DSGN Profile
Design Therapeutics, Inc., headquartered in Carlsbad, California, is a preclinical-stage biopharmaceutical company focused on developing innovative therapies for genetic diseases caused by nucleotide repeat expansions. Founded in 2017, the company is dedicated to addressing unmet medical needs through its advanced drug discovery and development platforms.
The company’s primary therapeutic efforts are centered on treating two major genetic conditions. Friedreich Ataxia is a rare, autosomal recessive disease that leads to progressive degeneration of the nervous system, including neurological, cardiac, and metabolic dysfunctions due to mitochondrial impairment. Myotonic Dystrophy Type-1 (DM1) is another target, a dominantly-inherited neuromuscular disorder that progressively affects skeletal muscle, cardiac function, brain, and other organ systems, significantly impairing quality of life.
Design Therapeutics is also advancing its GeneTAC platform, a portfolio of product candidates designed to address other nucleotide repeat expansion-driven diseases. This includes Fragile X syndrome, spinocerebellar ataxias, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Huntington's disease, and spinobulbar muscular atrophy. The GeneTAC technology aims to offer targeted, disease-modifying treatments by utilizing novel approaches to gene regulation and correction.
By leveraging cutting-edge biotechnology and a deep understanding of genetic disease mechanisms, Design Therapeutics seeks to provide transformative treatments for these challenging conditions. The company’s commitment to precision medicine and innovative therapies positions it as a key player in the development of targeted solutions for genetic disorders with significant unmet needs.
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