MALVERN, Pa., Feb. 23, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical firm centered on discovering, growing, and commercializing gene therapies to remedy blindness ailments and growing a vaccine to avoid wasting lives from COVID-19, right now introduced that on the advice of the European Medicines Agency (EMA), the European Commission has granted orphan medicinal product designation for OCU400 (AAV5-hNR2E3), for the therapy of each retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA). The prevalence of RP in Europe is estimated at roughly 165,000 sufferers and the prevalence of LCA in Europe is estimated at roughly 40,000 sufferers. Globally, the variety of folks affected by RP and LCA is estimated to be round 2.0 million and 0.2 million, respectively. “We imagine the granting of this designation by the European Commission validates the potential of our modifier gene remedy platform to deal with many inherited retinal ailments (IRDs). IRDs related to RP and LCA ailments are brought on by mutations in over 175 genes, and it’s impractical to develop therapies which are particular to every gene. OCU400 has the outstanding potential to deal with a big variety of sufferers globally who’re in determined want of rescue from these blindness ailments and we’re working diligently to maneuver this program to clinic,” stated Dr. Shankar Musunuri, Chairman of the Board, Chief Executive Officer, and Co-founder of Ocugen. “RP and LCA are chronically debilitating teams of IRDs characterised by extreme impairment in visible capabilities beginning as younger as infancy, typically progressing into night time blindness and tunnel imaginative and prescient and finally inflicting whole blindness as early because the affected person’s mid-40s. Since the present authorised remedy solely addresses a small share of this inhabitants, there may be an unmet want for brand spanking new therapy choices addressing a wider inhabitants of sufferers with IRDs,” stated Dr. Mohamed Genead, Chair of Retina Scientific Advisory Board and Acting Chief Medical Officer of Ocugen. Nuclear Hormone Receptors akin to NR2E3 are essential modulators of retinal improvement and performance appearing as “grasp genes” within the retina. NR2E3 is delivered to focus on cells within the retina utilizing an adeno-associated viral (AAV) vector. As a potent modifier gene, expression of NR2E3 inside the retina might assist reset retinal homeostasis, probably stabilizing cells and rescuing photoreceptor degeneration. Preclinical outcomes revealed in Nature Gene Therapy display the efficiency of modifier gene remedy to elicit broad-spectrum therapeutic advantages in early and superior levels of RP together with imaginative and prescient rescue in early and superior levels of the illness. Orphan medicinal product designation in Europe affords sure advantages to drug builders whereas they develop medicine supposed for secure and efficient therapy, analysis, or prevention of uncommon ailments or circumstances that influence fewer than 5 in 10,000 sufferers within the European Union. Benefits embrace protocol help, lowered regulatory charges, analysis grants, and 10 years of market exclusivity following regulatory approval. About Retinitis PigmentosaRetinitis pigmentosa is a clinically and genetically heterogeneous group of IRDs characterised by diffuse progressive dysfunction of predominantly rod photoreceptors, with subsequent degeneration of cone photoreceptors, and retinal pigment epithelium (RPE). Visual impairment normally manifests as night time blindness and progressive visible discipline loss. Its prevalence is 1 in 3,000 to 1 in 5,000. RP could also be seen in isolation (typical RP) or in affiliation with systemic illness. Over 150 gene mutations have been related to RP and this quantity represents solely 60% of the RP inhabitants. The remaining 40% of RP sufferers can’t be genetically recognized, making it tough to develop particular person remedies. About Leber Congenital AmaurosisLeber congenital amaurosis is a household of congenital retinal dystrophies that leads to extreme imaginative and prescient loss at an early age. Patients current normally with nystagmus, sluggish or near-absent pupillary responses, severely decreased visible acuity, photophobia and excessive hyperopia. It is essentially the most extreme retinal dystrophy inflicting blindness by the age of 1 12 months. This dystrophy is a genetically heterogeneous recessive illness affecting 1 in 30,000 to 1 in 81,000 topics. Mutations in one in every of greater than two dozen genes could cause LCA. About OCU400OCU400 is a novel gene remedy product candidate with the potential to be broadly efficient in restoring retinal integrity and performance throughout a variety of genetically various IRDs. OCU400 is the primary program that Ocugen is advancing based mostly on its breakthrough modifier gene remedy platform developed by Dr. Neena Haider, Associate Professor of Ophthalmology at Harvard Medical School and Associate Scientist on the Schepens Eye Research Institute (SERI) of Massachusetts Eye and Ear. Ocugen obtained an unique worldwide license from SERI to develop and commercialize ophthalmology merchandise based mostly on specified nuclear hormone receptor genes, together with NR2E3. Consisting of a useful copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to focus on cells within the retina utilizing an AAV vector. As a potent modifier gene, expression of NR2E3 inside the retina might assist reset retinal homeostasis, stabilizing cells and probably rescuing photoreceptors from degeneration. About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical firm centered on discovering, growing, and commercializing gene therapies to remedy blindness ailments and growing a vaccine to avoid wasting lives from COVID-19. Our breakthrough modifier gene remedy platform has the potential to deal with a number of retinal ailments with one drug – “one to many” and our novel biologic product candidate goals to supply higher remedy to sufferers with underserved ailments akin to moist age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 within the U.S. market. 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