Definition

A preparation of autologous T lymphocytes that have been genetically modified to express a chimeric antigen receptor (CAR) targeting the tumor-associated antigen (TAA) ganglioside GM2 (GM2) and producing the immune regulators interleukin-7 (IL-7) and C-C motif chemokine 19 (CCL19) using PRIME (proliferation-inducing and migration-enhancing) technology, with potential immunostimulating and antineoplastic activities. Upon administration, autologous IL-7/CCL19-expressing anti-GM2 CAR T cells NIB-101 target and bind to GM2-expressing tumor cells, thereby inducing selective toxicity in GM2-expressing tumor cells. In addition, as IL-7 promotes the proliferation and survival of T-cells and CCL19 enhances the migration of host T cells and dendritic cells (DCs), the production of IL-7 and CCL19 by NIB-101 allows for enhanced trafficking and accumulation of the PRIME CAR T cells and other endogenous immune cells, including T cells and DCs in tumor tissues. This further activates the host immune system to induce anti-tumor immune responses to various cancer antigens and against tumor cells lacking the CAR target antigen. GM2, a type of glycosphingolipid, is overexpressed on the surface of many cancer cells, such as multiple myeloma (MM) cells and neuroblastoma cells.