A Therapy for the Treatment of the Inherited Childhood Blinding Disorder Familial Exudative Vitreoretinopathy

We have made a class of novel drugs to treat the inherited childhood blinding disorder familial exudative vitreoretinopathy (FEVR). FEVR is characterized by lack of vascularization of the retina followed by secondary aberrant 'neo'-vascularization that can lead to retinal detachment and complete blindness. The current use of laser and surgery help reduce blindness caused by the second stage of FEVR or aberrant 'neo'-vascularization but do not treat the first stage of the disease which is the failure to develop retinal vasculature. In mouse models of FEVR, our novel drugs treat the first stage of FEVR and restore normal retina vascularization. This would translate to a lifetime of normal vision for FEVR patients. Moving this therapy into humans would be a breakthrough in the treatment of children born with FEVR.