Imagine your immune system is like a security team that has started attacking your own kidneys by mistake. This project teaches a specific group of immune cells to act like precision guided missiles that find and stop the exact cause of this attack. Instead of using risky old methods to reprogram these cells, they use a new 'blueprint' that is safer, cheaper, and more accurate.
Current treatments for IgA nephropathy and related autoimmune diseases fail to restore long-term immune balance, leading to organ failure and high societal costs. Existing gene therapies are often too expensive or carry risks of causing cancer due to viral vectors.
A lead candidate 'living drug' for IgAN and a toolbox containing a genome-editing benchmarking platform and an off-target identification pipeline.
If you are a cell therapy developer dealing with high manufacturing costs and safety risks from viral vectors — this project developed a non-viral genome editing blueprint that reduces oncogenic hazards and lowers production costs.
If you are a clinic network dealing with the high burden of end-stage renal disease — this project developed a 'living drug' candidate for IgA nephropathy that aims to provide a one-time treatment instead of lifelong management.
If you are a service provider dealing with inconsistent off-target effects in T-cell engineering — this project developed a benchmarking platform and off-target identification pipeline to accelerate the development of next-generation gene products.
The project utilizes non-viral genome editing, which is described as a cost-effective avenue for cell therapy manufacture compared to complex retroviral vector processes.
Yes, the approach is designed as a blueprint for other B-cell pathologies, including IgA myeloma, IgA-lymphoma, rheumatoid arthritis, and diseases linked to other immunoglobulin types like IgG4.
Based on available project data, the project delivers enabling technology toolboxes with specific exploitation options beyond the core project to de-risk and accelerate gene product development.
The project is developing new standards for safety assessment and a pipeline for off-target identification to mitigate oncogenic hazards associated with gene transfer.
The project runs from 2022-07-01 to 2026-12-31, with the goal of having a lead candidate ready to enter clinical First-in-Human (FIH) trials by the end of the period.
The consortium shows a strong commercial orientation with a 33% industry ratio, comprising 4 industry partners (including 4 SMEs) and 7 universities across 8 countries. This balance suggests a clear path from academic research at Charité Berlin to industrial application and scale-up.
Contact Charité - Universitätsmedizin Berlin for licensing and collaboration inquiries.
Contact SciTransfer to access the full benchmarking data and off-target pipeline specifications.
