Key Performance
More- Earnings Score: N/A
- Momentum Score: 87
- True Yield: N/A
- Financial Health Score: N/A
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Latest Research & News
Design Therapeutics announced positive Phase 1/2 data from its RESTORE-FA trial showing DT-216P2 demonstrated dose-dependent clinical improvements in Friedreich ataxia patients after four weeks of treatment, including improvements in mFARS scores, upright stability, and fatigue. The drug showed comprehensive biomarker activity with 65% increase in FXN mRNA and 22-27% increase in FXN protein levels. The company plans to advance toward registrational development with updates expected in Q4 2026.
05/18/2026, 7:00 AM • GlobeNewswire
Design Therapeutics announced it will present Phase 1/2 RESTORE-FA trial data for DT-216P2, its experimental treatment for Friedreich's ataxia, on May 18, 2026. The company is advancing multiple clinical-stage programs using its GeneTAC gene-targeted chimera platform, with trials ongoing for corneal dystrophy and myotonic dystrophy treatments, and dosing expected to begin for its Huntington's disease program in the first half of 2026.
05/17/2026, 4:00 PM • GlobeNewswire
Design Therapeutics to Participate in Upcoming Investor Conferences
Design Therapeutics plans to initiate patient dosing for DT-818, a potential treatment for Myotonic Dystrophy Type-1, in the first half of 2026. The company has obtained ex-US regulatory clearance and is developing gene-targeted therapies for various genetic diseases.
11/26/2025, 8:00 AM • GlobeNewswire
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Company Profile
Design Therapeutics, Inc. a clinical-stage biopharmaceutical company, research, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. The company was incorporated in 2017 and is headquartered in Carlsbad, California.