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ARL4C ASO-1316 is a chemically modified antisense oligonucleotide (ASO) targeting the ADP-ribosylation factor-like 4C (ARL4C) gene. It inhibits ARL4C expression at the RNA level by binding to the non-coding region of ARL4C mRNA, leading to reduced protein translation[1][2]. Preclinical studies demonstrate that ARL4C ASO-1316 suppresses the growth and invasiveness of xenograft tumors—particularly those induced by colon, lung, and pancreatic cancer cells—without inducing significant cell death in pancreatic cancer models[1][3]. In lung cancer cell lines, ARL4C ASO-1316 inhibits proliferation, migration, and tumor formation in vivo, even in cells with resistance to epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors[2]. In pancreatic cancer, it specifically reduces lymphatic metastasis and tumor cell invasion without affecting primary tumor size[1][3]. The drug is in preclinical development and has not yet entered clinical trials. It is considered a potential therapeutic for cancers that overexpress ARL4C, independent of KRAS or EGFR mutation status, and may be useful for targeting invasive and metastatic cancer phenotypes[1][2]. The ASO is chemically modified with AmNA and a phosphorothioate backbone for improved stability in vivo[2].
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