Ocugen, Inc. Announces Dosing Completion of Subjects with Stargardt in Cohort 1 of Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST

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Ocugen, Inc. completes dosing in the first cohort of its Phase 1/2 GARDian clinical trial for OCU410ST, a gene therapy candidate for Stargardt disease, with positive feedback from leading retinal surgery centers.
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Completion of dosing in the initial cohort of Ocugen's Phase 1/2 GARDian clinical trial signifies a pivotal step in the development of OCU410ST, a gene therapy candidate for Stargardt disease. Stargardt disease, an orphan disease, has a significant unmet medical need due to the absence of approved treatments. The therapy's modifier gene approach, which aims to provide a one-time treatment, could revolutionize the management of this inherited retinal disease, potentially halting or slowing the progression of vision loss.

The initial safety data from the low-dose cohort will be critical in determining the therapy's risk profile, which is a fundamental concern for patients and regulators alike. The study's design, including a dose-ranging Phase 1 and a blinded, dose-expansion Phase 2, adheres to standard clinical trial protocols for establishing both efficacy and safety. A successful trial could lead to significant long-term benefits for patients and reduce the socioeconomic burden of Stargardt disease. However, it is essential to await further data on efficacy and potential adverse events that may arise from higher dosages.

Ocugen's progression in its clinical trial phases could impact its valuation and investor confidence, particularly in the biotechnology sector where clinical milestones are closely monitored. The involvement of leading retinal surgery centers indicates a robust research environment and may enhance the credibility of the trial outcomes. It is important to note that gene therapies often command high prices in the market due to the complexity of development and the potential for long-lasting effects, which could lead to significant revenue for Ocugen if OCU410ST is approved.

However, the biotech industry is known for its high risk and reward profile, with clinical trial outcomes being a major inflection point for stock prices. Investors should monitor subsequent trial updates, as any negative developments could adversely affect the stock's performance. Conversely, positive results could lead to strategic partnerships or buyout opportunities, which are common in the industry for promising therapies.

The economic implications of a successful gene therapy for Stargardt disease are substantial. Currently, the lack of treatment options for this orphan disease imposes a continuous cost on healthcare systems and society, including long-term care and assistive services for affected individuals. By potentially offering a one-time treatment, OCU410ST could reduce these long-term costs.

Furthermore, the rarity of Stargardt disease means that OCU410ST could qualify for orphan drug status, which offers financial incentives such as tax credits and market exclusivity. This status can enhance the economic viability of developing treatments for rare diseases. However, the pricing strategy for OCU410ST will be a critical factor in its accessibility and affordability, which will be a point of discussion among payers, healthcare providers and patient advocacy groups.

MALVERN, Pa., Feb. 22, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that dosing is complete in the first cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease is an orphan blindness disease that affects approximately 35,000 people in the United States alone.

“This is an important clinical milestone for our first-in-class, potential one-time therapy for the treatment of Stargardt disease,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen. “Although Stargardt is one of the most common inherited retinal diseases, there remains no treatment option to address this condition. OCU410ST provides hope to these patients who may eventually lose their vision.”

Up to 10 leading retinal surgery centers across the United States are participating in the GARDian clinical trial. In the first cohort, 3 subjects received 200L single subretinal administration of the low dose (3.75x1010 vg/mL) of OCU410ST.

“I am very pleased to participate in this study and to offer a novel modifier gene therapy option to my patients,” said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at the University of Arizona, College of Medicine—Phoenix. “Until now, we have not had any effective treatment that can prevent the vision loss in patients with Stargardt disease. Now, I believe that this approach can offer a new therapeutic option to address the disease itself.”

The GARDian clinical trial will assess the safety of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose ranging study consisting of three dose levels [low dose (3.75×10E10 vg/mL), medium dose (7.5×10E10 vg/mL), and high dose (2.25×10E11 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated. The Company will continue to provide clinical updates.

About Stargardt Disease

Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.

Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.

About OCU410ST

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.

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 patients’ 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 and follow us on X and LinkedIn.

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. 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. 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. 

Tiffany Hamilton
Head of Communications


What is the purpose of Ocugen's Phase 1/2 GARDian clinical trial for OCU410ST?

The trial aims to assess the safety and effectiveness of OCU410ST, a gene therapy candidate for Stargardt disease.

How many people in the United States are affected by Stargardt disease?

Stargardt disease affects approximately 35,000 people in the United States.

Who is leading the GARDian clinical trial for Ocugen?

Up to 10 leading retinal surgery centers across the United States are participating in the trial.

What are the dose levels being tested in the Phase 1 of the GARDian trial?

Phase 1 consists of three dose levels: low dose (3.75×10E10 vg/mL), medium dose (7.5×10E10 vg/mL), and high dose (2.25×10E11 vg/mL).

What is the Phase 2 of the GARDian trial focused on?

Phase 2 is a randomized, dose-expansion study where subjects will be randomized to different OCU410ST dose groups or untreated.

Ocugen, Inc.


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About OCGN

ocugen, inc., is a rapidly growing ophthalmology company developing a rich clinical pipeline of innovative therapies that address rare and underserved ocular disorders. the company’s lead programs in ocular graft versus host disease (ocu300) and chronic dry eye disease (ocu310) are expected to enter pivotal clinical trials in 2018. ocu300 received the first and only orphan drug designation for ocular graft versus host disease, providing certain regulatory and economic benefits. ocugen is also developing novel biologic therapies for retinitis pigmentosa (ocu100) and wet amd (ocu200), as well as a groundbreaking modifier gene therapy platform with potential to address a broad spectrum of inherited retinal disorders (ocu400). for more information, please visit