GenSight Biologics is developing GS010, a therapy that causes ND4, a mitochondrial protein, to be expressed in the nucleus and shuttled to the mitochondria. This concept of moving mitochondrial DNA into the nucleus, where it is better protected, is a key approach for combating mitochondrial dysfunction, one of the hallmarks of aging. The company has taken GS010 into Phase 3 clinical trials for the treatment of Leber Hereditary Optic Neuropathy (LHON), a disease that causes blindness.
This company has had some issues with clinical trials in the past, as each patient had a single treated eye to test the efficacy of GS010 and the other eye was left untreated to serve as the control group. The problem was that administering this therapy seemed to affect the untreated eye along with the treated eye, leading to a lack of difference between the experimental group and the control group – thus possibly making an effective therapy seem ineffective.
GenSight is also developing GS030, a gene therapy that allows retinal cells to detect the presence of light.