GenSight Biologics � GS030 (RP)

GS030 is a unique combination of two parts. The first is a gene therapy that includes a gene for a light-sensitive protein. It’s delivered using a specially modified virus called AAV2 7m8. The second part is a pair of special goggles that stimulate the cells in the retina that have been altered by the gene therapy. The goggles project images onto your retina using a specific type of light. This approach, called optogenetics, involves inserting a gene for a light-sensitive protein into cells, which makes these cells respond to light. GS030 uses a bio-engineered virus to put the gene of this light-sensitive protein into the target cells in the retina, known as retinal ganglion cells (RGCs). This makes these cells sensitive to light, regardless of the genetic mutation that caused their initial damage. However, these altered cells aren’t as light sensitive as normal cells in the retina, so regular vision in normal daylight probably won’t be possible without the special goggles. These goggles work by capturing visual information and boosting the light signal to the altered cells in the retina, allowing them to function more like normal cells. GS030 is being tested as a treatment for retinitis pigmentosa