Design Therapeutics

Design Therapeutics, Inc. a preclinical-stage biopharmaceutical company, engages in the development of therapies for the treatment of genetic diseases caused by nucleotide repeat expansions. The company's portfolio of products comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function, eventually leading to neurological, cardiac, and metabolic dysfunction; and Myotonic Dystrophy Type-1 (DM1), a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs. It is also developing GeneTAC product candidate portfolio for the treatment of other nucleotide repeat expansion-driven monogenic diseases, such as Fragile X syndrome, spinocerebellar ataxias, amyotrophic lateral sclerosis, frontotemporal dementia, Huntington disease, and spinobulbar muscular atrophy. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.

Design Therapeutics' mission is to develop and deliver drug candidates that provide significant therapeutic benefit to patients with serious and life-threatening conditions.

Design Therapeutics' vision is for a future where their groundbreaking and innovative treatments are widely available and accessible, helping to improve and extend the lives of patients with rare diseases.

Design Therapeutics has been recognized for its dedication to medical science and was recently the recipient of the National Institutes of Health Grant for its research on cystinosis. The company has also been honored with awards from National Institute on Aging and the American Heart Association's Innovation in Medicine Prize for its research on gene therapy for atherosclerotic cardiovascular disease.