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Design Therapeutics Inc is a clinical-stage biopharmaceutical company engaged in the research and development of GeneTACTM 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 product candidate is in Friedreich ataxia (FA), its second product candidate is in Fuchs endothelial corneal dystrophy (FECD), and it is also advancing its GeneTACTM program to address other serious nucleotide repeat-driven monogenic diseases.
Lionheart Holdings is a blank check company.