ViGeneron’s pipeline in gene therapy addresses ophthalmic diseases with high unmet medical need, including two lead programs in development for inherited retinal diseases where no approved treatment options are currently available.
For more information about the indications we are working on and the therapies our products offer, please click on the individual indication.
Retinitis pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. There are currently no approved treatments for RP. RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnel vision,” central visual impairment, reduced visual acuity and, ultimately, complete blindness. Retinitis pigmentosa is the most common type of inherited retinal diseases (IRDs). It is estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively.
More than 250 different genes are linked to blinding conditions which are summarized as inherited retinal diseases (IRDs). Some patients living with IRDs experience a gradual loss of vision, eventually leading to complete blindness. Others may experience vision loss in infancy or early childhood or born blind. Obtaining a confirmed genetic diagnosis through genetic testing is the only way to verify the exact gene mutation that is the underlying cause of an IRD.
Age-related macular degeneration is the major cause of irreversible vision loss among elderly and affects currently 30 million individuals worldwide. The disease can manifest in a dry (atrophic) and wet (neovascular) form. AMD affects a small area near the center of the retina, the macula, which is responsible for central vision and is needed for detailed tasks such as reading, driving, and recognizing faces. Peripheral vision and night vision are generally not affected in AMD, but slow adjustment of vision to darkness and reduced dim light vision often occur in the early stages of the disease.