Design Therapeutics, Inc. (DSGN) reported that its GeneTAC candidate DT-216P2 delivered dose-dependent improvements in both biomarkers and clinical measures in patients with Friedreich ataxia after four weeks of intravenous dosing in the ongoing Phase 1/2 RESTORE-FA trial.
Friedreich ataxia (FA) is a rare, inherited degenerative disease caused by a GAA repeat expansion in the FXN gene, leading to reduced frataxin protein levels and progressive neurological decline. Current treatment options remain limited, and therapies that directly address the genetic root cause are urgently needed.
In the 1 mpk dose cohort, patients showed mean improvements of 6.4 points on the modified Friedreich's Ataxia Rating Scale (mFARS) and 2.7 points in upright stability, alongside clinically meaningful reductions in fatigue scores exceeding the threshold for minimal important change. Biomarker analysis revealed a 65% increase in whole blood FXN mRNA, 22-27% increases in FXN protein, and a 42% rise in muscle FXN mRNA, supporting the observed functional improvements. The therapy was generally well-tolerated, with only mild to moderate adverse events reported.
CEO Pratik Shah emphasized that this is the first time endogenous frataxin increases have been observed alongside clinical improvements of this magnitude in patients with FA, underscoring the potential of DT-216P2 as a best-in-disease treatment. Based on these findings, Design plans to advance DT-216P2 toward registrational development, with an update expected in the fourth quarter of 2026.
The RESTORE-FA trial represents a significant step in Design's broader GeneTAC platform, which aims to dial gene expression up or down to address the underlying causes of serious genetic diseases. In addition to DT-216P2 for FA, the company is advancing programs including DT-168, for Fuchs endothelial corneal dystrophy, and DT-818, for myotonic dystrophy type-1, and Huntington's disease.
DSGN has traded between $3.31 and $17.25 over the past year. The stock closed Friday's trading (May 15, 2026) at $14.37, down 1.17%. In pre-market trading, the stock is at $15.66, up 8.99%.
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