Ocugen Inc. (NASDAQ: OCGN) has announced that the FDA has cleared its Investigational New Drug application to commence the first-in-human study of OCU400, a modifier gene treatment candidate for retinitis pigementosa treatment due to genetic alterations found in Rhodopsin and NR2E3.
Ocugen partnering with eye care centers to advance OCU400
Board Chairman, CEO, and Co-Founder of Ocugen Shankar Musunuri said, “We are delighted to advance OCU400 into clinical trials, which exemplifies our goal of offering new options to people with genetic diseases where none currently exist. We’re collaborating with leading centers in eye care and have been vital partners to getting our trial launched and receive patients. With this final decision by the FDA, we are embarking on a new pathway of care through this innovative gene therapy.”
The Company’s modifier gene therapy platform targets nuclear hormone receptors (NHRs), which govern various processes in the retina, allowing it to treat multiple gene mutations. As a result, it is possible to treat multiple retinal illnesses with a single product. Conventional gene therapy, in which a functional form of a non-functional gene is transferred into target cells, focuses on a single gene mutation at ago.
OCU400 has potential of treatiung RP and LCA
Between 2019 and 2020, the FDA granted OCU400 four orphan drug disease designations for treating four separate gene mutation-associated retinal degenerative disorders. In 2021, the European Medicines Agency (EMA) granted Ocugen broad orphan medicinal product designation for OCU400 for the treatment of both Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP), indicating that once cleared, OCU400 could treat these diseases that are caused by mutations in over 175 genes. The novel therapy candidate seeks to offer better treatment alternatives to underserved diseases.
Professor of Ophthalmology and Chief of the Paul H Casey Ophthalmic Genetic Division, Oregon Health & Science University, and Ocugen’s Retina Scientific Advisory Board member Mark Pennesi said, “Ocugen’s game-changing approach to gene therapy could provide mutation agnostic therapies that raise the bar on how we could treat genetic diseases in the future.”