APN401

APN401 is a first-in-class individualized cellular therapy developed for the treatment of solid tumors. It consists of autologous peripheral blood mononuclear cells (PBMCs) collected from the patient and transfected ex vivo with small interfering RNA (siRNA) targeting Casitas B-lineage lymphoma protein-b (Cbl-b), an E3 ubiquitin ligase that acts as a key intracellular immune checkpoint limiting lymphocyte activation. By silencing Cbl-b in these immune cells, APN401 aims to enhance T cell and natural killer cell-mediated antitumor activity by overriding multiple immune checkpoints. The modified PBMCs are then reinfused into the patient via intravenous administration. This approach is designed to supercharge the patient's own immune system against cancer and has shown feasibility and safety in Phase I clinical trials for patients with refractory solid tumors such as pancreatic cancer, colorectal cancer, kidney cancer, lymphoma, and other advanced malignancies[1][4][5][7]. The therapy leverages rapid manufacturing technology enabling same-day treatment cycles[7].

Other names

siRNA-transfected peripheral blood mononuclear cells Apeiron