The European Commission provides the Ocugen Orphan Medicinal Product Specification for a gene therapy product candidate, OCU400, for the treatment of both Retinitis Pigmentosa and Congenital Leber Amaberosis

MALVERN, Pa., Feb. 23, 2021 (GLOBE NEWSWIRE) – Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company with a focus on discovering, developing, and commercializing gene therapies to treat blind diseases and develop vaccines to save. living from COVID-19, it was announced today that the European Commission, on the recommendation of the European Medicines Agency (EMA), has specified an orphan drug product specification for OCU400 (AAV5-hNR2E3), for the treatment of both retinitis pigmentosa (RP) and Congenital Leber amaurosis (LCA).

The prevalence of RP in Europe is estimated at around 165,000 patients and the incidence of LCA in Europe is estimated at around 40,000 patients. Globally, the number of RP and LCA sufferers is estimated at around 2.0 million and 0.2 million, respectively.

“We believe that the issuance of this specification by the European Commission demonstrates the potential of our gene modifier gene platform for the treatment of many hereditary retinal diseases (IRDs). IRDs associated with RP and LCA diseases are caused by mutations in more than 175 genes, and it is inconvenient to develop treatments that are specific to each gene. The OCU400 has an incredible potential to address the huge number of patients worldwide who are in dire need of rescue from these blind diseases and we are working diligently to move this program to the clinic, ” said Dr. Shankar Musunuri, Chairman of the Board, Chief Executive Officer, and Co-Founder of Ocugen.

“RP and LCA are harmful debilitating groups of IRDs characterized by severe adverse effects in visual functions beginning as early as childhood, often progressing to night blindness and tunnel vision and eventually causing complete blindness. as early as the mid-40s of the patient. With the current approved treatment treating only a small percentage of this population, there is an unmet need for new treatment options that address a wider population of patients with IRDs, ”Said Dr Mohamed Genead, Chair of the Retina Scientific Advisory Board and Acting Chief Medical Officer. of Ocugen.

Nuclear Hormone Receptors as NR2E3 they are important modulators of retinal development and function as “key genes” in the retina. NR2E3 delivered to target cells in the retina using adeno-bound viral vector (AAV). As a strong mutation gene, a sense of NR2E3 within the retina may help restore retinal homeostasis, which may stabilize cells and save photoreceptor degradation. Preclinical results published in Natural gene therapy demonstrates the potential of gene modification to obtain broad-spectrum therapeutic benefits in early and advanced stages of RP including visual salvage at early and advanced stages of the disease.

Specification of orphan drug product in Europe offers particular benefits to drug developers while developing drugs that are intended to be safely and effectively handled, detected or detected. prevention of rare diseases or conditions that affect less than 5 in 10,000 patients in the European Union. Benefits include protocol assistance, lower regulatory fees, research grants, and 10 years of market exclusion following regulatory approval.

In Retinitis Pigmentosa
Retinitis pigmentosa is a clinically and genetically heterogeneous group of IRDs characterized by a progressive diffuse disorder of predominantly rod photoreceptors, with subsequent decay of cone photoreceptors, and retinal pigment epithelium (RPE). Lack of vision usually manifests as night blindness and progressive loss of visual field. Frequency is 1 in 3,000 to 1 in 5,000. RP can be seen in isolation (normal RP) or in association with systemic disease. More than 150 gene mutations have been associated with RP and this number represents just 60% of the RP population. The other 40% of RP patients cannot be genetically diagnosed, making it difficult to develop individual treatments.

About Leber Congenital Amaberosis
Leber congenital amaurosis is a family of congenital retinal dystrophies that leads to severe vision loss at a young age. Patients present with nystagmus, slow or near-complete pupillary responses, a marked decrease in visual acuity, photophobia and elevated hyperopia are common. It is the most severe retinal dystrophy causing blindness before the age of 1 year. This dystrophy is a heterogeneous hereditary digestive disease affecting 1 in 30,000 to 1 in 81,000 subjects. Mutations in one of more than two dozen LCA genes can be caused.

Mu OCU400
OCU400 is a novel gene therapy product candidate with the potential to be generally effective in restoring retinal integrity and function across a range of genetically diverse IRDs. OCU400 is the first program that Ocugen is promoting based on its advanced gene mutation gene platform developed by Dr. Neena Haider, Associate Professor of Ophthalmology at Harvard School of Medicine and Associate Scientist at the Schepens Eye Research Institute (SERI) of Massachusetts Eye and East. Ocugen received special worldwide approval from SERI to develop and commercialize ophthalmology products based on specific genes of nuclear hormone receptors, including NR2E3. Consists of an active copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to target cells in the retina using AAV vector. As a strong mutation gene, a sense of NR2E3 inside the retina may help restore retinal homeostasis, stabilize cells and potentially save photoreceptors from decay.

About Ocugen, Inc.
The Ocugen, Inc. is a biopharmaceutical company with a focus on the discovery, development, and commercialization of gene therapies to treat blind diseases and the development of a life-saving vaccine from COVID-19. Our advanced gene mutation platform has the potential to treat a number of retinal diseases with a single drug – “one to many” and our novel biological product candidate aims to offer a better cure to patients with underdeveloped diseases such as age-related macular degeneration, diabetic macular degeneration. edema, and diabetic retinopathy. We are co-developing a COVAXIN ™ Bharat Biotech vaccine candidate for COVID-19 in the US market. For more information, visit

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Sanjay Subramanian
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