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BT820

Development stage: Preclinical
Lead developer: Base Therapeutics
Modality: Adenine Base Editors → Base Editing → Gene Editing → Gene Therapies, Cytosine Base Editors → Base Editing → Gene Editing → Gene Therapies

Overview

BT820 is an investigational gene therapy candidate being developed by Base Therapeutics for the treatment of liver disease. It utilizes the company's proprietary AccuBase base editing platform, which is designed to enable precise, single-nucleotide changes in the genome without inducing double-strand DNA breaks. While the specific molecular target (referred to as Target 2) remains undisclosed, the therapy is intended to address genetic drivers of liver pathology. The program is currently in the IND-enabling stage of development.

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