? We aim to transform the approach to an in vivo method for editing blood and immune Ex vivo gene therapy can now treat sickle cell disease, but the approach requires (i) expensive manufacturing facilities and (ii) sophisticated clinical infrastructure KEY QUESTION(S) What combination of gene editing technologies and nucleic acid delivery vehicles will optimally reach blood and immune cells and their stem cell progenitors? cells -- using emerging technologies associated with CRISPR and mRNA vaccines -- to create a safe and durable "single-shot" therapy Understand how to safely and durably edit hematopoietic stem cells in vivo WHAT POTENTIAL RELATIONSHIPS COULD ACCELERATE THIS WORK? Intezzia ¢ &® G2] th Novartis @ caemurcn (G) EMORY UNIVERSITY T ORS D Fiospital Unreveneeat I @ And more, including new POSSIBLE APPLICATIONS companies Single-shot cure for diseases like HIV va (infectious) and sickle cell disease (inherited) GreenLight BIOSCIENCESTM Gf Source: Kaufmann, et al., EMBO Mol Med, 2013. Not for distribution\7