Design Therapeutics (DSGN) Oppenheimer 36th Annual Healthcare Life Sciences Conference summary

Platform overview and innovation

• Developing small molecule genetic medicines to modulate gene expression for single-gene disorders.

• Small molecules offer pan-cellular distribution, overcoming delivery challenges of large genomic medicines.

• Three clinical-stage programs: Friedreich's ataxia (FA), Fuchs' endothelial corneal dystrophy (FECD), and myotonic dystrophy type 1 (DM1).

• Success in any program could drive significant advances and shareholder value.

• Preclinical Huntington's disease program also in development.

Friedreich's ataxia (FA) program

• RESTORE-FA trial aims to increase endogenous frataxin expression using DT-216.

• New DT-216P2 formulation shows more sustained exposure in muscle tissue.

• Any increase in frataxin from baseline is considered a positive outcome; measured in whole blood and muscle.

• Data from the multiple ascending-dose trial expected in the second half of this year.

• Significant unmet need remains despite existing treatments, with a large market opportunity.

Fuchs' endothelial corneal dystrophy (FECD) program

• DT-168 eye drops target toxic RNA foci caused by CTG expansion in TCF4 gene.

• Phase I healthy volunteer study showed no clinically significant adverse events.

• Developed a novel biomarker to measure splicing correction in corneal tissue.

• Exploratory biomarker study ongoing in patients scheduled for corneal transplant.

• Study aims to demonstrate DT-168's effect on splicing in diseased tissue.