Projektbeschreibung
Genomeditierung zur Behandlung von Immunkrankheiten
Chronische Krankheiten, die auf ein Ungleichgewicht des Immunsystems zurückzuführen sind und für die es nur begrenzte Behandlungsmöglichkeiten gibt, nehmen in einem noch nie dagewesenen Maße zu. Das EU-finanzierte Projekt geneTIGA entwickelt eine sichere und effiziente Zelltherapie auf der Grundlage genomeditierter T-Zellen zur nachhaltigen Bekämpfung der IgA-Nephropathie (IgAN), einer der häufigsten Ursachen für Nierenerkrankungen im Endstadium. Da es sich um eine IgA-Erkrankung handelt, kann dieser zelltherapeutische Ansatz als Blaupause für andere IgA-Erkrankungen dienen, darunter das IgA-Myelom, die IgA-assoziierte Zöliakie und rheumatoide Arthritis sowie andere Ig-Erkrankungen. Folglich erwartet das Forschungsteam von geneTIGA die Entwicklung eines „lebenden Arzneimittels“ zur Behandlung von IgAN und anderen IgA-assoziierten Krankheiten, das in die ersten klinischen Versuche am Menschen einfließen soll.
Ziel
"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."
Wissenschaftliches Gebiet
- 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
Schlüsselbegriffe
Programm/Programme
Aufforderung zur Vorschlagseinreichung
Andere Projekte für diesen Aufruf anzeigenFinanzierungsplan
HORIZON-RIA - HORIZON Research and Innovation ActionsKoordinator
10117 Berlin
Deutschland