In vivo CAR-T therapies dominated discussions at the American Society of Gene & Cell Therapy’s annual meeting in Boston this week, with developers showcasing a flurry of preclinical data. The approach, which aims to engineer immune cells directly inside the body, has drawn intense interest from researchers and investors alike.
The field of genetic medicine has not fully recovered from recent headwinds, making the ASGCT meeting a critical checkpoint for demonstrating progress. In vivo CAR-T represents a potential leap beyond traditional ex vivo methods, which are logistically complex and expensive. But the technology remains firmly in the lab.
Every presentation on in vivo CAR-T at the conference came from preclinical studies, with no clinical-stage data yet available. Developers are racing to solve key challenges, including targeting precision, safety, and manufacturing scalability. The sheer volume of early research signals high enthusiasm but also underscores the long road ahead.
If successful, in vivo CAR-T could dramatically expand patient access by eliminating the need to extract and engineer cells outside the body. However, skeptics warn that the preclinical results may not translate to humans, given the complexity of in vivo delivery. The next year or two will be telling as early clinical trials are expected to begin.
One leading researcher cautioned that the field must prioritize rigorous safety testing before rushing into the clinic, noting that historical failures in gene therapy highlight the high stakes involved.