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BTB-301

Development stage: Preclinical
Lead developer: BTB Therapeutics
Modality: Small Molecules
Administration: Oral (anticipated Based On Class And Developer Statements)

Overview

BTB-301 is a small molecule drug developed by BTB Therapeutics designed to enhance the expression of spliced neoantigens in cancer cells. By increasing the presence of these neoantigens, BTB-301 aims to improve the efficacy of immune checkpoint inhibitors, potentially making tumors more recognizable and vulnerable to immune system attack. The compound leverages transcriptomics-based drug discovery, focusing on modulating RNA splicing as a therapeutic strategy for neoplasms (cancers). As of 2025, it remains in preclinical development[1][2][4].

Targets

spliceosome (NineTeen Complex)

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