RTP-801i

RTP-801i is an siRNA drug candidate designed to inhibit the expression of the hypoxia-inducible gene RTP-801. The gene RTP-801 was discovered by Quark and initially identified by its dramatic up-regulation in response to hypoxia and/or oxidative stress both in vitro and in animal models. Since expression of RTP-801 is rapidly upregulated in response to ischemia, hypoxia and/or oxidative stress, it represents a unique gene target that may regulate hypoxia-induced pathogenesis by a mechanism that is independent of growth factors such as VEGF. This hypoxia-inducible gene promotes neuronal cell apoptosis and the generation of reactive oxygen species in vitro. In both genetic (RTP801-knockout) and therapeutic mouse and primate models of laser-induced choroidal neovascularization (CNV), inhibition of RTP801 expression leads to inhibition or reduction of CNV and vessel leakage following intravitreal injection of RTP-801i.