CAR-T Meeting 2025 | In vivo genome editing methods for targeting T-cells: the future of CAR-T?
In this interview, William Nyberg, PhD, Karolinska Institute, Solna, Sweden, discusses in vivo genome editing methods, highlighting their potential to improve accessibility to CAR T-cell therapy by reducing costs, lengthy manufacturing times, and the need for visits to a specialized center. Dr Nyberg mentions some challenges with ensuring this approach is safe, which must be addressed prior to widespread testing of this strategy in patients. This interview took place at the EHA-EBMT 7th European CAR T-cell Meeting, held in Strasbourg, France.
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Transcript (AI-generated)
At this conference I gave a talk about in vivo-based genome editing methods. So we’re trying to develop methods where we can deliver the Cas nuclease for genome editing and templates for homology-directed repair using evolved viruses and various particles. And this we’re trying to do to be able to generate T-cell therapies directly in vivo in patients.
I believe and we believe in the field that this could have a huge impact on the kind of spread and availability of CAR T-cells...
At this conference I gave a talk about in vivo-based genome editing methods. So we’re trying to develop methods where we can deliver the Cas nuclease for genome editing and templates for homology-directed repair using evolved viruses and various particles. And this we’re trying to do to be able to generate T-cell therapies directly in vivo in patients.
I believe and we believe in the field that this could have a huge impact on the kind of spread and availability of CAR T-cells. Not having to go to a specialized clinic where they can isolate your cells and engineer them and sort of produce these CAR T-cell therapies. And instead, we would be able to potentially ship a small vial of virus or particles around the world. And these can be produced so that they’re stable at room temperature, which means that they could essentially be sent everywhere or anywhere and infused directly into patients. And so we believe that this, although I don’t think that this will improve CAR T-cell therapy, I think it will improve the accessibility of the therapies. So really being able to reach more patients than what we can do today. So I think it can significantly reduce costs and significantly improve availability of the therapies.
There are definitely challenges and so I think the biggest challenges are finding good and efficient and safe delivery methods. And I think anything that, when it comes to gene therapies or anything that’s in vivo-based when we’re injecting things into patients, we need to make sure that the gene edits are where you think they are, there’s no off-target effects. We need to think about what cells do our vectors target and all of those things. And that’s going to be a massive challenge for the field moving forward.
The kind of methods that I’m working on is currently only in pre-clinical methods, but there are so-called engineered lentiviruses that have actually been injected into patients as we speak. So I think there are a couple of patients around the world that have gotten injected with these in vivo CAR T-cell therapies, so it’s going to be really exciting to see what comes out of those kind of first initial clinical trials.
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