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DVC1-0101 is an investigational gene therapy developed by ID Pharma (a subsidiary of I'rom Group) in collaboration with the Japan Agency for Medical Research and Development (AMED) for the treatment of retinitis pigmentosa. The therapy utilizes a simian immunodeficiency virus (SIV)-based lentiviral vector to deliver the human pigment epithelium-derived factor (hPEDF) gene directly to retinal cells via subretinal injection. PEDF is a potent endogenous neurotrophic and anti-angiogenic factor that plays a critical role in protecting retinal neurons. By providing sustained expression of hPEDF, DVC1-0101 aims to prevent the apoptosis of photoreceptor cells, thereby slowing or halting the progressive vision loss characteristic of retinitis pigmentosa. The use of an SIV-based vector is intended to provide efficient gene transfer and long-term expression with a favorable safety profile compared to other viral delivery systems.
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