Insight

“One-and-done” gene therapies

A near-term niche?

Alasdair Milton, PhD

Alasdair Milton, PhD

Managing Director, Healthcare and Life Sciences Strategy, KPMG US

+1 617-988-5419

Kristin C. Pothier

Kristin C. Pothier

Global and National Healthcare and Life Sciences Strategy Leader, and Global Deal Advisory Leader, Healthcare and Life Sciences, KPMG LLP

+1 617 549 2779

Jeff Scafaria

Jeff Scafaria

Director Advisory, Strategy, KPMG US

+1 213-430-2174

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?


Gene therapy video series


"Join me as I interview a number of key stakeholders including patients groups, supply chain experts and those involved in the development, manufacturing and launch of these innovative technologies as they provide their
point of view on the promise and challenges related to gene therapy." - Alasdair Milton, PhD.