Roger Hajjar, MD, on Helping to Bring Preclinical Gene Therapy Research to First-In-Human Trials

“That's really been the mission and the differentiator for the Institute: to really bring together a set of platforms to help our investigators get to first-in-human [trials] because overall, the mission of the academic medical center that we're in is to help patients and to bring novel therapies to them.”

Advanced modalities like gene therapy and cell therapy have attracted an enormous flurry of nonclinical and preclinical research activity in recent years. Unfortunately, because of the high costs and complex nature of these modalities, among other factors, only a fraction of the wealth of preclinical work being done is actively translated into in-human clinical research. This means that a substantial amount of innovative work that has great potential to help patients may never reach them.

Mass General Brigham (MGB)’s Gene and Cell Therapy Institute (GCTI) was established a few years ago with the intent of helping some of the innovative preclinical research in the field make the leap to clinical studies. Notably, GCTI brought several presentations related to its efforts to the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, LA.

At the conference, CGTLive® sat down with Roger Hajjar, MD, the director of the GCTI, to discuss the unique role of the institute and some of the highlights from what it brought to ASGCT this year. Hajjar noted that to help achieve its goals, GCTI has built a support platform that offers manufacturing resources, regulatory guidance, trial design assistance, and funding strategies. Furthermore, the Institute's unique focus is on translating diverse early-stage research—spanning areas like neurology and cardiovascular science—into clinical applications by addressing the complex challenges of manufacturing, regulatory compliance, and trial design specific to gene and cell therapies. Hajjar mentioned a poster GCTI brought to the conference this year covering that topic.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.