CARLSBAD, Calif. - Design Therapeutics Inc. (NASDAQ:DSGN), a biotechnology firm focused on degenerative genetic diseases, announced progress in its GeneTAC™ portfolio and financial results for the fourth quarter and full year of 2023.
The company's latest developments include a new drug product for Friedreich Ataxia (FA), advancements in a treatment for Fuchs Endothelial Corneal Dystrophy (FECD), and the unveiling of a program for Huntington's Disease (HD).
For FA, Design has developed DT-216P2, which has shown an improved pharmacokinetic profile and favorable injection site safety in nonclinical studies. Design plans to complete Good Laboratory Practice (GLP) studies by the end of 2024, with patient trials expected to start in 2025.
The U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for DT-168, Design's candidate for FECD, which is believed to be the first effective treatment addressing the root cause of this degenerative corneal disease.
An observational study is currently underway to confirm patient characteristics and evaluate potential endpoints, with Phase 1 development set to begin in 2024.
Moreover, Design has introduced a new program targeting HD, aiming to reduce mutant huntingtin gene expression with a GeneTAC™ small molecule. Preclinical studies have shown over 50% reduction in mutant gene expression in the brain striatum with systemic administration.
The company also reported financial results, with research and development expenses totaling $57.1 million for the year ended December 31, 2023. General and administrative expenses were $21.1 million for the same period, and the net loss stood at $66.9 million.
Design's cash position, including cash equivalents and marketable securities, was $281.8 million as of year-end 2023, which is expected to fund operations for the next five years.
Design will host a conference call and webcast today at 4:30 pm ET to discuss these updates, accessible through the Investors page of their website.
This news is based on a press release statement and reflects the company's current expectations and projections for its drug development programs. These statements are forward-looking and subject to risks and uncertainties that could cause actual results to differ materially. The company undertakes no obligation to revise or update this information unless required by law.
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