Press release
Ocugen, Inc. Announces U.S. FDA Acceptance of Investigational New Drug Application to Initiate a Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration
Gene therapy candidate has potential to address a large number of retinitis pigmentosa and Leber congenital amaurosis gene mutations with a single
About this update from Ocugen, Inc.
[{"type":"text","content":"Gene therapy candidate has potential to address a large number of retinitis pigmentosa and Leber congenital amaurosis gene mutations with a single productTrial to start in Q1 2022 will enroll patients with mutations in NR2E3 or RHO genes MALVERN, Pa., Dec. 09, 2021 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to fight COVID-19, announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug application (IND) to initiate a first-in-human clinical trial of OCU400 (AAV-NR2E3), a modifier gene therapy candidate for the treatment of retinitis pigmentosa resulting from genetic mutations found in NR2E3 and Rhodopsin. “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,” said Shankar Musunuri, PhD, MBA, Chairman of the Board, Chief Executive Officer, and Co-Founder of Ocugen. “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.” Ocugen’s modifier gene therapy platform aims to target nuclear hormone receptors (NHRs) that regulate multiple functions within the retina, giving it the potential to address many different gene mutations – and, in turn, multiple retinal diseases – with a single product. Traditional gene therapy, which transfers a functional version of a non-functional gene into target cells, targets only one individual gene mutation at a time. OCU400 was granted four orphan drug disease designations from the FDA for treating four different gene mutation-associated retinal degenerative diseases between 2019 and 2020. The European Medicines Agency (EMA) granted Ocugen broad orphan medicinal product designation in 2021 for OCU400 for the treatment of both retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) – meaning that, if approved, OCU400 by itself could treat these diseases that are rooted in mutations of more than 175 different genes. “Ocugen’s game-changing approach to gene therapy could provide mutation ag...