OBJECTIVES

Within the funding period geneTIGA aims to:

• identify specific Chimeric Antigen Receptor (CAR) and transgenic T Cell Receptor (tgTCR) candidates for redirected T cell mediated selective targeting of IgA-producing B and plasma cells
• establish an efficient genome editing process combined with an unbiased and in-depth safety analysis pipeline to benchmark different gene editing platforms and develop effective and safe tgTCR/CAR cell products
• set up a manufacturing process for IgA-targeting T cell products and to define and validate toolboxes for product specification and patient monitoring
• measure the efficacy (long-term IgA suppression) and safety of IgA-redirected T cells in humanised mouse models and advanced multi-organ-chip in vitro models
• develop a regulatory pathway ready for the First-in-Human study in IgAN and related IgA diseases
• serve as a blueprint for other genome-edited tgTCR/CAR-T cell products using the enabling technology toolboxes and regulatory pathway