Ocugen, Inc., a biotechnology company dedicated to uncovering, developing, and marketing novel gene and cell therapies, biologics, and vaccines, is proud to present its groundbreaking modifier gene therapy platform at the 2023 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in New Orleans.
Their innovative platform includes OCU400 for retinitis pigmentosa and Leber congenital amaurosis, OCU410 for dry age-related macular degeneration (dry AMD), and OCU410ST for Stargardt disease, as well as OCU200, a novel biologic candidate to treat diabetic macular edema (DME). Don’t miss this chance to learn more about the latest advancements in gene therapy!
We are delighted to present the details of our breakthrough modifier gene therapy platform and our innovative biologics ophthalmic product pipeline to the ARVO community! It is a particularly exciting time for us to be at ARVO as we have just announced positive preliminary safety and efficacy results from the Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa.
We are eager to share this news, along with what we are doing across our ophthalmology portfolio, to combat hard-to-treat blindness diseases that are impacting millions globally.
OCU400, a gene-agnostic modifier gene therapy product, is based on the NHR gene NR2E3. This powerful gene has the ability to regulate various retinal functions, such as photoreceptor development, metabolism, phototransduction, inflammation and cell survival.
By resetting and balancing altered/affected cellular gene-networks, OCU400 has the potential to improve retinal health and function in patients with inherited retinal diseases. These diseases are estimated to affect around 125,000 people in the United States alone.
About OCU410 and OCU410ST
Ocugen is developing OCU410, a groundbreaking gene therapy product, to treat dry Age-related Macular Degeneration (AMD). Utilizing a novel AAV delivery platform, OCU410 delivers the RAR Related Orphan Receptor A gene (RORA) to the retina. RORA plays an important role in lipid metabolism and inflammation, which can help combat dry AMD in vitro and in vivo animal studies.
Ocugen is additionally planning to submit an Investigational New Drug (IND) application for Stargardt Disease, an orphan eye disease, in the second quarter of 2023, using the same technology as OCU410. Get ready for a revolutionary approach to treating dry AMD and Stargardt Disease!
OCU200 is a revolutionary fusion protein that could revolutionize the treatment of retinal diseases such as diabetic macular edema (DME), diabetic retinopathy and wet age-related macular degeneration. It is composed of human transferrin linked to human tumstatin to provide anti-proliferative, anti-inflammatory, and anti-oxidative effects.
OCU200 has the potential to provide better bioavailability and tissue penetrance than tumstatin alone, due to the presence of transferrin, and can selectively target the retinal and choroidal tissues. The distinct MOA binding through αVβ3 integrin pathways found in OCU200 may reduce the need for multiple injections, and could potentially benefit the 10 million people affected by these diseases in the U.S.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company with a mission to revolutionize healthcare for patients around the world. Through courageous innovation, we are discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that can improve the lives of those affected by retinal diseases, infectious diseases, and orthopedic diseases.
Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are dedicated to finding innovative solutions to unmet medical needs. We believe that our commitment to groundbreaking research and human capital can make a real difference in people’s lives.