The University of Florida is seeking a company interested in commercializing a novel gene therapy for eliminating tissue scarring caused by ocular surgical procedures and chronic diseases. The therapy employs RNA enzymes (ribozymes) to inhibit the synthesis of connective tissue growth factor (CTGF), high levels of which are associated with excessive regenerative tissue. This therapy would spare patients undergoing ocular surgery the pain and suffering of deteriorating eyesight caused by scarring, a serious surgical complication. In addition, the technology would be a target-specific, non-toxic therapy for the millions of patients suffering from fibrotic diseases such as scleroderma and keloids.
Gene therapy to eliminate scarring from fibrotic diseases and ocular surgeries
- Ensures a significantly greater success rate for glaucoma and refractive surgeries, facilitating the restoration of eyesight to a greater population of patients
- Remains active for as long as a risk of scar tissue formation exists, minimizing the number of deliveries and thereby maximizing patient convenience
- Only affects target tissues, eliminating risk of long-term damage otherwise possible with currently available toxic, non-specific, chemical-based drugs
- Offers the possibility for more affordable treatment, since ribozyme gene therapy costs less than currently available scar-reducing antibody therapies
- Revolutionary preventative treatment approach offers major market opportunities
Elevated levels of the secreted protein CTGF are found in fibrotic tissues and in patients with fibrotic diseases such as scleroderma and keloids, indicating that its presence leads to scarring. Researchers at the University of Florida have developed a ribozyme-based gene therapy that inhibits the protein’s synthesis, effectively reducing symptoms of certain diseases and scarring-related surgical complications. This therapy would be delivered by plasmid DNA or viral vectors. Ribozyme treatment has the distinct advantages of specificity, non-toxicity and a regulable timeframe of activity, thereby circumventing side effects and inconveniences inherent to other scar reduction therapies currently in development.