According to a story from BioPortfolio, the biopharmaceutical company GenSight Biologics recently announced that the first patient has received treatment in a Phase I/II clinical trial testing the company’s experimental medical product GS030. This product is in development for the treatment of retinitis pigmentosa. GenSight specializes in the development of gene therapy treatments for central nervous system disorders and retinal neurodegenerative conditions.
About Retinitis Pigmentosa
Retinitis pigmentosa is a genetic disorder that affects vision. This disease can progress to total blindness, although this is rare. This disorder can appear on its own or as a complication alongside other disorders or systemic illnesses. Retinitis pigmentosa is usually inherited from the patient’s parents, and a number of genetic mutations can cause it to occur. Symptoms of this disorder include generalized vision loss, difficulty adjusting to lighting conditions, aversion to bright light, night blindness, tunnel vision, and blurred vision. There is a serious lack of treatment options for the condition, and there are no therapies that can definitively delay, halt, or reverse the decline in visual acuity. A retinal prosthesis, or bionic eye, is available in some places and can help improve vision to a limited degree. Gene therapy could be a viable option in the future. To learn more about retinitis pigmentosa, click here.
GS030 is an unique treatment technology that combines two different components with the goal of restoring vision to retinitis pigmentosa patients. The first component is a gene therapy, which introduces genes coding for a protein that is light sensitive to certain cells in the retina, via a one time injection. This is then enhanced with a wearable medical technology that serves to stimulate the altered cells to increase their light sensitivity further. The company hopes that GS030 will also have utility in other retinal diseases as well. The system is rooted in prior work from GenSight such as their Optogenetics technology.
This clinical trial will include multiple cohorts of patients. These different cohorts will receive various doses of the gene therapy and will include patients with a broad range of disease severity, including those who are almost completely blind and patients with less severe vision loss.