Non-viral gene delivery technology for clinical application

This project focuses on developing an innovative technology for non-viral gene delivery based on the targeted delivery of therapeutic genes into diseased cells. The underlying problem is the inefficient and unsafe delivery of genetic material, resulting from the current reliance on viral vectors and the associated safety concerns. In particular, insufficient endosomal escape efficiency poses a significant challenge, as the majority of the genetic material is degraded in lysosomal compartments, limiting clinical application. Current non-viral gene delivery techniques are often expensive and offer limited efficacy, hindering implementation in large patient populations.

The main goal of the project is to develop an effective, safe, and cost-efficient system for targeted gene delivery. To achieve this, a polymeric scaffold is being designed that combines endosomal escape enhancers (EEEs) with therapeutic genes. The work includes the synthesis and optimization of carrier systems as well as extensive biological testing. The expected project outcomes include a novel platform for targeted gene delivery, suitable for both the treatment of monogenic diseases and cancer therapies. The solution offers significant added value by reducing the cost of gene therapies while increasing their efficacy. The project’s results will benefit not only patients, but also the medical community and the biopharmaceutical industry, which will gain access to improved therapeutic options.

The role of Fraunhofer IME within the ENDOSCAPE project encompasses several key aspects:

1. Development of production processes: Fraunhofer IME is responsible for developing stable and scalable production methods for EEEs using plant-based or hairy root systems.

2. Optimization of cultivation and production conditions: IME will optimize growth and production parameters to maximize yield and product quality.

3. Biotechnological support: IME will provide biotechnological development support to ensure that EEE production complies with GMP standards.

4. Collaboration with partners: The institute will work closely with other project partners to ensure a seamless end-to-end production process, from plant cultivation to extraction and purification of EEEs.