应用基因科技公司

Applied Genetic Technologies Corporation is a clinical-stage biotechnology company. The Company develops gene therapy products designed to manage the lives of patients with severe diseases in ophthalmology. As of March 31, 2016, its product candidates included treatments for X-linked retinoschisis (XLRS), over two forms of achromatopsia (ACHM) and X-linked retinitis pigmentosa (XLRP). As of March 31, 2016, the Company was engaged in developing three discovery programs, targeting three indications based on its adeno-associated virus vector technologies. XLRS is an inherited retinal degenerative disease caused by mutations in the retinoschisis1 (RS1) gene. Achromatopsia is an inherited condition that is associated with visual acuity loss, extreme light sensitivity resulting in daytime blindness, and reduced or complete loss of color discrimination, achromatopsia is caused by mutations in any of several genes. The Company is also developing Alpha-1 Antitrypsin Deficiency (Alpha 1).