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Home / Health / The European Commission awarded Ocugen’s orphan drug product designation for the gene therapy product candidate OCU400 for the treatment of retinitis pigmentosa and Leber congenital Amat disease

The European Commission awarded Ocugen’s orphan drug product designation for the gene therapy product candidate OCU400 for the treatment of retinitis pigmentosa and Leber congenital Amat disease



February 23, 2021, Malvern, Pennsylvania (GLOBE NEWSWIRE)-Biopharmaceutical company Ocugen, Inc. (NASDAQ: OCGN) focuses on the discovery, development and commercialization of gene therapies for the treatment of blindness diseases, and development Developed a vaccine to save deaths from COVID-19. It was announced today that the European Commission has awarded OCU400 (AAV5-h) the title of orphan drug product according to the recommendations of the European Medicines Agency (EMA)NR2E3), for the simultaneous treatment of retinitis pigmentosa (RP) and congenital Leber black disease (LCA).

In Europe, the prevalence of RP is estimated to be about 165,000 cases, and in Europe, the prevalence of LCA is estimated to be about 40,000 cases. Globally, it is estimated that the number of people suffering from RP and LCA is approximately 2 million and 200,000, respectively.

“We believe that this title granted by the European Commission demonstrates the potential of our modified gene therapy platform to treat many inherited retinal diseases (IRD). IRDs related to RP and LCA diseases are caused by mutations in more than 1

75 genes, so It is impractical to develop specific therapies for each gene. OCU400 has great potential and can solve the large number of patients who urgently need to be rescued from these blind diseases worldwide. We are working hard to bring this plan to the clinic,” the chairman and chairman of the board CEO Dr. Shankar Musunuri said. Official and co-founder of Ocugen.

RP and LCA are chronic debilitating groups of IRD, which are characterized by severe visual impairment beginning in infancy, often developing into night blindness and tunnel vision, and eventually leading to complete blindness in the patient’s 40s. Since the existing approved therapies can only address a small part of this population, the demand for new treatment options has not been met, thereby addressing more IRD patient populations. “Okugan.

Nuclear hormone receptors, such as NR2E3 It is an important regulator of retinal development and function, and plays the role of “master gene” in the retina. NR2E3 It is delivered to target cells in the retina using an adeno-associated virus (AAV) vector. As an effective modifier gene, NR2E3 The retina may help reset the homeostasis of the retina, stabilize the cells and save the degeneration of photoreceptor cells.Preclinical results published in Natural gene therapy Prove that modified gene therapy in the early and late stages of RP (including restoration of vision in the early and late stages of the disease) can lead to broad-spectrum therapeutic benefits.

In Europe, designating orphan drug products to drug developers can bring certain benefits because the drugs they develop are designed to treat, diagnose, or prevent rare diseases or conditions safely and effectively, affecting less than 5 out of 10,000 patients in Europe example. Benefits include agreement assistance, reduced regulatory costs, research grants, and market exclusivity for 10 years after obtaining regulatory approval.

About Retinitis Pigmentosa
Retinitis pigmentosa is a clinical and genetic heterogeneity group of IRD, which is characterized by diffuse progressive dysfunction of mainly rod-shaped photoreceptors, followed by degeneration of pyramidal photoreceptors and retinal pigment epithelium (RPE). Visual impairment usually manifests as night blindness and progressive visual field loss. Its prevalence is one in 3,000 to one in 5,000. RP may appear alone (typical RP) or related to systemic diseases. There are more than 150 gene mutations related to RP, and this number represents only 60% of the RP population. The remaining 40% of RP patients cannot be genetically diagnosed, so it is difficult to develop a separate treatment method.

About Leber congenital Amart disease
Leber congenital amaurosis is a family of congenital retinal dystrophy that can cause severe vision loss in childhood. Patients usually experience nystagmus, dull or almost no pupil response, severely decreased vision, photophobia and high hyperopia. This is the most serious form of retinal dystrophy, which can lead to blindness by the age of one. This malnutrition is a genetically heterogeneous recessive disease that affects 1 in 30,000 to 1 in 81,000. Mutations in one of two or more genes can cause LCA.

About OCU400
OCU400 is a new gene therapy product candidate with broad potential to restore retinal integrity and function in a series of genetically diverse IRDs. OCU400 is the first program being developed by Ocugen. It was developed by a ground-breaking modified gene therapy platform developed by an associate professor of ophthalmology at Harvard Medical School and an associate scientist at the Schepens Institute of Ophthalmology (SERI) in Massachusetts. Developed by Dr. Neena Haider. Ocugen has obtained the exclusive global license of SERI to develop and commercialize ophthalmic products based on specific nuclear hormone receptor genes, including NR2E3.Consists of functional copies of nuclear hormone receptor genes NR2E3Then, the AAV vector is used to deliver OCU400 to target cells in the retina. As an effective modifier gene, NR2E3 The retina may help reset retinal homeostasis, stabilize cells and may rescue degraded photoreceptors.

About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company dedicated to discovering, developing and commercializing gene therapies for the treatment of blindness, and developing a vaccine that can save the lives of COVID-19. Our ground-breaking modified gene therapy platform has the potential to treat multiple retinal diseases with one drug “one-to-many”. Our new biological product candidate products are designed to serve underserved diseases such as wet age-related macular degeneration, Diabetic Macula) provides better treatment for edema and diabetic retinopathy. We are jointly developing Bharat Biotech’s COVAXIN™ vaccine candidate product against COVID-19 in the US market. For more information, please visit www.ocugen.com.

Warning statement regarding forward-looking statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. In some cases, we may use such things as “forecast”, “believe”, “potential”, “propose”, “continue”, “estimate”, “expect”, “expect”, “plan”, “intend” “May”, “may”, “may”, “will”, “should” or other words that express uncertainties about future events or results to identify these forward-looking statements. Such statements are subject to many important factors and risks These factors may cause actual events or results to be materially different from our current expectations. We have dealt with these and other risks and uncertainties in our regular documents filed with the United States Securities and Exchange Commission (SEC). A more comprehensive description, including the risk factors described in the section titled “Risk Factors” in our quarterly and annual reports submitted to the SEC. Any forward-looking statements we make in this press release represent only the release of this press release Except as required by law, we assume no obligation to update the forward-looking statements contained in this news release due to new information, future events or other reasons after the date of this news release.

Ocugen Contact:
Ocugen, Inc.
Sanjay Sulamania
Chief Financial Officer and Head of Corporate Development
ir@ocugen.com

Media Contact:
For Ocugen:
LaVoieHealthScience
Amy Tombley
etwombly@lavoiehealthscience.com
+1 857-389-6042


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