Design Therapeutics, Inc.

Design Therapeutics is a biopharmaceutical company advancing novel, small molecule therapeutic candidates designed to bring functional cures to serious genetic diseases with long-standing unmet need. Through its GeneTAC (Gene Targeted Chimera) platform, the company leverages its understanding of gene regulation and small molecules to develop candidates targeting the underlying cause of genetic disorders. Its pipeline has an initial focus on monogenic repeat expansion disorders, including Friedreich ataxia, Fuchs endothelial corneal dystrophy, myotonic dystrophy and Huntington's disease. The company is located in Carlsbad, United States.

Design was founded with a vision of restoring health to patients with genetic disorders by harnessing their body's own biology. Its team consists of recognized global leaders in the fields of DNA-targeted small molecules, transcription modulation, medicinal chemistry, translation, and development into clinical applications. The company is located in Carlsbad, United States.

Pioneering the field of Small Molecule Genomic Medicine

• GeneTAC (Gene Targeted Chimera) platform

  A platform through which the company leverages its understanding of gene regulation and small molecules to develop therapeutic candidates targeting the underlying cause of genetic disorders.

1. 1Design Therapeutics – Designing a New Class of Genomic Medicinesdesigntx.com
2. 2About Us – Design Therapeuticsdesigntx.com
3. 3Design Therapeutics, Inc. (United States)wikidata.org