Drug intelligence / Profile preview

OTX-2101

Development stage
Discontinued
Lead developer
Omega Therapeutics
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

OTX-2101 is a first-in-class, preclinical-stage mRNA-based epigenomic controller developed by Omega Therapeutics for the treatment of non-small cell lung cancer (NSCLC). It is designed to downregulate expression of the c-MYC (MYC) oncogene through pre-transcriptional epigenetic modulation. MYC is a master transcription factor implicated in cell proliferation, differentiation, and apoptosis, and its overexpression occurs in approximately 60% of NSCLC cases. OTX-2101 utilizes lipid nanoparticle (LNP) delivery to introduce programmable mRNA that modulates gene expression at the epigenetic level. Preclinical data have shown that combining this MYC-targeting approach with immune checkpoint or EGFR inhibitors enhances anti-tumor activity in NSCLC models. As of late 2024, development has been paused as part of a strategic refocus by Omega Therapeutics[6][9][5].

02

Targets

MYC (MYC proto-oncogene protein)

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