Periodic Reporting for period 1 - NANO-ENGINE (A revolutionary cell programming platform based on the targeted nano-deliveryof a transposon gene editing system)
Reporting period: 2023-04-01 to 2024-03-31
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.
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.
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.
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.