Key information

🔎 Fund Overview

Design Therapeutics, Inc. is a clinical-stage biopharmaceutical company. The Company is focused on research and development of GeneTAC molecules, which are a novel class of small-molecule gene targeted chimera therapeutic candidates designed to be disease-modifying by addressing the underlying cause of diseases caused by inherited nucleotide repeat expansion mutations. The Company’s lead product candidate is Friedreich ataxia (FA). Its second product candidate is Fuchs Endothelial Corneal Dystrophy (FECD). It is also advancing its GeneTAC program to address other serious nucleotide repeat-driven monogenic diseases. Its FA is a devastating monogenic, autosomal recessive progressive disease. The FECD program is focused on the development of a potentially disease-modifying medical treatment for FECD. Its Myotonic Dystrophy Type-1 (DM1) is a monogenic, autosomal dominant, progressive neuromuscular disease that affects skeletal muscle, heart, brain, and other organs.