MALVERN, Pa., Feb. 23, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical firm targeted on discovering, creating, and commercializing gene therapies to treatment blindness ailments and creating a vaccine to save lots of lives from COVID-19, as we speak introduced that on the suggestion of the European Medicines Company (EMA), the European Commission has granted orphan medicinal product designation for OCU400 (AAV5-hNR2E3), for the remedy 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 quantity of folks affected by RP and LCA is estimated to be round 2.0 million and 0.2 million, respectively.
“We consider 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 quantity 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 Govt Officer, and Co-founder of Ocugen.
“RP and LCA are chronically debilitating teams of IRDs characterised by extreme impairment in visible features beginning as younger as infancy, usually progressing into evening blindness and tunnel imaginative and prescient and finally inflicting whole blindness as early as the affected person’s mid-40s. Since the current authorized remedy solely addresses a small share of this inhabitants, there’s an unmet want for new remedy choices addressing a wider inhabitants of sufferers with IRDs,” stated Dr. Mohamed Genead, Chair of Retina Scientific Advisory Board and Performing Chief Medical Officer of Ocugen.
Nuclear Hormone Receptors similar to NR2E3 are essential modulators of retinal improvement and operate performing as “grasp genes” in the retina. NR2E3 is delivered to focus on cells in the retina utilizing an adeno-associated viral (AAV) vector. As a potent modifier gene, expression of NR2E3 inside the retina could assist reset retinal homeostasis, probably stabilizing cells and rescuing photoreceptor degeneration. Preclinical outcomes printed in Nature Gene Therapy reveal 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 gives sure advantages to drug builders whereas they develop medicine meant for secure and efficient remedy, analysis, or prevention of uncommon ailments or circumstances that influence fewer than 5 in 10,000 sufferers in the European Union. Advantages embody protocol help, diminished regulatory charges, analysis grants, and 10 years of market exclusivity following regulatory approval.
About Retinitis Pigmentosa
Retinitis 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). Visible impairment often manifests as evening blindness and progressive visible area 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 therapies.
About Leber Congenital Amaurosis
Leber congenital amaurosis is a household of congenital retinal dystrophies that leads to extreme imaginative and prescient loss at an early age. Sufferers current often with nystagmus, sluggish or near-absent pupillary responses, severely decreased visible acuity, photophobia and excessive hyperopia. It’s the most extreme retinal dystrophy inflicting blindness by the age of 1 yr. This dystrophy is a genetically heterogeneous recessive illness affecting 1 in 30,000 to 1 in 81,000 topics. Mutations in a single of greater than two dozen genes may cause LCA.
OCU400 is a novel gene remedy product candidate with the potential to be broadly efficient in restoring retinal integrity and operate throughout a variety of genetically numerous IRDs. OCU400 is the first program that Ocugen is advancing primarily based on its breakthrough modifier gene remedy platform developed by Dr. Neena Haider, Affiliate Professor of Ophthalmology at Harvard Medical Faculty and Affiliate Scientist at the Schepens Eye Analysis Institute (SERI) of Massachusetts Eye and Ear. Ocugen obtained an unique worldwide license from SERI to develop and commercialize ophthalmology merchandise primarily based on specified nuclear hormone receptor genes, together with NR2E3. Consisting of a practical copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to focus on cells in the retina utilizing an AAV vector. As a potent modifier gene, expression of NR2E3 inside the retina could assist reset retinal homeostasis, stabilizing cells and probably rescuing photoreceptors from degeneration.
About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical firm targeted on discovering, creating, and commercializing gene therapies to treatment blindness ailments and creating a vaccine to save lots of 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 similar to moist age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We’re co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market. For extra info, please go to www.ocugen.com.
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