European Commission awards Ocugen Orphan medical product for gene therapy candidate, OCU400, for the treatment of retinitis Pigmentosa and Leber congenital amaurosis

MALVERN, PA, February 23, 2021 (GLOBE NEWSWIRE) – Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused on the discovery, development, and commercialization of gene therapies for the treatment of blindness and the development of vaccines to save lives from COVID-19 , announced today that on the recommendation of the European Medicines Agency (EMA), the European Commission has awarded the orphan drug label to OCU400 (AAV5-hNR2E3), for the treatment of retinitis pigmentosa (RP) and Leber’s congenital amaurosis (LCA).

The prevalence of RP in Europe is estimated at approximately 165,000 patients, and the prevalence of LCA in Europe at approximately 40,000 patients. Globally, it is estimated that the number of people suffering from RP and LCA is around 2.0 million and 0.2 million, respectively.

“We believe that the award of this label by the European Commission has confirmed the potential of our gene therapy modifier platform for the treatment of many inherited retinal diseases (IRD). IRDs associated with RP and LCA diseases are caused by mutations in over 175 genes, and it is impractical to develop therapies specific to each gene. The OCU400 has tremendous potential to address a significant number of patients globally who desperately need rescue from these blindness diseases and we are working diligently to move this program to the clinic, ”said Dr. Shankar Musunuri, Chairman of the Board, Executive Director Officer and Co-Founder of Ocugen.

“RP and LCA are chronically debilitating groups of IRDs characterized by severe visual impairment starting in childhood, often progressing to night blindness and tunnel vision and eventually causing complete blindness as early as the mid-40s of the patient. “Since the existing approved therapy addresses only a small percentage of this population, there is an unmet need for new treatment options for the wider population of patients with IRD,” said Dr. Mohamed Genead, Chairman of the Retina Scientific Advisory Board and Acting Chief Medical Officer of Ocugen.

Nuclear hormone receptors such as NR2E3 are important modulators of retinal development and function that act as “major genes” in the retina. NR2E3 is delivered to retinal target cells using an adeno-linked viral (AAV) vector. As a potent gene modifier, expression NR2E3 within the retina can help reset retinal homeostasis, potentially stabilize cells, and rescue photoreceptor degeneration. Preclinical results published in Natural gene therapy demonstrate the power of modified gene therapy to elicit broad-spectrum therapeutic benefits in the early and advanced stages of RP, including vision rescue in the early and advanced stages of the disease.

The Orphan Medicinal Product Label in Europe offers certain benefits to drug manufacturers as they develop drugs designed to safely and effectively treat, diagnose or prevent rare diseases or conditions affecting less than 5,000 patients in the European Union. Benefits include protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity after regulatory approval.

About retinitis pigmentosa
Retinitis pigmentosa is a clinically and genetically heterogeneous group of IRDs characterized by diffuse progressive dysfunction of predominantly rod-shaped photoreceptors, with subsequent degeneration of conical photoreceptors and retinal pigment epithelium (RPE). Visual impairment usually manifests as night blindness and progressive loss of visual field. Its prevalence is 1 in 3000 to 1 in 5000. RP can be seen in isolation (typically RP) or in association with systemic disease. Over 150 gene mutations are associated with RP and this number represents only 60% of the RP population. The remaining 40% of RP patients cannot be genetically diagnosed, making it difficult to develop individual treatments.

About Leber’s congenital amaurosis
Leber congenital amaurosis is a family of congenital retinal dystrophies that results in severe vision loss at an early age. Patients are usually present with nystagmus, slow or almost absent pupillary reactions, severely reduced visual acuity, photophobia, and high hyperopia. It is the most severe retinal dystrophy that causes blindness up to 1 year of age. This dystrophy is a genetically heterogeneous recessive disease that affects 1 in 30,000 to 1 in 81,000 subjects. Mutations in one of more than two dozen genes can cause LCA.

About OCU400
The OCU400 is a new candidate for gene therapy with the potential to be widely effective in restoring retinal integrity and function in a range of genetically distinct IRDs. OCU400 is the first program that Ocugen is advancing based on its groundbreaking gene therapy modifier platform developed by Dr. Neena Haider, associate professor of ophthalmology at Harvard Medical School and associate scientist at the Schepens Eye Research Institute (SERI) in Massachusetts Eye and Ear. Ocugen has received an exclusive worldwide license from SERI to develop and commercialize ophthalmic products based on specific nuclear hormone receptor genes, including NR2E3. It consists of a functional copy of a nuclear hormone receptor gene NR2E3, The OCU400 is delivered to retinal target cells using an AAV vector. As a potent gene modifier, expression NR2E3 within the retina can help reset retinal homeostasis, stabilize cells, and potentially save photoreceptors from degeneration.

About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company focused on the discovery, development and commercialization of genetic therapies for the treatment of blindness and the development of a life-saving vaccine from COVID-19. Our breakthrough gene therapy modifier platform has the potential to treat multiple retinal diseases with a single drug – “one to many”, and our new candidate for a biological product aims to offer better therapy to patients with unsatisfactory diseases, such as age-related wet macular degeneration, diabetic macular edema and diabetic retinopathy. We are jointly developing Bharat Biotech’s candidate for the COVAXIN ™ vaccine against COVID-19 in the US market. For more information, visit

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Ocugen Contact:
Ocugen, Inc.
Sanjay Subramanian
Chief Financial Officer and Head of Corporate Development
[email protected]

Media contact:
For Ocugen:
Emmie Twombly
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