Gene Therapy Platform Developed by Neena Haider, PhD, Granted FDA Orphan Drug Designation

OCU400, a novel gene therapy platform developed by Neena Haider, PhD, Harvard Medical School Associate Professor of Ophthalmology and Associate Scientist at the Schepens Eye Research Institute of Mass. Eye and Ear, and licensed to biopharmaceutical company Ocugen, Inc., has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

The gene therapy has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited degenerative retinal disease. Consisting of a functional copy of the nuclear hormone receptor (NHR) gene NR2E3, OCU400 is delivered to target cells in the retina using an adeno-associated viral (AAV) vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, stabilizing cells and potentially rescuing photoreceptor degeneration. Ocugen plans to initiate a Phase 1/2a clinical study for OCU400 by 2020.