Therapeutic Focus

Our ther­a­peu­tic indi­ca­tions were cho­sen based on our con­vic­tion in the under­ly­ing biol­o­gy, exis­tence of val­i­dat­ing pre­clin­i­cal and clin­i­cal data, avail­abil­i­ty of phar­ma­co­dy­nam­ic and trans­la­tion­al tools to assess ear­ly proof-of-con­­cept, rel­e­vant val­ue sup­port­ing out­come mea­sures, and, most impor­tant­ly, ongo­ing clin­i­cal unmet need. These pro­grams are select­ed to both address dev­as­tat­ing dis­eases and to estab­lish new stan­dards in tar­getabil­i­ty, pre­ci­sion, effi­cien­cy, and scope of edit­ing capa­bil­i­ties. Using our tool­box, we aim to match the opti­mal genome edit­ing tool to each indi­ca­tion to ensure last­ing effects on the lives of patients.