“One-and-done” gene therapies

We are in a new era of scientific innovation where “one-and-done” gene therapies can potentially cure disease. However, headwinds still exist that will limit their use to rare and ultra-rare monogenic diseases for the next 5-10 years. In this paper, we seek to inform biopharma leaders of these ongoing issues and illustrate what they mean in terms of building a sustainable presence in this innovative field.