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Design Therapeutics
Design Therapeutics (DSGN) investor relations material
Design Therapeutics Evercore ISI 8th Annual HealthCONx Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Evercore ISI 8th Annual HealthCONx Conference summary4 Dec, 2025
Business and R&D overview
Focused on modulating gene expression with small molecules for monogenic diseases.
Clinical programs in Friedreich's ataxia (FA), myotonic dystrophy type 1 (DM1), and Fuchs' corneal dystrophy; preclinical in Huntington's.
Strong cash position with runway into 2029, supporting ongoing and planned trials.
Friedreich's ataxia program
Initial DT-216 formulation showed limited exposure and vein thrombophlebitis, but validated frataxin RNA increase.
New DT-216P2 formulation achieves over 10x exposure, sustained duration, and resolves thrombophlebitis.
Ongoing multiple-dose escalation study in patients, with 12-week data expected in H2 2026.
Primary focus on frataxin RNA and protein in whole blood and muscle using gold-standard assays.
Dose escalation continues until efficacy or safety ceiling is reached; baseline and post-treatment biopsies planned.
Fuchs' corneal dystrophy program
Developed first biomarker for splicing in Fuchs using discarded corneal tissue from transplants.
Patients receive eye drops before scheduled surgery; tissue analyzed for splicing correction.
Ex-vivo studies show toxic foci vanish in 6–14 days with treatment.
Data from ongoing phase 2 biomarker study expected in H2 2026.
What Frataxin increase signals FA program success?
DM1: Small molecule advantage vs. oligo therapies?
Fuchs: How interpret splicing data without baseline?
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Next Design Therapeutics earnings date
Q4 202511 Mar, 2026
Design Therapeutics
Next Design Therapeutics earnings date
Q4 202511 Mar, 2026
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Frequently asked questions
Design Therapeutics Inc. is a preclinical-stage biopharmaceutical company focused on developing therapies for genetic diseases caused by nucleotide repeat expansions. Their innovative approach centers on the development of GeneTAC™ gene targeted chimera small molecules. These molecules are designed to modulate the expression of disease-causing genes, offering potential treatments for a range of genetic disorders. The company's research portfolio includes projects targeting conditions such as Friedreich Ataxia, a severe progressive multi-system disease; Myotonic Dystrophy Type-1 (DM1), affecting muscle, heart, and other organs; and other nucleotide repeat expansion-driven diseases like Fragile X syndrome and Huntington disease. The company is headquartered in Carlsbad, California, and its shares are listed on the Nasdaq.
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