Design Therapeutics Inc (NASDAQ: DSGN) announced it would represent preclinical data on its GeneTAC therapy. The company developed this treatment for Fuchs endothelial corneal dystrophy (FECD). It will present this data at the annual meeting for the Association for Research in Vision and Ophthalmology.
The researchers’ tests found that the therapy restored regular splicing and lowered harmful nuclear foci endothelial cells of the cornea. Design Therapeutics will release more of the finding at the annual meeting between May 1 and 4 in Colorado. Moreover, the committee will hold another virtual meeting between May 11 and 12.
Corneal transplant is given for severe cases of FECD
When a patient develops FECD, their corneal endothelium begins to degenerate, which could cause vision loss. The Food and Drug Administration (FDA) has not approved any disease-modifying treatments for the illness. For this reason, many physicians opt for a corneal transplant to treat it.
Design Therapeutics uses its therapy to target the CTG repeats on TCF4 genes, which is the cause of the disease in about 75% of the patients. This move reduces mis-splicing and the formation of RNA foci.
In the preclinical studies, Design Therapeutics conducted, researchers tested the therapy on endothelial cells of the cornea. Researchers had previously extracted these cells, which had nuclear foci, from FECD patients.
The researchers noticed a significant reduction by about 99% of harmful nuclear RNA foci. Furthermore, GeneTAC molecules restored vital mis-spliced in the cells to normal.
According to Design Therapeutics’ CEO and President, Dr. João Siffert, most cornea transplants result from FECD. Unfortunately, physicians could only use this procedure for advanced disease stages. For this reason, Design Therapeutics hopes that GeneTAC could change this.
GeneTAC can treat early-stage FECD
The therapy modulates transcription and targets genomic sequences. This therapy removes the need for gene editing and surgery. It can be a better option for people in the early stage of disease than the testament the medical field currently offers.
From their clinical studies, the company believes that FECD GeneTAC therapy can correct the reason for FECD. Design Therapeutics also hopes to utilize the same model on other genetic conditions.
Design Therapeutics is a company focused on creating therapies using GeneTAC. This technology increases or lowers the expression of specific genes to treat a disease.