NEW YORK – The US Food and Drug Administration has cleared Skyline Therapeutics' investigational new drug application to begin human trials of a novel eye disease treatment, the gene therapy company said Monday.
Skyline is developing SKG0106, an adeno-associated virus vector-based gene therapy, for neovascular age-related macular degeneration, a progressive ocular disease that causes vision impairment. The vascular endothelial growth factor (VEGF) protein has been implicated in causing the formation of abnormal blood vessels in the retina and increased VEGF expression has been observed in patients with neovascular and non-neovascular age-related macular degeneration.
Skyline's one-time, intravitreally delivered therapy is designed to deliver a transgene that encodes for anti-VEGF proteins, which, based on preclinical studies, the company expects to suppress VEGF and inhibit intraocular neovascularization.
Now that the FDA has approved its IND application, Shanghai- and Boston-based Skyline said it will soon begin a global Phase I/IIa clinical trial of SKG0106.