Commentary|Videos|September 8, 2025
Robert Alexander Wesselhoeft, PhD, on Broader Trends in the RNA Therapeutic Research Landscape
The director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute expressed optimism for RNA’s potential role in CAR-T therapy, gene editing, and more.
Advertisement
This is the second part of an interview with Robert Alexander Wesselhoeft, PhD. For the first part, click here.
“I'm very optimistic about the development of coding RNA therapeutics and nonviral delivery technology in general. I think especially even in the last few months, we've seen a lot of people moving away from classical gene therapy vectors like AAV for a variety of different reasons. It seems like nonviral gene transfer and gene expression, especially through RNA, is becoming a preferred modality where it's applicable because of its enhanced safety.”
Historically, the production of chimeric antigen receptor T-cell (CAR-T) therapy has relied upon the use of lentiviral vectors to transduce T-cells outside the body. A number of companies and academic institutions are now exploring the potential of in vivo CAR-T therapy production, however, which could potentially be carried out without the use of a lentiviral vector by using RNA instead.
Meanwhile, while in vivo gene therapy for rare diseases has thus far primarily relied upon the use of adeno-associated virus (AAV) vectors, recent research has sought to apply the use of RNA to gene editing for rare diseases. This could enable a number of advantages, such as avoiding the potential immunogenic issues associated with AAV vectors.
The aforementioned cases are examples of how RNA-based approaches may potentially transform the cell and gene therapy landscape in the coming years. Notably, Robert Alexander Wesselhoeft, PhD, the director of RNA Therapeutics at the Gene and Cell Therapy Institute (GCTI) at Mass General Brigham (MGB), discussed this topic in an interview with CGTLive® at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting , held May 13 to 17, 2025.
Following up his discussion of his own work at the GCTI on potential therapeutic applications for circular RNA, Wesselhoeft shared his thoughts on the burgeoning field of RNA therapy in general. In particular, he drew attention to the potential of RNA to enable in vivo CAR-T therapy, which he pointed out may yield a major shift in the CAR-T space in the coming years.
Newsletter
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
Advertisement
Related Articles
Annaiz Grimm, BS, on Using EngTregs to Treat Autoimmune DiseaseAugust 28th 2025
Advertisement
Advertisement
