Ocugen to Present on Modifier Gene Therapy Platform at ARVO 2023
Ocugen announced that the company will present on its modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa and Leber congenital amaurosis; OCU410 for the treatment dry age-related macular degeneration (dry AMD); OCU410ST for the treatment of Stargardt disease; and OCU200, a novel biologic candidate to treat diabetic macular edema (DME), at The Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting in New Orleans from April 23-27, 2023.
“We are thrilled to share more detail on our unique modifier gene therapy platform, as well as our novel biologics ophthalmic product pipeline with the professional community at ARVO,” Arun Upadhyay, PhD, Chief Scientific Officer and Head of Research, Development and Medical at Ocugen, said in a company news release. “It is especially exciting to be at ARVO just following positive preliminary safety and efficacy results from the phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa. We look forward to highlighting this most recent news along with the work we are doing across our ophthalmology portfolio to combat hard-to-treat blindness diseases affecting millions of patients globally."
Poster Presentation:
Title: Modifier Gene Approach Using OCU410 for Dry AMD Therapy: One Gene—Multiple Targets
Authors: Dinesh Singh, Mohamed Nsaibia, Sree Kattala, Subechhya Neupane, Matthew Ritts, Arun Upadhyay
Presenter: Dinesh Singh, Associate Director, Discovery, Ocugen
Presentation Type/Number: Poster Session, 755-C0356
Location: Exhibit Hall
Date: Sunday, April 23, 2023
Time: Noon – 1:45 p.m. CDT
Exhibitor Presentations (Exhibitor Education Lounge):
Title: Ocugen—OCU400—Modifier Gene Therapy for Treatment of Inherited Retinal Diseases: Retinitis Pigmentosa & Leber Congenital Amaurosis
Presenter: Arun Upadhyay, PhD, CSO and Head of Research, Development, and Medical
Date: Monday, April 24, 2023
Time: 2 p.m. CDT
Title: Ocugen—OCU410 & OCU410ST—Nuclear Receptor Gene RORA as a Potential Therapeutic for Dry AMD and Stargardt disease
Presenter: Dinesh Singh, Associate Director, Discovery, Ocugen
Date: Tuesday, April 25, 2023
Time: 2 p.m. CDT
Title: Ocugen—OCU200—A Novel Biologic for the Treatment of DME, DR, and Wet AMD
Presenter: Pushpendra Singh, Director, Cell and Gene Therapy, Ocugen
Date: Wednesday, April 26, 2023
Time: 2 p.m. CDT
About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has potential to improve retinal health and function in patients with inherited retinal diseases. These diseases, combined, account for approximately 125,000 cases in the U.S.
About OCU410 and OCU410ST
OCU410 is a modifier gene therapy product candidate being developed for the treatment of dry AMD. OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. Various genes associated with AMD are regulated by RORA. The RORA protein plays an important role in lipid metabolism and demonstrates an anti-inflammatory role, which we believe could be a potential therapeutic candidate for dry AMD based on in-vitro and in-vivo (animal model) studies. Using the same technology as OCU410, Ocugen plans to submit an IND for Stargardt disease, an orphan eye disease, in Q2 2023.
About OCU200
OCU200 is a novel fusion protein consisting of human transferrin linked to human tumstatin. It exerts anti-proliferative, anti-inflammatory, and anti-oxidative effects by selective targeting to the retinal and choroidal tissues. OCU200 potentially showcases better bioavailability and tissue penetrance than tumstatin alone due to transferrin and provides distinct MOA binding through αVβ3 integrin pathways that can potentially reduce the number of injections for patients. OCU200 can potentially be used for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. These diseases, combined, account for approximately 10 million cases in the U.S.