Roaring 20s Redux: The Next Decade of Gene Therapy Innovation

Over the last five years, multiple gene therapies have been approved by regulatory agencies and a bolus of late-stage pipeline assets are approaching the market. As the first few gene therapies realize their potential as transformative treatments for the genetic rare diseases, the space is seen as a crucial part of growth for the biopharmaceutical industry. However, as the first gene therapies began to post early wins, several technical and commercial challenges emerged.

With these challenges in mind, leading companies have begun to search for new delivery technologies and commercial models. Only after addressing these roadblocks will gene therapies be able to fully deliver on their longstanding potential to transform patient outcomes, expand into larger diseases, and ultimately provide returns for investors.