A Novel Drug Candidate for the Treatment of Diabetic Retinopathy

The condition called diabetic retinopathy stems from damage to the blood vessels of the retina caused by diabetes. This is the most common diabetic eye disease and a leading cause of blindness in the United States. No treatment is available in the early stages of the disease, which also frequently have no symptoms. Once the disease is discovered, patients can prevent its progression by controlling their blood pressure and their blood sugar and blood cholesterol levels. In the advanced stage called proliferative retinopathy, treatment consists of laser surgery to shrink the abnormal blood vessels. This surgery is not always effective and can destroy normal retinal tissue. No approved pharmaceutical therapy is available. These researchers are developing a new drug candidate with the potential to have sustained effectiveness against the blood leakage into the retina associated with this condition.

Scientific Synopsis

Diabetic retinopathy (DR) is a common complication of diabetes. Diabetic macular edema (DME) is a major pathological feature of DR and a leading cause of blindness. Cystoid macular edema (CME) represents a common cause of vision impairment following cataract surgery. High blood-retinal barrier or retinal vascular leakage is believed to be responsible for DME and CME. Laser photocoagulation has remained the gold standard for the treatment of DME/CME over the past two decades. However, laser therapy is not always effective, may destroy normal retinal tissues and is often accompanied by side effects. Intravitreal steroids and vascular endothelial growth factor (VEGF) inhibitors have been developed for other ocular diseases. Clinical trials have shown that both therapies confer benefit to some patients with DME and CME, but many patients show only a partial response, and the therapies result in side effects. Because there is no FDA-approved drug for DME and CME, the development of effective therapies for these sight-threatening conditions is in demand.

Through a high-throughput screen of more than 200 small molecule compounds, we have identified CLT-003 with promising efficacy on DME. CLT-003NP is a promising drug candidate because it has new and multiple targets and can achieve a sustained efficacy on retinal vascular leakage. The ultimate goal of this project is to combine CLT-003 with nanotechnology to develop a new sustained-release drug for DME and CME.

Lead Collaborators

University of Oklahoma Health Sciences Center, Oklahoma City
Jian-xing Ma, M.D., Ph.D.

Ying Chen, M.D., Ph.D.

Public Health Impact

Currently, there is no FDA-approved pharmacotherapy for the treatment of diabetic retinopathy. The goal of this project is to combine nanotechnology and a novel promising small molecule drug candidate to develop a sustained-release drug treatment of diabetic retinopathy.


Work on this project is complete.

Project Details

  • Synthesis of Good Manufacturing Practice (GMP) and non-GMP material
  • Formulation development
  • Pharmacokinetic/absorption, distribution, metabolism, and excretion (PK/ADME) studies
  • Investigational New Drug (IND)-directed toxicology