Descripción del proyecto
La modificación del genoma para abordar enfermedades inmunitarias
Las enfermedades crónicas están aumentado como nunca antes debido al desequilibrio inmunitario, y sus opciones de tratamiento son limitadas. El equipo del proyecto geneTIGA, financiado con fondos europeos, está desarrollando una terapia celular segura y eficaz basada en linfocitos T genomodificados para combatir de forma sostenible la nefropatía por immunoglobulinas A (NIgA), una de las causas más comunes de la insuficiencia renal terminal. Este método de terapia celular puede constituir el modelo para otras enfermedades por inmunoglobulinas A (IgA), como el mieloma de IgA, la enfermedad celíaca relacionada con IgA y la artritis reumatoide, así como otras enfermedades por inmunoglobulinas. Por lo tanto, los investigadores de geneTIGA prevén desarrollar un «fármaco vivo» para tratar la NIgA y otras enfermedades asociadas a IgA para llevar a cabo los primeros ensayos clínicos en humanos.
Objetivo
"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 ActionsCoordinador
10117 Berlin
Alemania