MALVERN, Pa., February 23, 2021 (GLOBE NEWSWIRE) – Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused on the discovery, development and commercialization of genetic therapies to cure blindness diseases and develop a vaccine for blindness save lives from COVID-19, announced today that, on the recommendation of the European Medicines Agency (EMA), the European Commission has granted the designation of orphan drug to OCU400 (AAV5-hNR2E3), for the treatment of retinitis pigmentosa (RP) and Leber’s congenital amaurosis (ACL).
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 PR and ACL is estimated at 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 RP and ACL diseases are caused by mutations in more than 175 genes, and it is impractical to develop specific therapies for each gene. OCU400 has the remarkable potential to serve a significant number of patients worldwide who are in desperate need of rescue from these blindness diseases and we are working diligently to bring this program to the clinic, ”said Dr. Shankar Musunuri, chairman of the board, Official executive director and co-founder of Ocugen.
“RP and ACL are chronically debilitating groups of IRDs characterized by severe impairment of visual functions beginning in childhood, often progressing to night blindness and tunnel vision and eventually causing total blindness as early as the patient’s mid-40s. Since existing approved therapy addresses only a small percentage of this population, there is an unmet need for new treatment options that address a broader population of patients with IRDs, ”said Dr. Mohamed Genead, president of the Retina Scientific Advisory Board and Ocugen’s acting Medical Director.
Nuclear hormone receptors, such as NR2E3 they are important modulators of the development of the retina and function 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 potent modifying gene, the expression of NR2E3 inside the retina can help to redefine retina homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration. Pre-clinical results published in Nature Gene Therapy demonstrate the potency of gene modifier therapy to obtain broad-spectrum therapeutic benefits in early and advanced stages of RP, including rescue of vision in early and advanced stages of the disease.
The 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 less than 5 in 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 rod photoreceptors, with subsequent degeneration of cone photoreceptors and retinal pigment epithelium (RPE). Visual impairment usually manifests as night blindness and progressive loss of visual field. Its prevalence is 1 in 3,000 to 1 in 5,000. FRy can be seen in isolation (typical FRy) 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 patients with RP cannot be diagnosed genetically, making it difficult to develop individual treatments.
About Leber Congenital Amaurosis
Leber’s congenital amaurosis is a family of congenital retinal dystrophies that results in severe vision loss at an early age. Patients generally have nystagmus, slow or almost absent pupillary responses, severely decreased visual acuity, photophobia and high farsightedness. It is the most severe retinal dystrophy that causes blindness at 1 year of age. This dystrophy is a genetically heterogeneous recessive disease that affects 1 in 30,000 to 1 in 81,000 individuals. Mutations in one of more than two dozen genes can cause ACL.
About OCU400
OCU400 is a new candidate for gene therapy product with the potential to be widely effective in restoring retinal integrity and function in a variety of genetically diverse IRDs. OCU400 is the first program that Ocugen is advancing based on its innovative modifying gene therapy platform developed by Dr. Neena Haider, Associate Professor of Ophthalmology at Harvard Medical School and Associate Scientist at the Massachusetts Eye and Ear Schepens Eye Research Institute (SERI) . Ocugen has obtained an exclusive worldwide license from SERI to develop and market eye products based on specified nuclear hormone receptor genes, including NR2E3. Consisting of a functional copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to target cells on the retina using an AAV vector. As a potent modifying gene, the expression of NR2E3 inside the retina can help to redefine retina homeostasis, stabilizing cells and potentially rescuing the photoreceptors from degeneration.
About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company focused on the discovery, development and commercialization of genetic therapies to cure blindness diseases and develop a life-saving vaccine for COVID-19. Our innovative modifying gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one for many” and our new biological product candidate aims to provide better therapy for patients with unmet diseases, such as related macular degeneration at wet age and macular diabetes edema and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN ™ candidate vaccine for COVID-19 in the US market. For more information, visit www.ocugen.com.
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