PJ Brooks PhD, program director in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) in the USA, highlights the importance of going beyond one disease at a time in clinical trials, some of the roadblocks that still remain and why gene therapy and gene editing have significant potential for the rare disease community.
My main focus is on accelerating clinical trials in rare disease by moving beyond “one disease at a time” approaches.
