Goldman Sachs 45th Annual Global Healthcare Conference Optimized End-to-End in Genetic Medicine Future focus on in vivo delivery of biologic therapeutics for large indications and unmet needs Diverse Clinical Pipeline 3 late stage clinical programs pivotal/Phase 3 For prevalent non-inherited indications End-to-end GMP manufacturing Flexible and Scalable Next Generation Vector Optimization Potency, safety, dose, CofG Transformative RiboswitchTechnology In vivo delivery via oral small molecule • Retinitis Pig ...