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AD/PD 2026 | Delivery systems under investigation for gene therapy in AD and PD

Boris Kantor, PhD, Florida State University, Tallahassee, FL, shares insights into the delivery systems being explored for gene therapy in Alzheimer’s disease (AD) and Parkinson’s disease (PD). As well as the adeno-associated vector, lentiviral and non-viral delivery systems are being developed for central nervous system delivery of gene therapy products. This interview took place at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases in Copenhagen, Denmark.

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Transcript

Well, so the other products, again, the major caveat is a delivery system. So, people utilize other delivery systems besides the one I mentioned earlier, adeno-associated vectors, such as lentiviral vectors, and non-viral delivery systems such as nanoparticles, gold particles. The largest one has been proven very efficient in so-called ex vivo delivery. When you take cells out of the body, you manipulate the cells, you apply CRISPR-Cas, and then you put those cells back to the host, to the human...

Well, so the other products, again, the major caveat is a delivery system. So, people utilize other delivery systems besides the one I mentioned earlier, adeno-associated vectors, such as lentiviral vectors, and non-viral delivery systems such as nanoparticles, gold particles. The largest one has been proven very efficient in so-called ex vivo delivery. When you take cells out of the body, you manipulate the cells, you apply CRISPR-Cas, and then you put those cells back to the host, to the human. So, again, it’s not very feasible for CNS delivery. It’s more this technology based, again, on nanoparticle, gold particle, lipid compositions are quite efficient for, let’s say, blood disorders, sickle cell anemia, thalassemia. By the way, so far, the first CRISPR-Cas product called Casgevy has been approved by the FDA last year, and that technology based on nanoparticles used to deliver, to manipulate genes related to blood disorder ex vivo and then transplant this product back to the human. So that’s tremendous advance of the CRISPR system. However, again, for CNS, it’s way more challenging. So other systems besides AAV, such as lentiviral vectors, are very popular and really become very safe vehicles for therapeutic application and some other methods but again the AAV, adeno-associated vector, still remains to be the number one platform for CNS delivery and many aspects of this delivery we address here in this interview, this presentation, people working on that field to optimize, to enhance, to improve the system, specifically to cross the blood-brain barrier and to create a system which would be efficient for delivery of bulky CRISPR-Cas tools. So that’s all work in progress, but we’re making very good progress on that.

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