Project Background
Why Nano-Engine?
The use of genetically modified cells or direct in vivo gene editing— known as cell and gene therapy— to treat diseases has grown in recent years.
Currently, T cells are genetically modified outside the body (ex vivo) using viral vectors or electroporation in order to generate CAR-T therapy, and then injected into the patient. CAR-T therapy refers to T cells that are modified to express the CAR receptor, which enables them locate and eliminate cancer cells.
Existing gene therapies, like CAR-T cell therapy, face limitations that restrict their safe and widespread use in patients. These include high costs and complex procedures that need to be performed in highly specialised hospitals; the need for hospitalization due to preceding chemo-conditioning and the monitoring for severe side effects; limited accessibility due to a relatively small number of such specialized hospital beds; and difficulties in delivery of genetic material to specific cells of interest upon intravenous infusion.
During the project, the consortium will assemble, characterize, and test Targeted Nanoparticles that generate CAR-T cells in vivo, explore small-scale synthesis, conduct proof-of-concept studies and establish a basis for future commercialization.
The resulting Targeted Nanoparticles will address core issues limiting the use of such therapies by reducing cost, complexity and clinical risks – thereby greatly increasing accessibility of cell therapies to patients worldwide.
About Nano-Engine
NanoEngine is a EU-funded project aiming to establish proof-of-concept for a first-in-class and best-in-class in vivo cell engineering platform based on the targeted nano-delivery of a transposon gene editing system.
During the next 3 years (2023-2026), the project will demonstrate preliminary safety and efficacy of a Targeted Nanoparticle technology-based therapy for CD19+ B cell malignancies.
The cell-directed nanoparticle technology being deployed is designed to deliver DNA plasmids to specific cells of interest, such as T cells without the use of viral vectors. Because DNA stably integrates into the target cell’s genome, the therapies are designed to provide durable efficacy, thereby seeking to overcome limitations of RNA-based therapies. This new class of medicines has the potential to transform the fields of CAR- and TCR therapy.
Nano-Engine Technology
The Targeted Nanoparticle technology is based on three elements:
- A proprietary functionalized nanoparticle
Target nanodelivery will use novel NanoParticles (NPs) as carriers. The NPs are coated with a specific binder that recognizes and interacts with the T cell receptors, increasing their affinity and ability to enter the T cells more efficiently.
- A proprietary non-viral gene editing system
The non-viral gene editing system is based on a clinically proven method for genetic modification. It allows for the stable expression of DNA, ensuring long-term gene expression.
- A proprietary DNA technology
The system also uses “Recombination Based Plasmid Separation MiniCircle DNA” – a proprietary DNA plasmid technology that is devoid of bacterial components and highly scalable in terms of manufacturing. It can accommodate large genetic inserts and provides a durable therapeutic effect.
