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Design Therapeutics Announces Preclinical Data Highlighting Novel GeneTAC™ Therapy for the Treatment of Fuchs Endothelial Corneal Dystrophy to be Presented at ARVO 2022
Treatment with GeneTAC™ Small Molecules Reduced Nuclear Foci and Restored Normal Splicing In Vitro, Supporting Further Development CARLSBAD, Calif., March 09,
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Treatment with GeneTAC™ Small Molecules Reduced Nuclear Foci and Restored Normal Splicing In Vitro, Supporting Further Development\nCARLSBAD, Calif., March 09, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced that preclinical data supporting the potential of the company’s novel GeneTAC™ small molecules to correct the most common underlying genetic cause of Fuchs endothelial corneal dystrophy (FECD) will be presented at the Association for Research in Vision and Ophthalmology 2022 Annual Meeting (ARVO 2022). The in vitro data in corneal endothelial cells derived from patients with FECD showed that the company’s FECD GeneTAC™ molecules reduced toxic nuclear foci and restored normal splicing. The data will be presented at ARVO 2022, which is being held in Denver, Colorado from May 1- 4, 2022 and virtually from May 11-12, 2022. FECD is characterized by progressive degeneration of the corneal endothelium and subsequent loss of vision. This genetic eye disease affects more than one million people in the U.S., with approximately 75% of cases caused by a CTG trinucleotide repeat expansion within the transcription factor 4 (TCF4) gene, leading to the formation of toxic RNA foci, global splicing dysregulation, cellular dysfunction, and eventual death of corneal endothelial cells. Due to the lack of disease-modifying therapies approved for FECD, corneal transplantation is the only procedure currently used to restore vision. Design’s approach utilizes its FECD GeneTAC™ molecules to selectively target the expanded CTG repeats in the TCF4 gene to reduce RNA foci formation and mis-splicing. Design conducted preclinical studies to evaluate its FECD GeneTAC™ molecules in corneal endothelial cells, which contained nuclear foci, derived from patients with FECD. When tested in vitro, FECD GeneTAC™ molecules led to robust reductions in toxic nuclear RNA foci of up to 99% in a time- and concentration-dependent manner. The FECD GeneTAC™ molecules also corrected key mis-spliced transcripts in FECD corneal endothelial cells to levels observed in unaffected corneal endothelial cells. “FECD is the leading indication for corneal transplantation, which is usually reserved for more advanced stages of the disease. GeneTAC™ molecules are designed...