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Design Therapeutics Highlights Clinical and Research Progress and Reports First Quarter 2022 Financial Results
Clinical Execution of Lead GeneTAC™ Molecule, DT-216, with Initiation of Phase 1 Trial for the Treatment of Friedreich Ataxia Robust Research Pipeline of
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Clinical Execution of Lead GeneTAC™ Molecule, DT-216, with Initiation of Phase 1 Trial for the Treatment of Friedreich Ataxia Robust Research Pipeline of GeneTAC™ Candidates, with Promising New Data for Fuchs Endothelial Corneal Dystrophy Strong Cash Position with $371.2 Million to Support Multi-Year Operating Runway CARLSBAD, Calif., May 09, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted progress across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported first quarter 2022 financial results. “Design’s vision is to become a fully integrated biotech company that is designing, developing, and delivering transformative treatments for patients with debilitating inherited degenerative diseases,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “So far in 2022, we’ve made meaningful progress toward that vision with the initiation of clinical development with DT-216, our lead GeneTAC™ molecule, as a potential disease-modifying treatment for patients with Friedreich ataxia (FA). In our interactions with the FA community, they express tremendous interest in the development of DT-216, underscoring the urgent need among FA patients. We’ve continued to deliver on our milestones for this program, recently completing dosing in the first single-ascending dose cohort of our Phase 1 study, and we look forward to reporting topline data in the second half of this year. Other significant advancements in our GeneTACTM platform are exemplified by the exciting preclinical data we presented at ARVO. We showed that our GeneTAC™ molecules can correct the pathological hallmarks of the most common genetic cause of Fuchs endothelial corneal dystrophy – a disease that affects millions of people, leads to visual impairment and is the number one cause of corneal transplantation. Driven by our expert team, we believe we have developed an efficient operating model that supports a multi-year operating runway and enables us to execute our clinical and research milestones.” Recent Pipeline Progress Significant Clinical and Regulatory Progress with DT-216 for FA; Topline Phase 1 Data On-track for Second Half of 2022: Design completed dosing in the first cohort of its P...