- Design Therapeutics (DSGN, Financial) to present Phase 1 safety data for DT-168 at Eyecelerator @ Park City 2025.
- DT-168, an eye drop, targets genetic mutation causing Fuchs endothelial corneal dystrophy (FECD).
- Phase 1 trial involves single- and multiple-ascending dose studies in healthy volunteers.
Design Therapeutics, Inc. (DSGN), a clinical-stage biotechnology company, has announced it will present the Phase 1 safety data for its innovative DT-168 program at the Eyecelerator @ Park City 2025 conference on May 2, 2025. DT-168 is designed as a GeneTAC® small molecule eye drop aimed at treating Fuchs endothelial corneal dystrophy (FECD). The presentation will cover safety findings from single- and multiple-ascending dose trials conducted in healthy adult volunteers and outline plans for Phase 2 clinical development in FECD patients.
DT-168 specifically targets the CTG repeat expansion in the TCF4 gene, addressing the genetic mutation that underlies corneal endothelial cell dysfunction in FECD. This new approach hopes to provide a disease-modifying therapy for FECD, a condition that currently lacks such treatments and often necessitates corneal transplant surgery.
The significance of this development lies in its potential to shift the treatment paradigm for FECD from symptomatic management and surgical intervention to a non-invasive, gene-targeted therapy. While Phase 1 has focused on safety, the progression to Phase 2 trials in FECD patients will be crucial in assessing the efficacy of this novel treatment approach.