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BTB-201

Development stage: Preclinical
Lead developer: BTB Therapeutics
Modality: Small Molecules

Overview

BTB-201 is a preclinical-stage small molecule drug being developed by BTB Therapeutics, Inc. It functions as a GLA modulator (alpha-galactosidase modulator) and is being developed for the treatment of nervous system diseases and cardiovascular diseases, specifically Cardiac Fabry disease. Fabry disease is caused by abnormal RNA splicing of the alpha-galactosidase A (GLA) gene. The drug represents BTB Therapeutics' approach to addressing genetic disorders through transcriptomics-based drug discovery that intervenes with RNA splicing using small molecules.

Targets

GLA (Alpha-galactosidase A)

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