“One-and-done” gene therapies

The last decade has brought forth a revolution in the pharmaceutical industry with the launch of the first “one-and-done,” or potentially curative, gene therapies. From the launch of Luxturna for an inherited eye disease, to the entry of Zolgensma for spinal muscular atrophy (SMA), we are now in an era of innovation in which certain diseases can potentially be cured. However, for the time being we believe the focus will likely remain on rare and ultra-rare monogenic diseases until the field evolves and the major challenges of therapeutic delivery and manufacturing are addressed.

If this is the hand that biopharma companies have been dealt, how should they play it? What does this mean for short- and long-term business models? Should biopharma companies be spending big now in the hope that, in the long term, these therapies and their associated platforms will drive sustainable top- and bottom-line growth from more complex, larger-population diseases? In short, how can companies gain a competitive advantage in what will likely be a near-term niche while also developing a long-term presence in this innovative field?