Biotechnology company developing therapies for genetically defined degenerative diseases.
Design Therapeutics, Inc. is a pioneering preclinical-stage biopharmaceutical firm based in Carlsbad, California, dedicated to developing innovative therapies for genetic diseases caused by nucleotide repeat expansions. Founded in 2017, the company focuses on addressing challenging conditions such as Friedreich Ataxia and Myotonic Dystrophy Type-1 (DM1), both of which are debilitating diseases affecting multiple organ systems.
Friedreich Ataxia is a progressive, autosomal recessive disorder that primarily impacts organ systems reliant on mitochondrial function, leading to neurological, cardiac, and metabolic impairments over time. DM1, on the other hand, is a dominantly-inherited neuromuscular disease affecting skeletal muscle, heart, brain, and other vital organs.
Design Therapeutics is advancing a robust GeneTAC product candidate portfolio aimed at treating a spectrum of nucleotide repeat expansion-driven monogenic diseases. This includes conditions like Fragile X syndrome, spinocerebellar ataxias, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Huntington disease, and spinobulbar muscular atrophy, underscoring its commitment to addressing a broad range of genetic disorders.
With a strategic focus on innovative therapeutic approaches, Design Therapeutics collaborates with leading researchers and institutions to advance its promising pipeline. The company is poised to make significant strides in the field of genetic medicine, aiming to improve patient outcomes and quality of life for individuals affected by these challenging and currently untreatable conditions.