Business
Design Therapeutics Reports Pipeline and Business Progress and Fourth Quarter and Full Year 2021 Financial Results
Phase 1 Trial of DT-216, a Novel FA GeneTAC™ Molecule, in Patients with Friedreich Ataxia On Track to Begin Soon Preclinical Data Supporting Development of
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Phase 1 Trial of DT-216, a Novel FA GeneTAC™ Molecule, in Patients with Friedreich Ataxia On Track to Begin Soon Preclinical Data Supporting Development of Novel GeneTAC™ Small Molecules for the Treatment of Fuchs Endothelial Corneal Dystrophy (FECD) to be Presented at ARVO 2022 Well-Capitalized with $384.1 Million in Cash and Investments at the End of 2021 to Support Upcoming Milestones CARLSBAD, Calif., March 10, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced pipeline and business progress and reported fourth quarter and full year 2021 financial results. “This is an exciting time for Design as we continue to progress our pipeline of novel GeneTAC™ molecules toward patients, particularly those with limited therapeutic options or no disease modifying treatments available today,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “The recent clearance of our IND for DT-216 is a significant milestone for the company, and we’re excited to begin our clinical trial in patients with Friedreich ataxia, a genetic, progressive disease that has a life-altering impact. In parallel, we continue to advance our myotonic dystrophy type-1 GeneTAC™ molecules toward clinical development, and have recently generated encouraging new data showing that our GeneTAC™ molecules may be able to correct the most common underlying genetic cause of Fuchs endothelial corneal dystrophy, a prevalent genetic condition affecting over 1 million people in the United States that can lead to vision loss. 2022 is set to be a meaningful year for Design, with key upcoming milestones and a pipeline comprised of potentially transformative new genomic medicines.” “Design was founded to address the known monogenic causes of a range of inherited degenerative diseases and now, just two years from launch, is on the cusp of initiating its first clinical trial,” added Pratik Shah, Ph.D., executive chair of Design Therapeutics. “With a highly productive platform, an experienced team and a strong balance sheet in place, the company is well-positioned to advance its robust pipeline to bring forward a new class of treatments for patients living with devastating genetic diseases.” Recent Pipeline Progress IND Application for FA Deve...