Modifier gene therapy platform has potential to treat multiple retinal diseases with one product

MALVERN, PA, USA I November 08, 2021 I 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) to support clinical studies. Ocugen plans to follow the Phase 1/2 trial with a Phase 3 study, upon further review of the data.

Between 2019 and 2020, OCU400 was granted four orphan drug disease designations from the FDA from treating four different gene mutation-associated retinal degenerative diseases. In 2021, the European Medicines Agency (EMA) granted Ocugen broad orphan medicinal product designation 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.

About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. and Canadian markets. For more information, please visit

SOURCE: Ocugen