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GS030
- Development stage
- Phase 2
- Lead developer
- GenSight Biologics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravitreal
01
Overview
**GS030** is an investigational optogenetic gene therapy developed to restore vision in patients with retinitis pigmentosa (RP). It combines an adeno-associated virus vector (AAV2) encoding the light-sensitive protein ChrimsonR fused to tdTomato, which is delivered via intravitreal injection to retinal ganglion cells, making them responsive to light even after photoreceptor degeneration[1][4][5]. The therapy requires the use of a proprietary wearable device (GS030-MD), which projects specific wavelengths and intensities of light onto the treated retina, activating the introduced opsin and enabling visual perception[4][5][7]. This approach is mutation-independent and aims to provide functional vision for patients with advanced RP who have lost photoreceptor function[2][3].
- Other names
- AAV2.7m8-ChrimsonR-tdTomatoAAV-2.7m8-ChrimsonR-tdTomatoAAV 2.7m8-ChrimsonR-tdTomatoChrimsonR-tdTrAAV2.7m8-ChrimsonR-tdTrAAV-2.7m8-ChrimsonR-tdTrAAV 2.7m8-ChrimsonR-tdT
02
Targets
ChrimsonRRHO (Rhodopsin)
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