Drug intelligence / Profile preview

RROL inhibitor

Development stage
Preclinical
Lead developer
Dana-Farber Cancer Institute
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

RROL inhibitor is a first-in-class antisense oligonucleotide (ASO) designed to target and inhibit the long noncoding RNA (lncRNA) RNA Regulator of Lipogenesis (RROL), also known as lnc-17-92, which is derived from the MIR17HG gene. RROL acts as a nuclear-retained chromatin scaffold that mediates the assembly of the c-MYC (MYC) and WDR82 transcriptional complex at the promoter region of the ACACA gene (encoding acetyl-CoA carboxylase 1, ACC1). This complex drives de novo lipogenesis (DNL), a metabolic pathway critical for the proliferation and survival of multiple myeloma (MM) cells. By targeting RROL, the inhibitor dismantles the RROL-MYC-WDR82-ACC1 complex, suppressing DNL and exerting potent anti-tumor activity. Developed by researchers at the Dana-Farber Cancer Institute, RROL inhibitors have demonstrated strong preclinical efficacy in vitro and in vivo in multiple myeloma models.

Other names
ASO-1ASO1ASO 1lnc-17-92 inhibitorlnc17-92 inhibitorlnc 17-92 inhibitorMIR17HG pre-RNA antisense inhibitorMIR-17HG pre-RNA antisense inhibitorMIR 17HG pre-RNA antisense inhibitorRNA Regulator of Lipogenesis inhibitor
02

Targets

ACC (Acetyl-CoA Carboxylase 1)MYC (MYC proto-oncogene protein)RROL (RNA Regulator of Lipogenesis)

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