AGTC abandoned the Leber Congenital Amaurosis program for business reasons, according to the S-1/A filing prior to the Mar 2014 IPO: http://www.sec.gov/Archives/edgar/data/1273636/000119312514094588/d615962ds1a.htm (p.96) …we do not plan to conduct additional clinical trials with this product candidate, as we believe the small number of persons affected by the RPE65 form of LCA2, which we estimate at approximately 600 in the United States and Europe combined, are being adequately served by ongoing and planned clinical trials conducted by multiple academic research centers in the United States and several European countries. I recommend the Dec 2014 Piper Jaffray webcast, which is accessible at http://www.media-server.com/m/p/84gjky9f .