FDA grant given for Phase II trial

Applied Genetics Technology Corporation (AGTC) has received a grant from the FDA (FDA Orphan Drugs Programme) to fund a Phase II Human Clinical Trial evaluating the safety and efficacy of a treatment for Leber congenital amaurosis (LCA), a genetic retinal disease.

"This funding will enable us to complete enrolment of the Phase 2 trial that is designed to deliver recombinant adeno-associated virus (rAAV) vectors to larger areas of the retina than in clinical studies performed to date," said Sue Washer, president and CEO of AGTC. "The ultimate goal of this research is to improve the sight and quality of life of patients affected by this disorder."

The clinical trial will be coordinated by AGTC and is being conducted at the University of Massachusetts Medical School by Dr Shalesh Kaushal and Oregon Health and Science University by Dr Tim Stout. Currently there is no treatment for LCA available.