Applied Genetic Technologies Corporation (AGTC) is a clinical-stage biotechnology company that focuses on developing gene therapies to treat rare and debilitating diseases. The company's lead product candidates include treatments for X-linked retinitis pigmentosa (XLRP) and achromatopsia, which are both rare genetic diseases that cause severe vision loss or blindness.
AGTC's gene therapy approach involves using a modified viral vector to deliver a healthy copy of the defective gene to the patient's cells. The company has developed a proprietary AAV vector platform that it believes can provide superior gene delivery and expression compared to other vectors currently in use.
In addition to its lead product candidates, AGTC has a pipeline of potential gene therapies for other rare diseases, including a treatment for Alpha-1 antitrypsin deficiency (AATD), a genetic disorder that affects the lungs and liver.
AGTC was founded in 1999 and is headquartered in Gainesville, Florida. The company has partnerships with biopharmaceutical companies, academic institutions, and other organizations to advance its research and development efforts.
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