Design Therapeutics (DSGN) Corporate presentation summary

Platform overview

• GeneTACⓇ small molecules modulate gene transcription to treat monogenic disorders, offering broad tissue distribution and overcoming limitations of traditional genomic medicines.

• Proprietary platform supports multiple programs targeting severe genetic diseases, aiming for first- or best-in-class therapies.

Pipeline and clinical programs

• Four GeneTACⓇ programs in development: Friedreich Ataxia (FA), Fuchs Endothelial Corneal Dystrophy (FECD), Myotonic Dystrophy Type 1 (DM1), and Huntington's Disease (HD).

• Three programs (FA, FECD, DM1) are in clinical stages as of 2026, with HD in preclinical research.

Friedreich Ataxia (DT-216P2)

• DT-216P2 normalizes FXN levels in FA patient cells without affecting healthy cells, addressing the genetic root cause.

• Phase 1/2 RESTORE-FA study shows improvements in mFARS and Upright Stability Score, with significant biomarker increases at 1.0 mg/kg IV weekly.

• Safety profile is favorable, with no serious adverse events and only mild to moderate ALT elevations.

• Registrational development planned, with an update anticipated in Q4 2026.