The market for gene therapy is surging, with orphan diseases a notable hotspot. A key reason for all the excitement? The potential for cure from a single dose. At the same time, many therapies in development face challenges like side effects and unproven efficacy. Beyond that, says L.E.K.’s Ricardo Brau, companies will need innovative business models for engaging with payers, regulators and other agencies. Hear Ricardo’s synopsis of where we stand in the ongoing effort to make gene therapies work for everyone.