A revolutionary cell programming platform based on the targeted nano-delivery of a transposon gene editing system

Projektinformationen

NANO-ENGINE

ID Finanzhilfevereinbarung: 101098944

DOI

10.3030/101098944

EK-Unterschriftsdatum 15 März 2023

Startdatum 1 April 2023

Enddatum 31 Juli 2026

Finanziert unter

• The European Innovation Council (EIC)

Gesamtkosten

€ 2 988 377,95

EU-Beitrag

€ 2 988 377,70

Koordiniert durch

UNIVERSITEIT UTRECHT
Netherlands

Periodic Reporting for period 1 - NANO-ENGINE (A revolutionary cell programming platform based on the targeted nano-deliveryof a transposon gene editing system)

Berichtszeitraum: 2023-04-01 bis 2024-03-31

Zusammenfassung vom Kontext und den Gesamtzielen des Projekts

Chimeric antigen receptor (CAR) T-cell therapy represents a groundbreaking advance in cancer treatment, fundamentally changing the approach to managing previously intractable forms of blood cancers, such as certain leukemias and lymphomas. This innovative therapy involves genetically modifying a patient's T cells ex vivo to express a CAR that targets specific antigens on tumor cells, enabling the immune system to better recognize and destroy cancer cells. The results have been transformative, particularly for patients who have exhausted other treatment options, showing remarkable success rates in clinical trials. However, CAR T-cell therapy is not without its downsides. The treatment is highly specialized and expensive, limiting access to a broader patient population. In addition, the T cells run the risk of premature exhaustion due to the intensive culturing schedule.
The overall aim of the NANO-ENGINE project is to establish PoC for the first in vivo cell engineering platform technology by demonstrating efficient in vivo transfection of T cells and preliminary efficacy in treatment of CD19+ B cell malignancies.

Arbeit, die ab Beginn des Projekts bis zum Ende des durch den Bericht erfassten Berichtszeitraums geleistet wurde, und die wichtigsten bis dahin erzielten Ergebnisse

We have successfully generated the four components of our targeted nanoparticle and have shown that the nanoparticle can be assembled selectively target T cells and transfect them. We have set up QC measures to evaluate the critical quality attributes of our targeted nanoparticle.

Fortschritte, die über den aktuellen Stand der Technik hinausgehen und voraussichtliche potenzielle Auswirkungen (einschließlich der bis dato erzielten sozioökonomischen Auswirkungen und weiter gefassten gesellschaftlichen Auswirkungen des Projekts)

Task 1.6 was performed ahead of schedule. To the best of our knowledge the presented transfection levels (60 -70%) are in a great excess of reported levels of non-viral DNA transfection of T cells in literature. These results need to be validated in vivo to ensure further uptake and success.