Until recent years, polycystic kidney disease (PKD) was a renal indication in search of solutions – a situation even truer for its autosomal dominant (ADPKD) and autosomal recessive (ARPKD) subgroups. That dearth of drug development in the space is beginning to change, however, thanks to greater understanding of the genetic drivers of kidney disease and of the molecular pathways that are subsequently up-regulated and cause disease.Those findings are enabling researchers to apply precision medicine approaches that are the hallmark of targeted cancer therapies to indications such as PKD. Buy-in from regulators, who have shown willingness to explore creative alternatives to historically broad trial designs, are beginning to attract both big pharma and newer entrants to the opportunities in renal disease.