Peter Marks, MD, PhD, Director of the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA), delivered a keynote address about the promise of gene therapy and what the FDA is doing to try to help realize that promise faster.

“My interactions with parents and family members of people with rare diseases have really helped me to realize that every day counts,” he said.

The FDA, which is responsible for ensuring the safety, efficacy, and security of medical therapies in the US market, recognizes that for many rare diseases, there is also a sense of urgency to find effective treatments. And gene therapy is the most promising path to effective therapies.

According to Dr. Marks, the FDA is implementing several strategies — from international collaboration to accelerated approval — to help researchers and pharmaceutical companies address the challenges of developing gene therapies for rare diseases and speed the review process. Among those strategies is a pilot project to apply methods used during Operation Warp Speed — the federal initiative that sped up the development of COVID-19 vaccines — to develop promising therapies.

“We’re very committed to advancing the time and availability of gene therapies for all sorts of disorders,” he concluded.