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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 diseases and the development of ‘a vaccine to save lives from COVID-19, today announced that, on the recommendation of the European Medicines Agency (EMA), the European Commission has granted orphan drug designation for OCU400 (AAV5-hNR2E3), for the treatment of retinitis pigmentosa (RP) and congenital Leber’s amaurosis (LCA).
The prevalence of RP in Europe is estimated at around 165,000 patients and the prevalence of stroke in Europe is estimated at around 40,000 patients. Globally, the number of people with RP and stroke is estimated to be 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 modifying gene therapy platform to treat many inherited diseases of the retina (IRD). IRDs associated with RP and LCA disease are caused by mutations in more than 175 genes, and it is impractical to develop specific therapies for each gene. The OCU400 has the remarkable potential to treat a significant number of patients worldwide who are in desperate need of being rescued from these diseases of blindness and we are working diligently to move this program to a clinic, ”said Dr Shankar Musunuri , Chairman of the Board of Directors, General Manager Officer and co-founder of Ocugen.
“RP and stroke are chronically debilitating groups of DRIs characterized by severe impairment of visual function from an early age, often progressing to night blindness and tunnel vision and eventually causing total blindness by mid-life. quarantine. Since the existing approved therapy is only for a small percentage of this population, there is an unmet need for new treatment options for a larger population of patients with DRI, ”said Dr Mohamed. Genead, Chairman of Retina’s Science Advisory Board and Interim Chief Medical Officer. of Ocugen.
Nuclear hormone receptors such as NR2E3 are important modulators of retinal development and function acting as “master genes” in the retina. NR2E3 is delivered to target retinal cells using an adeno-associated viral vector (AAV). As a potent modifier gene, the expression of NR2E3 in the retina can help reset retinal homeostasis, potentially stabilizing cells, and saving photoreceptor degeneration. Preclinical results published in Gene therapy from nature demonstrate the power of gene modifier therapy to achieve broad-spectrum therapeutic benefits in the early and late stages of RP, including vision saving in the early and late stages of disease.
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 support, 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 DRIs characterized by progressive diffuse dysfunction of predominantly rod-shaped 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 3000 to 1 in 5000. RP can be seen in isolation (typical RP) or in association with systemic disease. Over 150 gene mutations have been associated with RP and this number represents only 60% of the RP population. The remaining 40% of RP patients cannot be genetically diagnosed, making it difficult to develop individual treatments.
About Leber’s 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 present with nystagmus, slow or almost absent pupillary responses, severely reduced visual acuity, photophobia, and high hyperopia. It is the most severe retinal dystrophy leading to blindness at the age of 1 year. This dystrophy is a genetically heterogeneous recessive disease affecting 1 in 30,000 to 1 in 81,000 people. Mutations in any of more than two dozen genes can cause stroke.
About the OCU400
OCU400 is a new gene therapy product candidate with the potential to be widely effective in restoring retinal integrity and function in a range of genetically diverse IRDs. OCU400 is the first program offered by Ocugen based on its revolutionary modifying gene therapy platform developed by Dr. Neena Haider, associate professor of ophthalmology at Harvard Medical School and associate researcher at the Schepens Eye Research Institute (SERI) at Massachusetts Eye and Ear. Ocugen has obtained an exclusive worldwide license from SERI to develop and commercialize ophthalmic products based on specified nuclear hormone receptor genes, including NR2E3. Made up of a functional copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to target cells in the retina using an AAV vector. As a potent modifier gene, the expression of NR2E3 in 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 to cure blindness diseases and the development of a vaccine to save lives against COVID-19. Our revolutionary modifying gene therapy platform has the potential to treat multiple retinal diseases with a single drug – “one to many” and our new biologic product candidate aims to provide better therapy for patients with underserved conditions such as degeneration. wet age-related macular, diabetic macular edema and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN ™ vaccine candidate for COVID-19 in the US market. For more information, please visit www.ocugen.com.
Caution regarding forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. In some cases we may use terms such as “predict”, “believe”, “potential”, “proposed”, “continue”, “estimate”, “anticipate”, “expect”, “plan”, ” intends, “may”, “could”, “could”, “will”, “should” or other terms that reflect the uncertainty of future events or results to identify such forward-looking statements. These statements are subject to many important factors, risks and uncertainties which may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are further described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section titled “Risk Factors” in the Reports. quarterly and annually that we file with the SEC. All forward-looking statements we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update any forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release. Press.
Ocugen contact:
Ocugen, Inc.
Sanjay subramanian
Chief Financial Officer and Head of Business Development
[email protected]
Media contact:
For Ocugen:
LaVoieHealthScience
Emmie twombly
[email protected]
+ 1857-389-6042
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