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; and
- 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 believe that in the promise of our optogenetics platform for treating RP and restoring patients’ visual function. Our preclinical proof-of-concept studies have demonstrated that GS030 can restore light sensitivity in the retina of blind mice and non-human primates. In other preclinical studies, the use of GS030 has restored visual behaviors in vivo in blind rats, with demonstrable effects upon their visual cortex.
Studies being initiated
We have initiated the PIONEER study, a dose-escalation study to evaluate the safety and tolerability of GS030 in subjects with Retinitis Pigmentosa.
Expanded access / Compassionate use
In some circumstances, a patient may not be able to participate in a clinical trial. Seeking use of an investigational medication under these circumstances is permitted by the FDA and commonly referred to as compassionate use or expanded access. Our policy is intended to comply with U.S. Food and Drug Administration (FDA) requirements for any such use or access. Please see the attached document for details.
Scientific publicationPartial recovery of visual function in a blind patient after optogenetic therapy. Sahel J.A. et al., Nature Medicine, May 2021
Scientific publicationOptogenetic therapy: high spatiotemporal resolution and pattern discrimination compatible with vision restoration in non-human primates. Gauvain G. et al., Communications Biology, Feb. 2021