Project description
Genome editing to tackle immune diseases
There is an unprecedented increase in chronic diseases due to immune imbalance for which there are limited treatment options. The EU-funded geneTIGA is developing a safe and efficient cell therapy based on genome-edited T cells to sustainably combat IgA nephropathy (IgAN), one of the most common causes of end-stage renal disease. As an IgA disease, this cell therapy approach can form the blueprint for other IgA diseases including IgA myeloma, IgA related coeliac disease and rheumatoid arthritis, as well as other Ig diseases. Thus, geneTIGA researchers anticipate the delivery of a ‘living drug’ to treat IgAN and other IgA-associated diseases for entry to first-in-human clinical trials.
Objective
"There is an increasing prevalence of chronic diseases caused by undesired immune reactions (>10%) with high burden for the both patients (chronicity, organ failure, early death, decreased QoL) and society (EU:>100 bn €/a direct health costs) as current therapies are limited in efficacy and do not reshape sustainably the disturbed immune balance.
Our ultimative goal is to develop a safe and efficient cell therapy based on genome-edited T cells with redirected specificity to sustainably combat IgA nephropathy (IgAN) - the most common glomerulonephritis and one of the most common causes of end-stage renal disease with unmet medical need. Our specific cell therapy approach is also suitable for other diseases with selective B-cell pathogenesis, such as IgA myeloma, IgA related celiac disease and rheumatoid arthritis, but as a blueprint also for diseases of other Ig classes (e.g. IgG4).
Our novel concept offers a specific form of immunosuppression via Ig-(sub)class targeting & glycosylation targeting with redirected T cells in autoimmune diseases. Methodically, we benchmark three promising genome editing technologies, develop new standards for safety assessment and preclinical performance evaluation.
At the end we will have a lead candidate of a new ""living drug"" product envisioned as a one-time treatment for IgAN and other IgA-associated that will be ready to enter clinical FIH trials (entry into TRL6). In addition, geneTIGA delivers enabling technology toolboxes with exploitation options beyond of the core project. They might de-risk and accelerate the development of next-generation gene and cell products in general. The project has thus, besides its scientific value, a high impact not only on the affected patients with IgAN and related immune diseases, but also for the European society by reducing the health economic burden caused by progressive chronic kidney disease, as well as by triggering innovation and business options in Europe's biotech and pharma field."
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Funding Scheme
HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinator
10117 Berlin
Germany