Driving RNAi Therapies Forward for Patients Vision Strategy Build a fully integrated company by • Build Value in rare diseases by independently developing and commercializing programs developing innovative RNAi-based with high value and high probability of success therapies for high unmet medical needs and broadly capture the value of • Capture Value in common diseases by our GalXC™ platform developing high probability of success programs through clinical proof of concept before seeking development and commercialization partners • Enhance Value by partnering with therapeutic field leaders on discovery stage programs 3
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