Ocugen Inc. (NASDAQ: OCGN) has submitted an Investigational New Drug application with the FDA to commence a Phase 1/2 clinical study of its modifier gene therapy candidate, OCU400 (AAV-NR2E3), in treating retinitis pigmentosa that results from NR2E3 and RHO gene mutation.
Ocwen to commence study on OCU400 in retina pigmentosa treatment
CEO and co-founder of Ocugen, Dr. Shankar Musunuri, said, “This important milestone brings us one step closer towards achieving our company’s vision of fostering a future where no one feels hopeless in the face of disease. Our novel modifier gene therapy platform is a strong example of our approach to drug development.”
The company’s modifier gene therapy platform targets nuclear hormone receptors (NHRs) which regulate various processes in the retina, allowing it to treat multiple gene mutations and thus multiple retinal illnesses using a single treatment. Conventional gene therapy in which the functional gene forms of a non-functional gene are transferred into target cells focuses on a single gene mutation.
Ocugen VP and Research and Development Head Atun Upadhyay said, “The goal with OCU400 (AAV-NR2E3) is to offer people living with genetic vision conditions – people whose vision is slowly deteriorating and who have no current treatment options – a reason to hope. And we’re very pleased to be on this journey with the creator of our modifier gene therapy platform, Dr. Neena Haider from Harvard Medical School. Collaboration is a mother of innovation, and our work with Dr. Haider is really driving us to see a whole new potential for care.”
OCU400 has four orphan designations
The FDA granted OCU400 four orphan drug disease designations between 2019 and 2020 for treating four separate gene mutation-related retinal degenerative disorders. In addition, the company received broad orphan medicinal product designation for its modifier gene therapy from the European Medicines Agency (EMA) in 2021 for the treatment of both Leber Congenital amaurosis (LCA) and retinitis pigmentosa (RP). Therefore, this means that once approved, the therapy will be vital in the treatment of these diseases that are caused by mutations in over 175 different genes.
