Descrizione del progetto
L’editing del genoma per affrontare le malattie immunitarie
Si registra un aumento senza precedenti delle malattie croniche dovute a squilibri immunitari, per le quali le opzioni terapeutiche sono limitate. Il progetto geneTIGA, finanziato dall’UE, sta sviluppando una terapia cellulare sicura ed efficace basata su cellule T modificate a livello genomico per combattere in modo sostenibile la nefropatia da IgA (o malattia di Berger), una delle cause più comuni di malattia renale allo stadio terminale. Essendo una malattia da IgA, questo approccio di terapia cellulare può costituire il modello per altre malattie simili, fra cui il mieloma secernente IgA, la celiachia a produzione di antitransglutaminasi IgA e l’artrite reumatoide, oltre ad altre malattie Ig-correlate. I ricercatori di geneTIGA prevedono dunque la somministrazione di un «farmaco vivente» per il trattamento della malattia di Berger e di altre malattie associate alle IgA, per poi passare alla prima sperimentazione clinica sull’uomo.
Obiettivo
"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."
Campo scientifico
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesbasic medicineimmunologyautoimmune diseases
- medical and health sciencesmedical biotechnologycells technologies
- medical and health sciencesclinical medicinenephrologykidney diseases
- medical and health sciencesclinical medicinegastroenterology
Parole chiave
Programma(i)
Meccanismo di finanziamento
HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinatore
10117 Berlin
Germania