MALVERN, Pa., 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 and develop a vaccine to save lives COVID-19, announced today that, on the recommendation of the European Medicines Agency (EMA), the European Commission has designated the orphan medicinal product OCU400 (AAV5-hNR2E3), for the treatment of both retinitis pigmentosa (RP) and congenital Leber amaurosis (LCA).
The prevalence of RA 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 about 2.0 million and 0.2 million, respectively.
“We believe that the European Commission’s award of this designation validates the potential of our modifying gene therapy platform for the treatment of many inherited retinal diseases (RHDs). IRD associated with RP and ACL diseases are caused by mutations in over 175 genes and it is not practical to develop therapies specific to each gene. OCU400 has the remarkable potential to address a significant number of patients globally who are in desperate need of rescue from these blinding diseases, and we are working diligently to move this program to the clinic, ”said Dr. Shankar Musunuri, President of board, executive director Officer and co-founder of Ocugen.
“RP and ACL are debilitating chronic groups of IRD characterized by severe impairment of visual function since childhood, often progressing to night blindness and tunnel vision and eventually causing total blindness since the mid-40s of the patient. As the existing approved therapy addresses only a small percentage of this population, there is an unmet need for new treatment options that address a wider population of patients with IRD, ”said Dr. Mohamed Genead, chair of the Retina Scientific Advisory Committee and physician. interim chief. 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 it is administered to retinal target cells using an adeno-associated viral vector (AAV). As a powerful modifier gene, the expression NR2E3 inside the retina can help reset retinal homeostasis, stabilizing cells and saving photoreceptor degeneration. Preclinical results published in Gene therapy of nature demonstrates the potential of gene modifier therapy to achieve broad-spectrum therapeutic benefits in the early and advanced stages of PR, including saving vision in the early and advanced stages of the disease.
The designation of the orphan medicine in Europe offers certain benefits to drug developers, while they develop medicines for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting less than 5 in 10,000 patients in the Union. European. 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 heterogeneous clinical and genetic group of IRD characterized by diffuse progressive dysfunction of stem photoreceptors, with subsequent degeneration of conical 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. RP can be seen in isolation (typical RP) or in association with systemic disease. Over 150 genetic mutations have been associated with RP and this number represents only 60% of the RP population. The remaining 40% of patients with RA cannot be genetically diagnosed, which makes it difficult to develop individual treatments.
About Leber’s congenital Amaurosis
Leber congenital amaurosis is a family of congenital retinal dystrophies that results in severe vision loss at an early age. Patients usually have nystagmus, slow or near-absent pupillary responses, severely low visual acuity, photophobia, and high farsightedness. It is the most severe retinal dystrophy that causes blindness until the age of 1 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 ACL.
About the OCU400
The OCU400 is a new candidate gene therapy product with the potential to be widely effective in restoring retinal integrity and function in a wide range of genetic IRDs. OCU400 is the first program Ocugen is advancing based on its advanced genetic modification therapy platform developed by Dr. Neena Haider, an associate professor of ophthalmology at Harvard Medical School and an associate scientist at the Schepens Eye Research Institute (SERI) in Massachusetts Eye and Ear. Ocugen has obtained an exclusive worldwide license from SERI to develop and market ophthalmology products based on specific nuclear hormone receptor genes, including NR2E3. Consisting of a functional copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to retinal target cells using an AAV vector. As a powerful modifier gene, the expression NR2E3 inside 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 for the cure of blindness and the development of a vaccine to save the lives of COVID-19. Our discovery-modifying gene therapy platform has the potential to treat multiple retinal diseases with a single drug – “one to many,” and our new biologic candidate aims to provide better therapy for patients with underlying diseases, such as would be macular degeneration related to wet age, edema and diabetic retinopathy. We are co-developing the COVAXIN vaccine ™ Bharat Biotech candidate for COVID-19 on the US market. For more information, please visit www.ocugen.com.
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Sanjay subramanian
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