AGTC is developing treatments for rare lung and eye diseases, offering hope to patients with unmet medical needs. 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.