Rare Disease Day® (link is external) takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare diseases and their impact on patients’ lives. Each year, NCATS and the NIH Clinical Center sponsor Rare Disease Day at NIH as part of this global observance. Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH research collaborations underway to address scientific challenges and to advance new treatments.

Over the last several years, new genome engineering tools have revolutionized the ability to interrogate biological systems, engineer innovative biotechnologies, and model and treat human disease. The genome engineering field continues to evolve at a frenzied pace, including both the development of new fundamental tools and their application to addressing societal needs. This Keystone Symposium will cover this spectrum of advances, including the most recent technological innovations in genome, epigenome, and transcriptome precision engineering.