Biologicals
OCU200
Currently, Ocugen is developing OCU400 for the treatment of multiple IRDs encompassing RP and LCA.
OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin, that are already present normally in retinal tissues. OCU200 unique features which we believe will enable it to (a) efficiently target leaky blood vessels, (b) regress the existing abnormal blood vessels, and (c) inhibit the growth of new blood vessels in the retina and choroid. Tumstatin, which acts as an anti-VEGF, anti-inflammatory and anti-oxidative agent, is the active component of OCU200. It binds to integrin receptors, which play a crucial role in disease pathogenesis. Transferrin facilitates the targeted delivery of tumstatin into the retina and choroid and potentially helps increase the interaction between tumstatin and integrin receptors.
In preclinical studies, OCU200 demonstrated efficacy in different animal models of neovascularization. In an animal model for DME and DR (Oxygen-induced retinopathy in mice), OCU200 demonstrated comparable efficacy at significantly lower dose compared to existing approved anti-VEGF therapy in preventing disease manifestation and progression. In animal models for wet AMD (laser induced CNV in mice and rats), OCU200 demonstrated superior activity compared to anti-VEGF control groups in preventing the formation and growth of new leaky blood vessels and subsequent disease symptoms.
OCU200 Demonstrated Efficacy in Animal Models for DME, DR, and Wet-AMD

OCU200 Publications
- Raquel Lima e Silva, Yogita Kanan, Adam C. Mirando, Jayoung Kim, Ron B. Shmueli, Valeria E. Lorenc, Seth D. Fortmann, Jason Sciamanna, Niranjan B. Pandey, Jordan J. Green, Aleksander S. Popel, Peter A. Campochiaro. Tyrosine kinase blocking collagen IV-derived peptide suppresses ocular neovascularization and vascular leakage. Sci Transl Med. 2017 Jan 18;9(373).
- Venugopal Gunda and Yakkanti A Sudhakar. Regulation of Tumor Angiogenesis and Choroidal Neovascularization by Endogenous Angioinhibitors. J Cancer Sci Ther.;5(12): 417-426.
- Maguire, MG et al. Five-Year Outcomes with Anti-Vascular Endothelial Growth Factor Treatment of Neovascular Age-Related Macular Degeneration: The Comparison of Age-Related Macular Degeneration Treatments Trials. Ophthalmology. 2016 Aug; 123(8):1751-1761. 2016 May 2.
- Patel, PJ et al. Aflibercept treatment for neovascular AMD beyond the first year: consensus recommendations by a UK expert roundtable panel, 2017 update. Clin Ophthalmol 11, 1957-1966, (2017).
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