Ocugen retinitis pigmentosa drug ocu100 obtained the FDA orphan drug qualification

Source: Ding Xiangyuan announced a license agreement with the University of Colorado on June 9, 2014, allowing ocugen to continue to develop two candidate drugs for the treatment of ophthalmic diseases, one of which is ocu100 (recombinant lens epithelial growth factor 1-326, ledgf1-326) for the rare ophthalmic disease retinitis pigmentosa (RP) The treatment has obtained the qualification of FDA orphan drug "The orphan drug qualification from the FDA orphan product development office is an important milestone, which will allow ocugen to accelerate the clinical development of ocu100, which may become the first drug approved for the treatment of retinitis pigmentosa," said musunuri, founder and chairman of ocugen's board of directors, Ph.D., and MBA Kompella, founder of ocugen science, is the inventor of ocu100 Ocugen licensed all ledgf related assets, including ledgf1-326 and ocu200, an angiostatin fusion protein that will be developed by the University of Colorado for the treatment of wet age-related macular degeneration (AMD) Retinitis pigmentosa is a rare ophthalmic disease caused by genetic mutations that cause retinal degeneration According to information provided by the American Foundation for the fight against blindness, the disease affects about 100000 people in the United States People with RP have a gradual decline in vision due to the death of photoreceptor cells in the retina It is a progressive disease and most RP patients are usually blind at the age of 40 At present, there is no FDA approved RP treatment drug "Ocu100, as a promising therapeutic drug, can reduce protein aggregation and related cell pressure It has shown potential for the treatment of retinitis pigmentosa It is known that protein aggregation and related cell pressure can cause this disease," Dr kompella said "Based on impressive preclinical data, we look forward to promoting phase 1 Research on this drug in patients in 2015 to observe its safety and tolerability." Dr kompella said various mutations, including the P23H mutation in rhodopsin, a key protein in the retina responsible for vision, were associated with RP P23H rhodopsin is known to form a large number of clusters or polymers in retinal cells, leading to cell pressure and eventually cell death Ocu100 has the ability to prevent protein aggregation and related pressure in retinal cells This drug has the potential to change the treatment of RP Original link: http://www.firstwordpharma.com/node/1215432? TSID = 28 & region? Id = 4? Axzz33jct1tvy