Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress

Rhea-AI Summary

Ocugen announced upcoming presentations of its modifier gene therapy platform at two major ophthalmology events: The ARVO 2025 Annual Meeting in Salt Lake City (May 4-8) and Retina World Congress in Ft. Lauderdale (May 8-11). The company will showcase three key therapies:

• OCU400 for retinitis pigmentosa (Phase 3 LiMeliGhT trial)
• OCU410ST for Stargardt disease (Phase 2/3 pivotal trial)
• OCU410 for geographic atrophy (Phase 2 ArMaDa trial)

CEO Dr. Shankar Musunuri confirmed plans to file three Biologics License Applications/Marketing Authorization Applications within three years. The presentations include clinical trial updates, safety data, and efficacy results across multiple sessions. Dr. Huma Qamar, Chief Medical Officer, will lead the company showcase at Eyecelerator and participate in panel discussions at both events.

Positive

• Three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) planned for submission in next three years
• Multiple clinical trials in advanced stages - Phase 3 for retinitis pigmentosa, Phase 2/3 for Stargardt disease, Phase 2 for geographic atrophy
• Gene-agnostic approach potentially addressing large patient populations
• Diversified pipeline with three distinct products: OCU400, OCU410ST, and OCU410

Negative

• None.

MALVERN, Pa., April 29, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410ST for the treatment of Stargardt disease (Phase 2/3 pivotal confirmatory clinical trial), and OCU410 for the treatment of geographic atrophy (Phase 2 ArMaDa clinical trial), at The Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting at the Calvin L. Rampton Salt Palace Convention Center in Salt Lake City, Utah from May 4-8, 2025, and Retina World Congress at the Marriott Harbor Beach Resort in Ft. Lauderdale, Florida from May 8-11, 2025.

“We look forward to sharing more about the potential of our modifier gene therapy platform and the meaningful results we are seeing in the clinic during these two important meetings for the retina community,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Ocugen remains on track to deliver on our commitment to file three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) in the next three years—potentially addressing significant unmet medical need for large patient populations through our gene-agnostic approach.”

The ARVO Annual Meeting is a premiere gathering for eye and vision scientists from across the globe, students, and those in affiliated fields to share the latest research findings and collaborate on innovative solutions. Retina World Congress brings together leading retina specialists from every continent to achieve a global scientific and clinical exchange in retinal health.

Ocugen’s presence in Utah kicks off with the Company Showcase at Eyecelerator, presented by Dr. Huma Qamar, Chief Medical Officer at Ocugen, and continues through presentations and thought leadership engagement at ARVO.

Eyecelerator @ Park City 2025

Session: Retina—Gene Therapy and Novel Mechanisms of Action Showcase
Location: Grand Hyatt Deer Valley, Strawberry Ballroom, Park City, UT
Date: Friday, May 2, 2025
Time: 2:06 p.m. MDT
Presenter: Dr. Huma Qamar

ARVO

Exhibitor Education Forum
Two-Year Follow-Up of a Phase 1/2 Clinical Trial for the Safety and Efficacy of OCU400 Novel Modifier Gene Therapy for Retinitis Pigmentosa
Location: Exhibitor Floor, Section 1037
Date: Monday, May 5, 2025
Time: 2 p.m. MDT 
Presenter: Benjamin Bakall, MD, Ph.D., Assistant Clinical Professor, University of Arizona, College of Medicine–Phoenix, and Director for Clinical Research, Director for The Inherited Retinal Disease and Visual Function Clinic, Associated Retina Consultants

Safety and Efficacy of OCU410ST: A Phase 1/2 Trial of a Novel Modifier Gene Therapy for Stargardt Disease (GARDian)
Location: Exhibitor Floor, Section 1037
Date: Tuesday, May 6, 2025
Time: 2 p.m. MDT
Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin

An Evaluation of the Safety and Efficacy of Novel Modifier Gene Therapy OCU410 for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
Location: Exhibitor Floor, Section 1037
Date: Wednesday, May 7, 2025
Time: 2 p.m. MDT
Presenter: Neena Haider, Ph.D., Faculty Harvard Medical School and Founder, CEO, Shifa Precision

Paper Session
Preliminary Safety and Efficacy of OCU410 for Treatment of Geographic Atrophy: Phase 1/2 OCU410: The Age-related Macular Degeneration (ArMaDa) Study Update
Presentation Number: 3675
Session Number and Title: 358/AMD Clinical research II
Location: Ballroom J
Date: Tuesday, May 6, 2025
Time: 4:15 p.m. MDT
Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin

Poster Session
A0513: Safety and Efficacy of OCU410ST for the Treatment of Stargardt Disease: Phase 1/2 Study Update
Location: Hall A-E
Date: Thursday, May 8, 2025
Time: 2 p.m. MDT
Presenter: Ramiro Maldonado, MD, Duke Center for Ophthalmic Genetics, Duke Pediatric Retina, Adult vitreo-Retinal diseases

Dr. Qamar will represent Ocugen at Retina World Congress to share the Company presentation and serve alongside notable retinal surgeons and industry peers during a panel discussion.

Retina World Congress

Retina Unplugged
Inherited and Rare Retinal Diseases Session
Moderators: Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD
Location: Grand Ballroom
Date: Thursday, May 8, 2025
Time: 10:35 am – 11:12 a.m. EDT

Ocugen is committed to bringing game-changing therapies to treat inherited retinal diseases as well as blindness diseases affecting millions to market and working even harder to provide access to patients globally.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that 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 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.

Contact:
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

FAQ

What clinical trials is Ocugen (OCGN) presenting at ARVO 2025?

Ocugen will present three key clinical trials at ARVO 2025: Phase 3 LiMeliGhT trial for OCU400 (retinitis pigmentosa), Phase 2/3 trial for OCU410ST (Stargardt disease), and Phase 2 ArMaDa trial for OCU410 (geographic atrophy).

When and where will Ocugen (OCGN) present its modifier gene therapy platform in May 2025?

Ocugen will present at two events: ARVO Annual Meeting in Salt Lake City, Utah (May 4-8, 2025) and Retina World Congress in Ft. Lauderdale, Florida (May 8-11, 2025).

How many BLA/MAA applications does Ocugen (OCGN) plan to file in the next three years?

Ocugen plans to file three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) in the next three years for their gene therapy treatments.

What is unique about Ocugen's (OCGN) gene therapy approach for eye diseases?

Ocugen uses a gene-agnostic approach through their modifier gene therapy platform, potentially addressing large patient populations with unmet medical needs in blindness diseases.

Which eye diseases is Ocugen (OCGN) targeting with their gene therapy treatments?

Ocugen is targeting retinitis pigmentosa with OCU400, Stargardt disease with OCU410ST, and geographic atrophy (related to dry age-related macular degeneration) with OCU410.