Rarecast

Emerging cell and gene therapies represent areas of great promise for people with rare, genetic diseases, but the translation gap for these medicines can leave them stalled at the lab. Mass General Brigham in 2022 launched the Gene and Cell Therapy Institute, a research hub dedicated to advancing gene and cell therapies for various diseases to bridge the divide between academic labs and clinical development of therapies. The institute brings together more than 500 researchers and clinicians and boasts unique assets, such as its RNA Therapeutics Core, which enable it to produce cutting-edge circular RNA on-site. We spoke to Nathan Yozwiak, head of research at the Mass General Brigham Gene andCell Therapy Institute, about the need it is seeking to address, how the institute operates, and what it might do to enable the development of bespoke therapies for ultra-rare diseases.