DSGN
Design TherapeuticsยทNASDAQ
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DSGN Profile
Design Therapeutics, Inc.
A clinical-stage biotech company that develops treatments for serious degenerative genetic diseases
6005 Hidden Valley Road, Suite 110, Carlsbad, California 92011
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Design Therapeutics, Inc. was incorporated on December 18, 2017 under the laws of the State of Delaware. They are a clinical-stage biopharmaceutical company pioneering new small molecule therapeutic candidates, called GeneTACs, designed for disease treatment. Certain nucleotide repeat amplification diseases, such as FA, can lead to decreased expression of specific mRNAs; in other diseases, such as myotonic dystrophy type 1 (DM 1), FECD and Huntington's disease, nucleotide repeat amplification can lead to the production of toxic gene products, usually associated with pathological nuclear lesions. Their genes are designed to selectively bind to gene repeats, regulate gene expression by restoring or blocking mRNA transcription, and restore cell health. As a platform, they believe that GeneTACs have broad potential applicability in single-gene nucleotide repeat amplification diseases.
