Update from Design Therapeutics

Friedreich Ataxia (FA) Patient Dosing to Begin in mid-2025

Today, March 10, 2025, Design Therapeutics announced progress across its lead GeneTAC® programs including DT-216P2, a candidate being developed for the treatment of FA. A Phase 1 dosing study in healthy volunteers has been initiated in Australia to evaluate the safety and pharmacokinetics (PK) of single ascending doses of DT-216P2 via multiple routes of administration (intravenous infusion, subcutaneous infusion and subcutaneous injection). A Phase 1/2 multiple ascending dose (MAD) clinical trial to assess safety, PK and pharmacodynamics (PD) in FA patients is anticipated to begin in mid-2025.

Read the full press release
3 10 2025 Design