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Ocugen and University of Colorado Report Orphan Designation for OCU100

June 10, 2014

Ocugen and the University of Colorado reported license agreements that allow for Ocugen to continue developing two drug candidates for the treatment for ophthalmology indications, and that one of the assets, OCU100, recombinant lens epithelium derived growth factor 1- 326 (LEDGF1-326), received orphan-drug status from the U.S. Food and Drug Administration for treatment of retinitis pigmentosa, a rare eye disease.

"Orphan drug designation from the FDA'sOffice of Orphan Products Development is a significant milestone that will allow Ocugen to accelerate the clinical development of OCU100, which has the potential to be the first approved therapeutic for retinitis pigmentosa," said Shankar Musunuri, founder and chairman of the Ocugen Board of Directors, in a release.

Ocugen scientific founder and board member Uday Kompella, a professor of Pharmaceutical Sciences, Ophthalmology and Bioengineering at CU's Anschutz Medical Campus, is the inventor of OCU100.

Ocugen licensed all assets related to LEDGF, including LEDGF1- 326 and OCU200, an anti-angiogenic tumstatin fusion protein, to be developed for treatment of wet age-related macular degeneration from the University of Colorado in March.

"OCU100 has shown the potential as a promising therapeutic agent for treating retinitis pigmentosa by reducing protein aggregation and associated cellular stresses, which are known to contribute to this condition," said Kompella. "With impressive preclinical data, we look forward to progressing with a phase 1 study for safety and tolerability in patients sometime in 2015."

The Company said FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S.

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Source: Professional Services Close - Up

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