Our pipeline currently comprises two lead product candidates for the treatment of retinal degenerative diseases that lead to blindness, together with products in preclinical development targeting ophthalmic and neurodegenerative diseases. Below is a table summarizing our development programs:
Our lead product candidate, GS010, aims at treating LHON, a rare mitochondrial genetic disease associated with mutation in the ND4 gene. GS010 is based on our first core technology platform, our MTS, which permits missing mitochondrial proteins to be shuttled into the mitochondrion, thereby aiming to restore mitochondrial function. There is currently no approved treatment to prevent loss of sight and/or restore vision in LHON patients. We believe that GS010 will deliver the first treatment for LHON, addressing a significant unmet medical need.
Our second product candidate, GS030, is based on optogenetics, a technology that makes cells responsive to light by using a viral vector (an engineered AAV2) to introduce a DNA sequence that encodes a photosensitive protein belonging to channelrhodposin (ChR) family, into the nucleus of the target cells. Once this protein is expressed, it confers a photoreceptor-like function to the target cell, hence enabling restoration of vision in patients with extremely reduced vision or total blindness due to RP.