1. Why Gene Therapy in the Eye?
The eye is one of the most promising targets for gene therapy. It's a small, self-contained organ, which allows precise and localized delivery of therapeutic genes. This makes it possible to act directly at the root of the disease, with minimal systemic effects.
Thanks to AAV-based vectors, a single injection can deliver a functional gene to the retina, offering long-term—or even permanent—benefits. Gene therapy has already shown encouraging results in halting or slowing down vision loss in severe inherited conditions like Leber Congenital Amaurosis (LCA).
2. Why Use Retinal Transcription Factors?
Rather than correcting one mutation at a time, we focus on retinal transcription factors—the proteins that orchestrate the genetic activity of photoreceptors.
These "genetic conductors" regulate the development, function, and survival of retinal cells. By targeting them, we aim to reactivate entire gene networks that are essential for vision.
3. Why We Focus on the CRX Gene
Our therapeutic candidate is based on CRX, one of the most critical transcription factors in the retina. CRX acts as a master switch, controlling the genetic programs that make photoreceptors function—and survive.
CRX is essential for:
- Building the structure of rods and cones
- Driving phototransduction, the process that converts light into visual signals
- Coordinating the maturation and maintenance of photoreceptors over time
At Variant, we use gene therapy to deliver a healthy version of CRX, with the goal of restoring balance to the retinal gene network. By doing so, we don’t just replace a missing component—we reboot the entire system at its source.