OCU100 is a biologic drug candidate that Ocugen is preparing to enter human clinical trials as a treatment for retinitis pigmentosa (RP), a form of degenerative eye disease that causes progressive vision impairment and blindness, usually by the time the individual reaches their 40s. RP is a relatively rare disease with no treatment options to slow or stop the cellular damage to the retina, which is why OCU100 was granted Orphan Drug Designation by the U.S. FDA and the European Medicine’s Agency (EMA) for this indication.
OCU100 is a stablized synthetic protein based on the ocular transcription factor lens epithelium-derived growth factor (LEDGF), which is naturally expressed in the retina and broadly promotes cellular survival in response to cellular stress. Treatment with OCU100 has been shown to rescue photoreceptor function and morphology in well-established models of retinal degeneration, which means OCU100 may have a protective effect for a variety of retinal degenerative disorders in addition to RP. Follow-on indications may include geographic atrophy (advanced dry-AMD), and orphan diseases Leber’s congenital amaurosis and Stargardt’s disease.
Ocugen is completing preclinical studies and expects to begin a Phase 1 clinical trial in RP patients in 2018. OCU100 may also be eligible for FDA Fast Track and Breakthrough status, which may accelerate its pathway to potential approval for treating RP.
About Retinitis Pigmentosa
RP is a degenerative eye disease with multiple primary and secondary causes. Inherited genetic mutations to one of more than 70 different genes have been linked to RP, making it the most common form of inherited retinal degeneration and blindness in the U.S. (~100,000 U.S. patients; 1.5 million globally). RP can be diagnosed as early as infancy and the disease is characterized by the progressive impairment and loss of photoreceptor cells in the retina. As different types of photoreceptors and other retinal cells are affected, patients may experience decreased night vision, photophobia, blurred vision, progressive “tunnel” vision, and eventually complete blindness. There are currently no approved therapies for RP, highlighting the medical need for new therapies.
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