Restore Vision is developing a gene therapy specifically targeting retinitis pigmentosa, a genetic disorder that leads to progressive vision loss. This therapy aims to restore visual function by addressing the underlying genetic causes of the disease.
Funding
$15.3M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Retinitis pigmentosa (RP) is a genetic disorder causing progressive vision loss due to the degeneration of photoreceptor cells in the retina. Current treatments offer limited efficacy in halting or reversing the progression of RP, leaving a significant unmet need for therapies that address the underlying genetic causes of the disease.
Solution
Restore Vision is developing a gene therapy designed to restore visual function in patients with retinitis pigmentosa. The therapy targets the genetic mutations responsible for photoreceptor cell dysfunction and death, aiming to deliver functional genes to the affected cells. By correcting the underlying genetic defect, the treatment seeks to prevent further vision loss and potentially restore some degree of visual acuity. The approach involves delivering a therapeutic gene using a viral vector to transduce retinal cells and promote long-term expression of the corrected gene.
Target Audience
The primary target audience includes individuals diagnosed with retinitis pigmentosa who carry specific genetic mutations addressable by the gene therapy, as well as ophthalmologists and retinal specialists involved in their care.
Features
- AAV-based gene therapy vector for efficient delivery of therapeutic genes to retinal cells.
- Targeted gene correction to address specific mutations associated with retinitis pigmentosa.
- Preclinical studies demonstrating improved photoreceptor cell survival and function.
- Clinical trial program to evaluate safety and efficacy in RP patients.