The success or failure of Dicerna Pharmaceuticals (Dicerna) as an emerging pharmaceutical company would likely hinge on its lead drug candidate Nedosiran and the company's ability to see it successfully through clinical development. Ralf Rosskamp, Chief Medical Officer, had an ambitious clinical development plan that leveraged novel technology and a dynamic regulatory strategy to rapidly accelerate Nedosiran's development as a treatment for a rare disease. The case elaborates on Rosskamp's plan to design, operationalize and execute the clinical development plan, with a limited staff and an aggressive time line.