Design Therapeutics (DSGN) announced that the first Friedreich ataxia, FA, patient has been dosed via intravenous infusion in its RESTORE-FA open-label Phase 1/2 multiple-ascending dose clinical trial of DT-216P2. The RESTORE-FA trial is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of IV and subcutaneous administration of DT-216P2 in patients with FA. DT-216P2 has been administered in one patient to date with no adverse events reported, including no injection site thrombophlebitis. The trial is currently open for enrollment in Australia. Design anticipates reporting data from the MAD trial, including levels of frataxin expression based on 12 weeks of dosing, in 2026.
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