Drug intelligence / Profile preview

aAVC-WT1

Development stage
Unknown
Lead developer
RIKEN
Modality
Cell Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Vaccines & Immunotherapeutics
Administration
Intravenous
01

Overview

aAVC-WT1 is an artificial Adjuvant Vector Cell (aAVC) therapy targeting the Wilms' Tumor 1 (WT1) protein, developed by RIKEN and licensed to BrightPath Biotherapeutics (as BP1209). The therapy consists of allogeneic cells transfected with mRNA encoding the WT1 antigen and loaded with the glycolipid alpha-galactosylceramide (α-GalCer). Upon administration, α-GalCer is presented by CD1d molecules on the aAVC surface, leading to the potent activation of invariant Natural Killer T (iNKT) cells. This innate immune activation triggers the maturation of the patient's endogenous dendritic cells, which then present WT1 epitopes to prime and expand WT1-specific cytotoxic T lymphocytes (CTLs) and memory T cells. This dual mechanism—combining innate NKT cell activation with adaptive T cell responses—is designed to provide a more robust and durable anti-tumor effect compared to traditional peptide or dendritic cell vaccines. It is primarily being investigated for the treatment of acute myeloid leukemia (AML) and other WT1-positive malignancies.

Other names
artificial Adjuvant Vector Cells expressing WT1
02

Targets

CD1D (Tumor-associated macrophage/myeloid-derived suppressor cell CD1d complex)pMHC-II (Peptide-major histocompatibility complex class II complex)pMHC-I (Peptide–MHC class I complex)

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