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, 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.