First IND Application Filed for RNAi-Based Drug

This is the first IND filed to take an RNAi-based drug into clinical trials.

The drug, called Cand5, is a small interfering RNA (siRNA) that uses RNAi to shut down genes that promote the overgrowth of blood vessels that lead to vision loss in wet AMD, a leading cause of adult blindness estimated to affect more than two million people in the U.S. alone by 2010.

Specifically, Cand5 shuts down the production of vascular endothelial growth factor (VEGF), which has been shown to be the central stimulus in the development of wet AMD as well as diabetic retinopathy, another leading cause of blindness. The efficacy of Cand5 in reducing new blood vessel growth and leakage that cause AMD has been demonstrated in primates and rodents. Its novel siRNA mechanism of action is expected to provide efficacy, safety, and administration advantages compared to other compounds for the same condition. Cand5 was developed by Acuity Pharmaceuticals (Philadelphia, PA, USA).

"Treatments based on harnessing RNAi to shut down production of VEGF may offer an entirely new approach to treating AMD, so the progress of Acuity's Cand5 towards human clinical studies is an important milestone for the millions of individuals at risk for vision loss from these incurable conditions,” noted Lawrence Yannuzzi, M.D., clinical professor of ophthalmology at Columbia University College of Physicians and Surgeons (New York, NY, USA) and director of retinal services and research at the Manhattan Eye, Ear, and Throat Hospital (New York, NY, USA).




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