We are a preclinical-stage biopharmaceutical company pioneering novel small-molecule therapeutic candidates, called gene targeted chimeras (GeneTACs), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases. Certain nucleotide repeat expansion diseases, such as Friedreich ataxia (FA), can result in reduced expression of specific mRNAs; in other diseases, such as myotonic dystrophy type-1 (DM1), Fuchs endothelial corneal dystrophy (FECD), and Huntington disease, the nucleotide repeat expansions result in the generation of toxic gene products, often associated with pathological nuclear foci. Our GeneTACs 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 GeneTACs have broad potential applicability across monogenic nucleotide repeat expansion diseases.

We plan to initiate clinical trials with our lead product candidate in Friedreich ataxia (FA) patients to evaluate its safety, pharmacokinetics (PK) and effect on FXN levels by the first half of 2022, subject to receiving regulatory clearance to proceed into clinical trials. Note: Friedreich ataxia is caused by a defect (mutation) in a gene labeled FXN, which carries the genetic code for a protein called frataxin. In this rare inherited disease, the nerve fibers in the spinal cord and peripheral nerves degenerate, according to a fact sheet published by the National Institutes of Health (NIH) – National Institute of Neurological Disorders and Stroke. The disease, which does not impair cognitive ability, affects about 1 in every 50,000 people, male and female. FA can lead to loss of mobility, requiring the patient to use a wheelchair. FA also causes heart disease, the leading cause of death in people with this disease.

(Note: Design Therapeutics priced its IPO on March 25, 2021, at $20 – the top of its $18-to-$20 range – on 12 million shares, the same number of shares as in the prospectus, to raise $240 million.)