Helping to Bring Preclinical Gene Therapy Research to First-In-Human Trials

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Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, also discussed the Institute’s presentations at ASGCT’s 2025 conference.

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute

Roger Hajjar, MD

A few years ago, Mass General Brigham established its Gene and Cell Therapy Institute (GCTI) with the goal of helping innovative preclinical research in the field make its way into first-in-human clinical trials. At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, the GCTI gave several presentations related to its mission.

During the conference, CGTLive® spoke with Roger Hajjar, MD, the director of the GCTI, to learn more about the Institute and its activities. Hajjar described what makes the GCTI unique in the field and discussed some highlights from the Institute's presentations at ASGCT this year.

CGTLive: Could you give some background info about the GCTI? What makes it unique?

Roger Hajjar, MD: The GCTI at Mass General Brigham was initiated around 2 and a half to 3 years ago with the specific mission of bringing together over 500 investigators who focus on gene and cell therapies across Mass General and Brigham. These laboratories are focused very much on early stage and preclinical work, and our goal as an institute is to bring these projects that are early stage into first-in-human trials. To do that, we've taken the approach of creating a platform for these investigators to help with manufacturing, with regulatory interactions, with trial design, and also focusing on funding and different sources of funding to bring these projects into first-in-human trials. That's really been the focus of the Institute since its start.

We've also been able to create specific type of "cores" that help with these types of investigations. These include AAV cores, RNA therapeutics cores... other cores, such as cell therapy cores, for specific types of induced pluripotent stem cell-derived neuronal cells, for example—and to have a good manufacturing practice (GMP) facility within Mass General Brigham, to have more point-of-care type of treatments—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.

Is there anything GCTI is presenting at the conference this year that you would like to highlight?

Our investigators across MGB have different areas [of interest such as] in pediatric neurology, cardiovascular disease, etc.—the Institute has a focus on how to translate early stage investigators to clinical trials. We have a poster depicting that. This transition from early-stage to clinical trials in the gene and cell therapy space is quite unique because of all the different areas that need to be addressed for gene and cell therapies. How do you manufacture, since our investigators use different modalities and different delivery systems? How do you engage with the regulatory entities? How do you design a trial that's going to be able to effectively show biological effects for the treatment? That's what we've been focusing on and that's what's being presented here at the ASGCT meeting this year.

This transcript has been edited for clarity.

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

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