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Ocugen, Inc. Announces Submission of Investigational New Drug Application with U.S. FDA to Initiate a Phase 1/2 Clinical Trial Evaluating Gene Therapy Candidate OCU400 (AAV-NR2E3) to Treat Inherited Retinal Degeneration
Modifier gene therapy platform has potential to treat multiple retinal diseases with one product MALVERN, Pa., Nov. 08, 2021 (GLOBE NEWSWIRE) -- Ocugen, Inc.
About this update from Ocugen, Inc.
[{"type":"text","content":"Modifier gene therapy platform has potential to treat multiple retinal diseases with one product\nMALVERN, Pa., Nov. 08, 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 it has submitted an Investigational New Drug application (IND) with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2 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 RHO. “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,” said Dr. Shankar Musunuri, Chairman of the Board, Chief Executive Officer, and Co-Founder of Ocugen. “Our novel modifier gene therapy platform is a strong example of our approach to drug development.” 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. “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,” commented Arun Upadhyay, PhD, Vice President and Head of Research & Development at Ocugen. “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.” The planned Phase 1/2 clinical study will evaluate the safety and proof-of-concept of OCU400, utilizing unilateral sub-retinal injection (one eye per study subject) in 18 patients, with an extension for contralateral eye and long-term safety follow up. Ocugen has already successfully completed manufacturing at commercial scale (200L...