OCU100 is a recombinant form of Lens Epithelial Derived Growth Factor (LEDGF) which has been shown to rescue photoreceptor function and morphology in well-established models of retinal degeneration (Baid, R, Upadhyay AK, Shinohara, T, Kompella UB. Biosynthesis, characterization, and efficacy in retinal degenerative diseases of lens epithelial derived growth factor fragment (LEDGF1-326), a novel therapeutic protein. J Biol Chem. 2013 Jun 14;288(24):17372-83). LEDGF is an endogenous ocular transcription factor that enhances cell survival. LEDGF was originally isolated by researchers at the National Eye Institute, and has been shown to enhance photoreceptor and retinal pigment epithelial cell survival (Shinohara, T., Singh, D.P., and Fatma,N. LEDGF, a survival factor, activates stress-related genes. Prog Retin Eye Res. 2002;21(3):341-358). Ocugen is also investigating LEDGF’s regenerative potential.
In May 2014, Ocugen was granted Orphan Drug Designation from the FDA for the use of OCU100 as a treatment for Retinitis Pigmentosa. Products with Orphan Drug Designation benefit in a number of ways including:
- Seven years of market exclusivity
- Protocol assistance
- Tax credits of up to 50% of R&D costs
- FDA fee waivers and research grants
In addition, Ocugen met with FDA in a Pre IND meeting and aligned on a number of important pre-clinical toxicology, manufacturing, and clinical development plans.
- Baid,R., Scheinman,R.I., Shinohara,T., Singh,D.P., Kompella,U.B. (2011) LEDGF1–326 decreases P23H and wild type rhodopsin aggregates and P23H rhodopsin mediated cell damage in human retinal pigment epithelialcells.PLoSOne6,e24616
- Baid R, Upadhyay AK, Shinohara T, Kompella UB., Biosynthesis, characterization, and efficacy in retinal degenerative diseases of lens epithelium-derived growth factor fragment (LEDGF1-326), a novel therapeutic protein. J Biol Chem. 2013 Jun 14;288(24):17372-83.
- Shinohara, T., Singh, D. P., and Fatma, N.,2002. LEDGF, a survival factor, activates stress-related genes. Prog Retin Eye Res, Vol. 21, 3, pp. 341-358.
- Matsui, H., Lin, L. R., Singh, D. P., Shinohara, T., and Reddy, V. N.,2001. Lens epithelium-derived growth factor: increased survival and decreased DNA breakage of human RPE cells induced by oxidative stress. Invest Ophthalmol Vis Sci, Vol. 42, 12, pp. 2935-2941.
- Machida, S., Chaudhry, P., Shinohara, T., Singh, D. P., Reddy, V. N., Chylack, L. T., Jr., Sieving, P. A., and Bush, R. A.,2001. Lens epithelium-derived growth factor promotes photoreceptor survival in light-damaged and RCS rats. Invest Ophthalmol Vis Sci, Vol. 42, 5, pp. 1087-1095.
- Raz-Prag, D., Zeng, Y., Sieving, P. A., and Bush, R. A.,2009. Photoreceptor protection by adeno-associated virus-mediated LEDGF expression in the RCS rat model of retinal degeneration: probing the mechanism. Invest Ophthalmol Vis Sci, Vol. 50, 8, pp. 3897-3906.