Applied Genetic Technologies Corporation (NASDAQ: (AGTC) is a clinical-stage biotechnology company developing transformational genetic therapies for patients suffering from rare and debilitating diseases with a partcular focus on the field of ophthalmology Gene therapy replaces broken genes with normal functional genes, allowing a patients own body to produce proteins to treat their illness. A single treatment provides long-lasting benefit sometimes even for a lifetime. The firm's lead product candidates in the preclinical stage include treatments for X-linked retinoschisis, Achromatopsia, and X-linked retinitis pigmentosa, which are diseases of the eye caused by mutations in single genes. The company has also completed preclinical proof-of-concept studies, and Phase I and Phase II clinical trials of a treatment for alpha-1 antitrypsin deficiency, an inherited orphan lung disease. In addition, it has proof-of-concept programs for other eye diseases, such as leber congenital amaurosis (type 2), and wet form of age-related macular degeneration. AGTC's active programs include inherited retinal disorders (achromatopsia, X-linked retinoschisis, and Leber's congenital amaurosis), acute macular degeneration (licensed to Genzyme/Sanofi) and alpha-1-antitrypsin deficiency.The firm has clinical programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), and achromatopsia (ACHM), as well as a preclinical program in optogenetics. The company also has initiated preclinical programs in adrenoleukodystrophy and otology indications. Applied Genetic Technologies Corporation has collaboration agreements with Synpromics Limited; Bionic Sight, LLC; and Otonomy, Inc.
