GS030 is an innovative combination of two complementary components:
- A gene therapy product encoding a photoactivatable channelrhodopsin protein, delivered via a modified AAV2 vector known as AAV2 7m8;
- Biomimetic goggles that stimulate the engineered retinal cells. Images are projected onto the retina by a light source that uses a specific wavelength.
GS030 uses optogenetics, a biologic technique that involves the transfer of a gene that encodes for a light-sensitive protein, which in turn causes neuronal cells to respond to light stimulation. GS030 includes a bio-engineered AAV2 gene therapy vector that introduces the gene of a photosensitive protein (to which we have exclusive rights in optogenetics) into the nucleus of the target cells, in our case the retinal ganglion cells, or RGCs. This gene confers a photoreceptive function to healthy and preserved RGCs, independent of any specific underlying genetic mutation that caused the photoreceptor degeneration.
However, because cells expressing optogenetic proteins are less light sensitive than normal photoreceptors, vision under regular daylight conditions is unlikely to be possible without further intervention. GS030 also features biomimetic goggles, which mimic the normal retinal activity by capturing visual information and amplifying the light stimulation upon the transduced neuronal cells at the appropriate wavelength.
We plan to investigating the potential for GS030 to treat GA in Dry-AMD after the PIONEER study has been concluded.