Design Therapeutics is a biotechnology company developing a new class of therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company’s lead program is focused on the treatment of Friedreich’s ataxia and discovery efforts are ongoing in other degenerative diseases.
Our GeneTAC™ molecules are designed to selectively bind to genetic repeat sequences, modulate gene expression either by...
Design Therapeutics is a biotechnology company developing a new class of therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company’s lead program is focused on the treatment of Friedreich’s ataxia and discovery efforts are ongoing in other degenerative diseases.
Our GeneTAC™ molecules are designed to selectively bind to genetic repeat sequences, modulate gene expression either by restoring or blocking transcription, and restore cellular health. As a platform, we believe that GeneTAC™ molecules have broad potential applicability across monogenic nucleotide repeat expansion diseases.
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