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ZVS203e is an investigational gene editing therapy designed for the treatment of retinitis pigmentosa (RP) caused by site-specific mutations in the RHO gene. It is administered via subretinal injection and utilizes a gene transfer approach to target and correct pathogenic mutations in the RHO gene, which are a common cause of autosomal dominant RP. The drug is currently being evaluated in a Phase 1 clinical trial to assess its safety, tolerability, and efficacy in adult patients with genetically confirmed RHO-related RP. The study involves single ascending doses and also monitors vector shedding characteristics.
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