Roger Hajjar, MD, on Broader Trends in Cell and Gene Therapy

“One area that's been pervasive within CAR-T is whether you can deliver in vivo material that will directly infect T-cells and not have to go through an ex vivo CAR-T treatment. We've heard a lot of progress being made in that area so that's clearly exciting for the field.”

In recent years, cell and gene therapy products have become more and more of amainstay in clinical practice for a variety of conditions. With regard to gene therapy, adeno-associated virus (AAV) vector-based gene therapies have become an important treatment option for some monogenic rare diseases, such as Duchenne muscular dystrophy. As for cell therapy, chimeric antigen receptor T-cell (CAR-T) therapy has become a staple of treatment for some relapsed/refractory hematologic malignancies, and efforts are being made to expand the modality to solid tumors and even autoimmune disease.

Despite this progress, however, the field of cell and gene therapy still faces a number of challenges, such as the high cost of manufacturing the products and potential safety risks. Fortunately, a number of companies and academic institutions are now seeking to address such concerns with a myriad of innovative methods.

At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2025, in New Orleans, LA, CGTLive® spoke with Roger Hajjar, MD, the director of the Mass General Brigham’s Gene and Cell Therapy Institute, to get his insight on the broader trends within the cell and gene therapy field. Hajjar pointed out several of the ongoing challenges in the field, but also noted progress on several fronts. For gene therapy viral vectors, he highlighted that more organ-tropic vectors are emerging, along with novel vector families such as anelloviruses and boca viruses. With regard to cell therapy, Hajjar emphasized the importance of in vivo CAR-T production. He also touched on the recent progress for in vivo gene editing.

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