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AGTC is developing cures 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.

AGTC has five programs in development from pre-clinical through Phase 2


An inherited condition that is associated with visual acuity loss, extreme light sensitivity resulting in daytime blindness, and total loss of color discrimination.

Age-Related Macular Degeneration

The leading cause of blindness in the U.S. which leads to loss of central vision, effectively blinding a patient.

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.

Leber Congenital Amaurosis

A rare inherited retinal degenerative disease characterized by severe loss of vision early in life.

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.

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