Targeting microRNAs for inhibition or replacement allows for the manipulation of biological pathways that are important in a range of disease states.As with any drug modality, it is vital to identify and validate the best target microRNA and drug pair that leads to the desired beneficial outcome in the disease state.By applying a combination of genetic and pharmacological agent validation we have been able to identify compelling microRNA targets in several diseases with high unmet medical need.We believe our strategy will allow us to advance multiple clinical candidates into innovative translational medicine studies allowing for rapid mechanistic proof of concept in humans.