Leadership Series: The Promise of Gene Therapy for Rare Disease


Eli Kirshbaum


State of Reform had the pleasure of speaking with Bert Bruce, the US president for rare disease at Pfizer, about gene therapy, its promise for treating genetic diseases, and the potential it has for lowering healthcare costs for long-term disease management.

Bruce discussed gene therapy’s life-saving capabilities, the challenges in developing them and rendering them accessible, and Pfizer’s ongoing commitment to making these therapies available for those in need.

“Here are Pfizer, we have only one mission, and that is to deliver breakthroughs that change patients’ lives. And the gene therapy portfolio, we really believe, fits well within that ethos.”

Pfizer currently has three late-stage (Phase 3) development programs investigating gene therapy in populations where there is a high unmet need: hemophilia B, hemophilia A, and Duchenne muscular dystrophy

“Our goal and our vision is that, as these medicines come to market, there’s an aligned vision with those who would be delivering it—that would be us as the manufacturer—and those who would be reimbursing for it, so that appropriate patients have access to the medicine as quickly as possible. And we’ve been able to work, as we understand the medicine, to develop pathways to ensure that they have access collectively.”

Watch the full conversation above!