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OPTIMIZE IN VIVO GENE THERAPY @) What combination of gene editing technologies and nucleic acid delivery vehicles will optimally reach blood and immune cells and their stem cell progenitors? We aim to transform the approach to an in vivo method for editing blood and immune cells - using Ex vivo gene therapy can Ex vivo now treat sickle cell disease, = but the approach requires * expensive manufacturing facilities emerging technologies + sophisticated clinical associated with CRISPR and infrastructure mRNA vaccines - to create a safe and durable "single-shot" therapy Cell culture ro Isolation OF of HSCs °° ®"Gene therapy vector o Understand how to safely and durably edit hematopoietic stem cells 7 vivo Ex vivo expansion Gene transfer Reinfusion Single-shot cure for diseases like HIV (infectious) and sickle cell disease (inherited) PARTNERS: gw Intetia Greenlight 1, NOVARTIS INCENTIVES: Regulatory pathways for approval of in vivo gene CRITICAL PARTNERS therapies, orphan drug status equivalency for populations in low- and ffs FRED HUTCH (&) EMORY WwW eins § FEBROAD middle-income countries, costing infrastructures (to be developed) een INSTITUTE ( if Source: Kaufmann, et al., (MBO Mol Med, 2013. Not for distribution