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

Informazioni relative al progetto

NANO-ENGINE

ID dell’accordo di sovvenzione: 101098944

DOI

10.3030/101098944

Data della firma CE 15 Marzo 2023

Data di avvio 1 Aprile 2023

Data di completamento 31 Luglio 2026

Finanziato da

• The European Innovation Council (EIC)

Costo totale

€ 2 988 377,95

Contributo UE

€ 2 988 377,70

Coordinato da

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)

Periodo di rendicontazione: 2023-04-01 al 2024-03-31

Sintesi del contesto e degli obiettivi generali del progetto

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.

Lavoro eseguito dall’inizio del progetto fino alla fine del periodo coperto dalla relazione e principali risultati finora ottenuti

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.

Progressi oltre lo stato dell’arte e potenziale impatto previsto (incluso l’impatto socioeconomico e le implicazioni sociali più ampie del progetto fino ad ora)

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.