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BTB-202

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

Overview

BTB-202 is a small molecule drug in preclinical development by BTB Therapeutics. It is designed to treat Long QT syndrome by targeting abnormal RNA splicing of cardiac ion channels, which is the most common cause of this condition. The drug aims to correct the underlying genetic defect at the RNA level, offering a novel approach for rare cardiovascular diseases. Additionally, preclinical studies suggest that BTB-202 may induce splice-derived neoantigens in cancer cells and enhance the efficacy of immune checkpoint inhibitors, indicating potential applications in cancer immunotherapy[1][2][3].

Targets

Aberrantly spliced cardiac ion channel pre-messenger RNA

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