Description
Applied Genetic Technologies Corp is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa, achromatopsia (ACHM CNGB3 and ACHM CNGA3), and X-linked retinoschisis. In addition to its clinical trials, company has preclinical programs in optogenetics, adrenoleukodystrophy, which is a disease of the central nervous system, other ophthalmology, and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight.
