Description du projet
L’édition du génome pour lutter contre les maladies immunitaires
Les maladies chroniques dues à un déséquilibre immunitaire, pour lesquelles les options thérapeutiques sont limitées, connaissent une augmentation sans précédent. Le projet geneTIGA, financé par l’UE, s’emploie à développer une thérapie cellulaire sûre et efficace fondée sur des lymphocytes T dont le génome a été modifié pour lutter durablement contre la néphropathie à immunoglobulines A (IgA), l’une des causes les plus courantes de néphropathie en phase terminale. En tant que maladie à IgA, cette approche de thérapie cellulaire peut constituer un modèle pour d’autres maladies à IgA, notamment le myélome à IgA, la maladie cœliaque liée à l’IgA et l’arthrite rhumatoïde, ainsi que d'autres maladies à Ig. Les chercheurs de geneTIGA prévoient ainsi de créer un «médicament vivant» destiné au traitement de la néphropathie à IgA et d’autres maladies associées à l’IgA, qui fera l’objet des tout premiers essais cliniques sur l’homme.
Objectif
"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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HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinateur
10117 Berlin
Allemagne