Ocugen - Modifier Gene Therapy Platform Cutting-Edge Technology

Ocugen’s modifier gene therapy platform is a cutting-edge technology licensed from the Schepens Eye Research Institute of Massachusetts Eye and Ear (Harvard Medical School) and involves targeted delivery and expression of one or more nuclear hormone receptor genes (NHRs) in the disease tissues.  NHRs play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, survival, and maintaining the cellular and molecular homeostatic various tissues, including the retina.  Multiple animal models have shown that expression of NHRs within the retina can stabilize retinal cells and suppress/prevent the development of inherited diseases, demonstrating their modifier function.