Drug intelligence / Profile preview

ASO-4733

Development stage
Unknown
Lead developer
Nagoya University
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intraperitoneal
01

Overview

ASO-4733 is an antisense oligonucleotide (ASO) designed to target and inhibit the expression of **Transforming growth factor beta 2 (TGF-beta2)**. Developed by researchers at the University of Tokyo with support from the Japan Agency for Medical Research and Development (AMED), it is primarily investigated for the treatment of advanced gastric cancer and gastroesophageal junction adenocarcinoma with peritoneal dissemination (peritoneal carcinomatosis). TGF-beta2 is a cytokine that plays a critical role in promoting the epithelial-mesenchymal transition (EMT), tumor invasion, and the creation of a fibrotic, immunosuppressive microenvironment within the peritoneal cavity. By binding to TGF-beta2 mRNA and inducing its degradation, ASO-4733 aims to suppress these metastatic drivers. In clinical settings, the drug is administered via the **intraperitoneal** route to achieve high local concentrations at the site of metastasis, often in combination with systemic chemotherapy regimens such as SOX (S-1 and oxaliplatin).

02

Targets

TGFB2 (Transforming growth factor beta 2)

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