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AGTC Files Investigational New Drug Application for the Treatment of X-Linked Retinoschisis

2.6 from 10 votes
Tuesday, March 10, 2015

Applied Genetic Technologies Corporation (Nasdaq:AGTC), a clinical stage biotechnology company developing adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, announced that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to conduct a Phase I/II clinical trial of the company's gene therapy product candidate for the treatment of X-linked retinoschisis (XLRS). Utilizing technology licensed from the University of Florida, AGTC's XLRS product candidate uses an AAV capsid with surface residues that have been specifically engineered for better penetration to the back of the eye.

"This filing is a significant milestone for AGTC and represents an opportunity to further explore the clinical utility of our novel AAV-based gene therapy for the treatment of XLRS," said Sue Washer, President and CEO. "Following favorable preclinical study results, we are excited to advance our mission to introduce new treatment options for patients with critical unmet medical needs."

Pending the FDA's acceptance of the IND application, the Company plans to initiate a clinical study evaluating the safety and efficacy of AGTC's proprietary gene therapy for treating XLRS during the second quarter of 2015 and expects to have initial data during the second half of 2015.

XLRS is an inherited retinal disease caused by mutations in the RS1 gene, which encodes the retinoschisin protein. XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual acuity in young boys, which can worsen as the patients age. There are currently no approved treatments for XLRS.

About AGTC

AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases in ophthalmology. AGTC's lead product candidates focus on X-linked retinoschisis, achromatopsia and X-linked retinitis pigmentosa, which are inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC is also pursuing pre-clinical development of treatments for wet AMD using the company's experience in ophthalmology to expand into disease indications with larger markets.


David Carey
Lazar Partners Ltd.
Tel: (212) 867-1768


Larry Bullock, Chief Financial Officer
Applied Genetic Technologies Corporation
Tel: (386) 462-2204

Source: Applied Genetic Technologies Corporation
2.6 from 10 votes
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