European Commission Grants Ocugen Orphan Medicinal Product Designation for Candidate Gene Therapy Product, OCU400, for Treatment of Leber’s Retinitis Pigmentosa and Congenital Amaurosis

MALVERN, Pennsylvania, February 23, 2021 (GLOBE NEWSWIRE) – Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing and commercializing gene therapies to cure diseases of blindness and develop a vaccine to save lives of COVID-19, announced today that, on the recommendation of the European Medicines Agency (EMA), the European Commission has granted orphan drug designation for OCU400 (AAV5-hNR2E3), for the treatment of both retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA).

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

“We believe that the granting of this designation by the European Commission validates the potential of our gene modifying therapy platform to treat many inherited retinal diseases (IRDs). IRDs associated with PR and LCA diseases are caused by mutations in more than 175 genes, and it is not practical to develop therapies that are specific for each gene. OCU400 has the remarkable potential to address a significant number of patients worldwide who desperately need to be rescued from these diseases of blindness and we are working diligently to bring this program to the clinic, ”said Dr. Shankar Musunuri, President of the Board, Official Executive Director and co-founder of Ocugen.

“RP and ACL are chronically debilitating groups of IRDs characterized by severe impairment in visual functions beginning in infancy, often progressing to night blindness and tunnel vision, and eventually causing total blindness as soon as the patient is 40 years old. . Since the existing approved therapy only targets a small percentage of this population, there is an unmet need for new treatment options targeting a broader population of DKD patients, ”said Dr Mohamed Genead, Chairman of the Advisory Board. Retina Scientist and Acting Medical Director at Ocugen.

Nuclear hormone receptors like NR2E3 they are important modulators of retinal development and function that act as “master genes” in the retina. NR2E3 It is delivered to target cells in the retina using an adeno-associated viral vector (AAV). As a powerful modifier gene, the expression of NR2E3 within the retina it can help to restore retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration. Preclinical results published in Nature’s gene therapy demonstrate the potency of gene modifying therapy to obtain broad-spectrum therapeutic benefits in the early and advanced stages of RP, including vision rescue in the early and advanced stages of the disease.

Orphan drug designation in Europe offers certain benefits to drug developers as they develop drugs for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 5 out of 10,000 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 photoreceptors, with subsequent degeneration of cone photoreceptors and retinal pigment epithelium (RPE). Visual impairment usually manifests as night blindness and progressive visual field loss. Its prevalence is 1 in 3000 to 1 in 5000. PR can be seen in isolation (typical PR) or in association with systemic disease. More than 150 genetic mutations have been associated with RP and this number represents only 60% of the RP population. The remaining 40% of RP patients cannot be genetically diagnosed, which makes 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 a young age. Patients often present with nystagmus, slow or almost absent pupillary responses, severely decreased visual acuity, photophobia, and elevated hyperopia. It is the most serious retinal dystrophy causing blindness at the age of 1 year. 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
OCU400 is a new candidate gene therapy product with the potential to be broadly effective in restoring the integrity and function of the retina in a variety of genetically diverse IRDs. OCU400 is the first program that Ocugen is advancing based on its innovative gene-modifying therapy platform developed by Dr. Neena Haider, associate professor of ophthalmology at Harvard Medical School and associate scientist at Schepens Eye Research Institute (SERI) of Massachusetts Eye and Ear. Ocugen obtained an exclusive worldwide license from SERI to develop and commercialize ophthalmic products based on specific nuclear hormone receptor genes, including NR2E3. Consists of a working copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to target cells in the retina using an AAV vector. As a powerful modifier gene, the expression of NR2E3 within the retina it can help restore retinal homeostasis, stabilize cells, and potentially rescue photoreceptors from degeneration.

About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure diseases of blindness and develop a life-saving vaccine from COVID-19. Our innovative gene modifying therapy platform has the potential to treat multiple retinal diseases with one drug: “one to many” and our new candidate biologic aims to offer better therapy to patients with neglected diseases such as macular degeneration. Moist age-related, diabetic macular edema and diabetic retinopathy. We are jointly developing the Bharat Biotech COVAXIN ™ vaccine candidate for COVID-19 in the US market. For more information, visit

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

Contact with the media:
For Ocugen:
Emmie Twombly
[email protected]
+ 1857-389-6042


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