MALVERN, Pennsylvania, February 23, 2021 (GLOBE NEWSWIRE) – Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused on the discovery, development and commercialization of gene therapies to cure blindness diseases and develop a vaccine for save alive 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 for OCU400 (AAV5-hNR2E3), for the treatment of both retinitis pigmentosa (RP) and congenital Leber amaurosis (LCA).
The prevalence of PR in Europe is estimated at approximately 165,000 patients and the prevalence of ACL in Europe is estimated at approximately 40,000 patients. Globally, it is estimated that the number of people suffering from PR and stroke is about 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 therapy modifying platform to treat many inherited retinal diseases (RHDs). RDIs associated with RP and ACL diseases are caused by mutations in more than 175 genes, and it is not practical to develop specific therapies for each gene.OCU400 has the remarkable potential to care for a significant number of patients worldwide who are in desperate need of rescue from these diseases of blindness and we are working diligently to move 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 groups of IRDs that are chronically weakened and are characterized by severe impairment of visual function from childhood, often progressing to night blindness and tunnel vision, and eventually they cause total blindness as early as the mid-1940s of the patient. Given that existing approved therapy only targets a small percentage of this population, there is an unmet need for new treatment options targeting a wider population of patients with IRD, ”said Dr. Mohamed Genead, Chairman of the Scientific Advisory Board of Retina and Acting Medical Director of Ocugen.
Nuclear hormone receptors such as NR2E3 they are important modulators of retinal development and function that act as “master genes” in the retina. NR2E3 is administered to retinal target cells via an adeno-associated viral vector (AAV). As a potent gene modifier, expression of NR2E3 within the retina can help restore retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration. Preclinical results published in Gene Therapy of Nature demonstrate the power of gene-modifying therapy for broad-spectrum therapeutic benefits in early and advanced stages of PR, including vision rescue in early and advanced stages of the disease.
Orphan drug designation in Europe offers certain benefits for drug developers while developing drugs intended for 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.
On retinitis pigmentosa
Retinitis pigmentosa is a clinically and genetically heterogeneous group of IRDs characterized by a progressive diffuse dysfunction of predominantly rod photoreceptors, with subsequent degeneration of cone photoreceptors and retinal pigment epithelium (RPE). Visual impairment usually manifests as nocturnal blindness and progressive loss of visual field. Its prevalence is 1 in 3,000 to 1 in 5,000. RP can be seen in isolation (typical RP) or in association with systemic diseases. More than 150 gene 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 genetically diagnosed, making it difficult to develop individual treatments.
On Leber’s Congenital Amaurosis
Leber’s congenital amaurosis is a family of congenital retinal dystrophies that produces severe vision loss at an early age. Patients generally present with nystagmus, slow or near-absent pupillary responses, greatly decreased visual acuity, photophobia, and high farsightedness. It is the most severe retinal dystrophy that causes blindness at the age of one 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 stroke.
About OCU400
OCU400 is a new candidate gene therapy product with the potential to be widely effective in restoring integrity and retinal function across a range of genetically diverse IRDs. OCU400 is the first program that Ocugen is advancing based on its advanced gene modification therapy platform developed by Dra. Neena Haider, associate professor of ophthalmology at Harvard Medical School and associate scientist at the Schepens Eye Research Institute (SERI) in Massachusetts Eye and Ear. Ocugen obtained a worldwide exclusive license from SERI to develop and market ophthalmology products based on specified nuclear hormone receptor genes, including NR2E3. It consists of a functional copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to retinal target cells using an AAV vector. As a potent gene modifier, expression of NR2E3 within the retina can help restore retinal homeostasis, stabilizing cells and potentially rescuing photoreceptors from degeneration.
About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing and marketing gene therapies to cure blindness diseases and develop a life-saving vaccine for COVID-19. Our breakthrough gene therapy platform has the potential to treat multiple retinal diseases with one drug, “one in many,” and our new candidate biologic product aims to offer better therapy to patients with untreated diseases, such as macular degeneration related to wet age, macular diabetes edema, and diabetic retinopathy. We are jointly developing the COVAXIN ™ vaccine from Bharat Biotech candidate against COVID-19 in the US market. For more information, visit www.ocugen.com.
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