Tag: OCGN

Research News and Market Data on OCGN

March 18, 2024

MALVERN, Pa., March 18, 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 the appointment of Dr. Huma Qamar as Chief Medical Officer (CMO).

“I’m very pleased to name Dr. Qamar as the CMO at Ocugen,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This appointment recognizes her invaluable contributions to building a strong clinical team, overseeing a well-conducted Phase 1/2 gene therapy trial, and establishing relationships with leading medical and academic institutions and key opinion leaders.”

Dr. Qamar is a distinguished healthcare professional with a rich background in clinical research, having contributed her expertise to esteemed Ivy League institutions including Yale University, Harvard University, and University of Pennsylvania. Her expertise encompasses the development of Phase I-IV clinical protocols and execution of clinical studies, FDA inspections, and effective leadership of medical affairs teams.

“Having been a part of Ocugen’s mission and vision for more than three years now, it’s a pleasure to lead Ocugen’s clinical development at this pivotal time,” said Dr. Qamar. “I am committed to addressing patients’ unmet medical needs and bringing novel modifier gene therapy products to the market globally.”

Dr. Qamar has expertise in multiple therapeutic areas, including gene and cell therapy, vaccines, oncology (Heme-Onc, CAR-T, rare tumors, sarcoma, melanoma, women’s health, GU & GI, fetal oncology), rheumatology, dermatology, neurology, cardiology, hepatology, and infectious diseases.

“Dr. Qamar has a deep understanding of Ocugen’s ongoing clinical trials and a close working relationship with the clinical and regulatory teams, both of which are fundamentally important to the success of Ocugen’s programs,” said Dr. Prabhavathi Fernandes, Independent Lead Director, Ocugen board. “I’m confident that her determination and dedication will bring the Company’s first-in-class candidates through the upcoming clinical milestones to deliver on Ocugen’s long-term strategy.”

Prior to joining Ocugen, Dr. Qamar was Senior Vice President, Head of R&D at FSD Pharma—demonstrating her commitment to advancing scientific innovation and pharmaceutical development.

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 www.ocugen.com 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. 

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

Research News and Market Data on OCGN

March 13, 2024

PDF Version

MALVERN, Pa., March 13, 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 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age related macular degeneration (dAMD). GA affects approximately 1 million people in the United States alone.

“We are very enthusiastic about the potential of OCU410 as a one-time treatment for life with a single sub-retinal injection,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “While there are currently two recently approved products for the treatment of GA, both require approximately 6-12 intravitreal injections annually and target only the complement system. OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress.”

Up to 13 leading retinal surgery centers across the United States are participating in the ArMaDa clinical trial. The enrollment in the first cohort is now complete and 3 subjects received 200µL single subretinal administration of the low dose (2.5×10¹⁰ vg/mL) of OCU410.

“As a retinal surgeon, I am encouraged by therapeutic options that can potentially provide long-term benefit to my patients,” said Lejla Vajzovic, MD, FASRS, Director of Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure in Adult and Pediatric Vitreoretinal Surgery and Diseases, Duke University Eye Center. “OCU410 is a novel modifier gene therapy approach that could initiate a paradigm shift in the field of ophthalmology.”

The ArMaDa clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×10¹⁰ vg/mL), medium dose (5×10¹⁰ vg/mL), and high dose (1.5 ×10¹¹ vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group.

“The American Macular Degeneration Foundation (AMDF) has supported the research of Dr. Neena Haider, inventor of modifier gene therapy, and OCU410 in particular, and is pleased that Ocugen is now spearheading the clinical trials necessary to bring this therapy closer to patients,” said Matthew Levine, Director of Grants, Advocacy and Partnerships at AMDF. “The continued advancement of OCU410 offers hope to those whose vision is already deteriorating that their remaining vision could be preserved and could potentially prevent others with an early dAMD diagnosis from developing any significant vision loss.”

The Company will continue to provide clinical updates.

About dAMD and GA
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in one’s direct line of sight.

dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.

About OCU410
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.

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 www.ocugen.com 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. 

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

Research News and Market Data on OCGN

December 21, 2023

MALVERN, Pa., Dec. 21, 2023 (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 the Company received alignment from FDA on key aspects of the Phase 3 clinical trial design to assess the safety and efficacy of OCU400 in patients with RHO and other gene mutations associated with Retinitis Pigmentosa (RP).

“This news brings us even closer to fulfilling our mission to bring our first-in-class, gene-agnostic therapies to market and provide access to patients globally,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We look forward to beginning the Phase 3 clinical trial, which we plan to initiate in early 2024.”

During a multidisciplinary meeting with FDA, based on preliminary results from an ongoing Phase 1/2 study, Ocugen received alignment on key aspects of the Phase 3 study design—including the study endpoint, patient enrollment strategy, and study duration of one year. The Phase 3 clinical trial will enroll a broader group of RP patients, including patients with the most common RHO gene mutation, based on OCU400’s potentially gene-agnostic mechanism of action.

With orphan drug and RMAT designations in place for OCU400, FDA’s alignment on key aspects of the Phase 3 study design positions Ocugen to confidently move forward in pursuing product development and licensure for OCU400.

Currently there are approximately 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation. Advancing OCU400 to Phase 3 clinical development will be an important step toward addressing unmet needs in the RP patient community.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies 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 therapeutic and clinical benefits of our product candidates, expectations for clinical trial timing and results, anticipated timing of clinical trial updates and expectations for timing and outcome of regulatory interactions, 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; that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities; that receipt of orphan drug and RMAT designations may not lead to faster development or regulatory review; and that regulatory authorities may disagree with additional aspects of our clinical trial designs or may not approve our future IND applications on the anticipated timeline or at all. 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
Head of Communications
IR@ocugen.com

Research News and Market Data on OCGN

December 19, 2023

• RMAT designation intended to help expedite development of new regenerative medicines

MALVERN, Pa., Dec. 19, 2023 (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 the FDA has granted RMAT designation to Ocugen’s investigational product OCU400 for the treatment of retinitis pigmentosa (RP) associated with RHO mutations.

“RMAT designation is a significant accomplishment for the OCU400 clinical development program, as it validates the potential for our game-changing gene therapy approach to fulfill an unmet medical need for people who are facing blindness due to RP,” said Arun Upadhyay, PhD, Chief Scientific Officer and Head of R&D at Ocugen. “FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”

RMAT designation for OCU400 was based on preliminary clinical data supporting the maintenance and improvement of visual acuity and function in RP patients in the OCU400 -101 Phase 1/2 clinical trial as measured by Best Corrected Visual Acuity (BCVA), Low Luminance Visual Acuity (LLVA), and Multi-Luminance Mobility Test (MLMT).

RMAT designation is part of the 21st Century Cures Act. The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA. Ocugen is working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development for OCU400 to support an application for marketing authorization.

Current data support the gene-agnostic mechanism of action for OCU400, which suggests that it may be able to provide treatment benefit to a broader group of RP and Leber congenital amaurosis (LCA) patients. Ocugen intends to submit additional efficacy and safety data for OCU400 in RP and LCA patients to the FDA in the future to potentially expand this RMAT designation to broader RP and LCA patient populations.

RHO mutations affect more than 10,000 of the 110,000 people in the United States diagnosed with RP. In the latest clinical study update from the Phase 1/2 trial of OCU400, 86% (6/7) of RHO mutation subjects experienced either stabilization or improvement in MLMT scores from baseline, among which 29% (2/7) demonstrated 3 Lux luminance level improvement. There are currently no treatment options available for RP patients with RHO gene mutations.

OCU400 represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions, such as homeostasis, reproduction, development, and metabolism to potentially improve retinal health and function.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies 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, the expected benefits of RMAT designation, and anticipated regulatory interactions, 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
Head of Communications
IR@ocugen.com 

Research News and Market Data on OCGN

December 13, 2023

MALVERN, Pa., Dec. 13, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the first patient has been dosed in the ArMaDa Phase 1/2 clinical trial of OCU410 (AAV-RORA), a modifier gene therapy product candidate being developed for dry AMD (dAMD).

“OCU410, our first-in-class modifier gene therapy for dAMD, addresses gaps among other therapies available and in development for dAMD as a potential one-time treatment for life,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “We are very pleased to continue advancing our ophthalmic gene therapy pipeline, which remains the Company’s primary focus.”

This Phase 1/2 trial will assess the safety and efficacy of OCU410 for geographic atrophy (GA) secondary to dAMD and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study. Phase 2 is a randomized expansion phase in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 dose groups or to an untreated control group.

OCU410 is a potential curative therapy with a single sub-retinal injection that targets multiple pathways causing dAMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually.

“Breaking new ground in the pursuit of vision restoration, our pioneering modifier gene therapy candidate, OCU410, achieves another major milestone by dosing a GA patient in a Phase 1/2 clinical trial,” said Arun Upadhyay, PhD, Chief Scientific Officer, Head of R&D at Ocugen. “OCU410 offers hope for those battling GA that are faced with limited treatment options and the real prospect of ultimately losing their vision.”

“There remains a great unmet need for novel durable and effective treatments for GA, which remains one of the most common causes of vision loss globally,” said Benjamin Bakall, MD, PhD, director of clinical research at Associated Retina Consultants (ARC) and clinical assistant professor at University of Arizona, College of Medicine – Phoenix. “I am excited that we performed the first surgery with this novel therapeutic approach—designed to restore homeostasis and slow disease progression following a single treatment—at ARC in Phoenix, AZ, with the surgical team led by Dr. Mark Kwong, medical director of ARC.”

The first surgery was successful in delivering the new gene underneath the retina; the light sensitive nerve tissue lining the inside of the eye.

About dAMD and GA
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in one’s direct line of sight.

dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.

GA, an advanced form of dry age-related macular degeneration, affects approximately 1 million people in the United States alone.
About OCU410
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually.

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.

About Associated Retina Consultants
Associated Retina Consultants is the largest independent retina practice in the state of Arizona, taking part in groundbreaking clinical trials to bring new treatments for the benefit of patients with diseases affecting the retina. In addition to collaborating with Ocugen on the OCU410 clinical trial, in October 2023, Associated Retina Consultants—with a surgical team led by Dr. Mark Kwong—performed gene therapy with OCU400 on a pediatric CEP290 patient. This was the first retinal gene therapy of its kind performed on a child in Arizona.

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.

Contact:
Tiffany Hamilton
Head of Communications
IR@ocugen.com

Research News and Market Data on OCGN

November 10, 2023

MALVERN, Pa., Nov. 10, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the first patient has been dosed in its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease, a rare genetically inherited disease that directly affects the retina, often resulting in slow progressive vision loss in children and adults.

“There is a significant unmet medical need for the approximate 35,000 patients in the U.S. living with Stargardt disease,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “It is critical to our mission to develop innovative treatments for inherited retinal diseases and this milestone is an important step in bringing our novel modifier gene therapies to people who desperately need them.”

This Phase 1/2 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. 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 control group.

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.

“It is important and exciting to pursue novel therapies for untreatable blinding diseases,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas. “Initiation of this trial program investigating a new mechanism of action for the treatment of Stargardt disease is inspiring and brings hope to patients and families.”

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 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 www.ocugen.com 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. 

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