DVC1-0401

DVC1-0401 is an investigational gene therapy designed for the treatment of retinitis pigmentosa (RP). It utilizes a recombinant, non-integrating Sendai virus (SeV) vector to deliver the human pigment epithelium-derived factor (hPEDF) gene directly to retinal cells. PEDF is a potent neurotrophic factor that has been shown to protect photoreceptors and retinal pigment epithelial cells from apoptosis and oxidative stress, which are key drivers of vision loss in RP. The Sendai virus platform, developed by ID Pharma (a subsidiary of I'rom Group), is characterized by its ability to achieve high levels of transgene expression within the cytoplasm without the risk of genomic integration. The therapy is administered via subretinal injection, aiming to provide a sustained supply of PEDF to the degenerate retina to slow or halt disease progression.

Other names

SeV-hPEDFSendai virus-human pigment epithelium-derived factor