From GlobeNewswire: 2025-03-10 07:07:00

Design Therapeutics has made progress in its gene therapy programs, with a Phase 1 trial in healthy volunteers underway for a treatment for Friedreich Ataxia (FA). Dosing is complete for another trial, with data expected in the first half of 2025. The company is well-funded with $245.5 million in cash and securities to support operations.

In addition to FA, Design Therapeutics is working on treatments for Fuchs Endothelial Corneal Dystrophy (FECD) and myotonic dystrophy type-1 (DM1). The company is on track to select a development candidate for DM1 in 2025. Preclinical work is also ongoing for Huntington’s disease.

Design Therapeutics reported fourth quarter and full year 2024 financial results, with $12.2 million in research and development expenses for the quarter. The company had a net loss of $13.7 million for the quarter but has a cash position of $245.5 million to fund operations into 2029.

The company’s GeneTAC® platform aims to develop small molecules that target disease-causing genes. Design Therapeutics is optimistic about its pipeline, with the potential for multiple clinical proof-of-concept data sets in the coming years. The company’s CEO, Pratik Shah, highlighted the busy year ahead with upcoming data releases and clinical activities.



Read more at GlobeNewswire:: Design Therapeutics Highlights Progress Across Lead