MALVERN, Pa., February 23, 2021 (GLOBE NEWSWIRE) – Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and develop a vaccine to lives off COVID-19, announced today that the European Commission has granted orphan designation for OCU400 (AAV5-h) on the recommendation of the European Medicines Agency (EMA)NR2E3), 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 LCA in Europe is estimated at approximately 40,000 patients. Worldwide, the number of people suffering from RP and LCA is estimated to be approximately 2.0 million and 0.2 million, respectively.
“We believe the award of this designation by the European Commission confirms the potential of our modifying gene therapy platform to treat many inherited retinal disorders (IRDs). IRDs associated with RP and LCA diseases are caused by mutations in more than 175 genes, and it is impractical to develop therapies specific for each gene. OCU400 has the remarkable potential to address a significant number of patients worldwide in urgent need of rescue from these blindness diseases and we are working hard to bring 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 chronically disabling groups of IRDs characterized by severe impairment of visual function that starts at an early age, often progressing to night blindness and tunnel vision, and eventually causing total blindness as early as mid-40s. Since the existing approved therapy treats only a small percentage of this population, there is an unmet need for new treatment options for a broader population of patients with IRDs, ”said Dr. Mohamed Genead, Chairman of 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 as “master genes” in the retina. NR2E3 is delivered to target cells in the retina using an adeno-associated viral (AAV) vector. As a powerful modification gene, expression of NR2E3 in the retina can help reset retinal homeostasis, potentially stabilizing cells and saving photoreceptor degeneration. Preclinical results published in Nature gene therapy demonstrate the potential of modifying gene therapy to elicit broad spectrum therapeutic benefits in early and advanced stages of RP, including vision rescue in early and advanced stages of the disease.
Orphan designation in Europe offers certain benefits to drug developers as they develop drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect less than 5 in 10,000 patients in the European Union. Benefits include protocol support, 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 visual field loss. Prevalence is 1 in 3,000 to 1 in 5,000. RP can be seen in isolation (typically RP) or in conjunction with systemic disease. More than 150 gene 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, making it difficult to develop individual treatments.
About Leber Congenital Amaurosis
Leber’s congenital amaurosis is a family of congenital retinal dystrophies that lead to severe vision loss at a young age. Patients usually exhibit nystagmus, slow or near-absent pupillary responses, severely reduced visual acuity, photophobia and high hyperopia. It is the most severe retinal dystrophy to cause blindness by the age of 1 year. This dystrophy is a genetically heterogeneous recessive disease that affects 1 in 30,000 to 1 in 81,000 people. Mutations in one of more than two dozen genes can cause LCA.
About OCU400
OCU400 is a new gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function in a range of genetically diverse IRDs. OCU400 is the first program promoting Ocugen based on the groundbreaking modifying gene therapy platform developed by Dr. Ocugen obtained an exclusive worldwide license from SERI to develop and commercialize ophthalmic products based on specified nuclear hormone receptor genes, including NR2E3Consisting of a functional copy of the nuclear hormone receptor gene NR2E3OCU400 is delivered to target cells in the retina using an AAV vector. As a powerful modification gene, expression of NR2E3 in 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 gene therapies to cure blindness diseases and the development of a vaccine to save lives from COVID-19. Our breakthrough modifying gene therapy platform has the potential to treat multiple retinal disorders with one drug – ‘one in many’ and our new biologic product candidate aims to provide better therapy for patients with deprived diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN ™ vaccine for COVID-19 in the US market. For more information, please visit www.ocugen.com.
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