Noregen, a regenerative medicine ocular therapeutic

ABSTRACT Ischemic retinopathies, such as diabetic retinopathy and retinal vein occlusion, are the leading cause of blindness in the United States. Common features are leaky vessels, swelling, capillary drop out, aberrant vessel growth and retinal detachment. Current treatments include laser ablation, anti-VEGFs and steroids. All are sub-optimal and only serve to slow disease progression and vision loss, and do not repair or regenerate the damaged vessels. Laser treatment is used to ablate avascular/ischemic regions of the retina to reduce the hypoxic drive which causes the abnormal vascular permeability, exudation and neovascularization. Anti-VEGFs and steroids transiently reduce vascular permeability but do not aid in normal retinal blood vessel repair or regeneration. However, the current product, Noregen (a norrin derived growth factor) restores vessel integrity and regenerates new non- fenestrated capillaries. Proof of principal was established in the most widely employed animal model for ischemic retinopathies, the mouse Oxygen Induced Retinopathy (OIR) model. In this Phase II project, we will optimize dosing of intravitreal injections, create and qualify analytical methods that will be used to establish product release criteria, and run preliminary pharmacokinetics studies to support an FDA IND submission. The long term goal of this project is to create a novel therapy for ischemic retinopathies that will restore the retina rather than simply slow pathology and vision loss.