AGTC

With a highly specialized team of physicians and researchers, we use cutting-edge techniques to develop treatments for patients that have diseases caused by broken genes. We use gene therapy, which replaces those broken genes with normal functional genes, allowing a patient’s own body to produce proteins to treat their illness. A single injection provides long-lasting treatment, leading to a better quality of life for patients worldwide. The innovative delivery method AGTC uses is the non-toxic adeno-associated virus (AAV), a safe virus that delivers healthy copies of the gene, replacing defective copies. AAV is an ideal delivery method because: It is safe, having never been shown to cause disease. It is effective and provides long-lasting benefit. Its production is fully scalable and does not require animal-derived products. It has been approved for use in human clinical trials by U.S. and European regulatory agencies. AGTC has three ongoing ophthalmology development programs and proof-of-concept data in multiple indications. X-Linked Retinoschisis An inherited form of retinal degeneration affecting young males, presenting with poor vision by school age. Visual acuity usually worsens during the teenage years and then can lead to serious complications such as vitreous hemorrhage or retinal detachment during adulthood. Achromatopsia An inherited condition that is associated with visual acuity loss, extreme light sensitivity resulting in daytime blindness, and total loss of color discrimination. X-Linked Retinitis Pigmentosa An inherited condition that causes boys to develop night blindness by the time they are ten and progresses to legal blindness by their early forties. Alpha-1 Antitrypsin Deficiency One of the most common hereditary disorders in the world and the most commonly known genetic risk factor for emphysema, Alpha-1 can also cause liver disease.