Tag: OCGN

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  

Research News and Market Data on OCGN

November 9, 2023

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Conference Call and Webcast Today at 8:30 a.m. ET

• OCU400 demonstrated favorable safety and tolerability profile in retinitis pigmentosa (RP) and Leber congenial amaurosis (LCA) subjects
• Completed dosing of three LCA patients including a pediatric patient
• OCU400 Phase 1/2 study results suggest stabilization or improvement of Best-Corrected Visual Acuity (BCVA) or Multi-Luminance Mobility Testing (MLMT) or Low-Luminance Visual Acuity (LLVA) in treated eyes of 83% (10/12) subjects
• Stabilization or improvement in MLMT scores from baseline in 86% (6/7) of RHO subjects demonstrated gene-agnostic property of OCU400 modifier gene therapy and its potential benefits in broader RP and LCA subjects
• Ocugen’s inhaled mucosal vaccine candidate for COVID-19—OCU500—selected by National Institutes of Health (NIH)/National Institute of Allergy and Infectious Diseases’ (NIAID) Project NextGen for inclusion in clinical trials

MALVERN, Pa., Nov. 09, 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 reported third quarter 2023 financial results along with a general business update.

“Ocugen has made significant pipeline progress in the third quarter,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “In particular, based on the OCU400 data presented in September, I am as enthusiastic as ever regarding the potential for our modifier gene therapy approach to make an important difference in the lives of people living with blindness diseases. OCU400—our lead candidate—has the potential to address multiple genetic mutations with a single product compared to traditional gene therapies that target one gene at a time.”

This clinical efficacy update provided in September 2023 included data for 12 subjects who have completed a minimum of 6 months follow up. 83%, 83%, and 75% of subjects demonstrated stabilization or improvements in OCU400 treated eyes on BCVA, LLVA, and MLMT scores, respectively from baseline. 86% of subjects with the RHO gene mutation experienced either stabilization or increase in MLMT scores from baseline, including a subset of 29% that demonstrated a three Lux luminance level improvement. Preservation of remaining vision, slowing disease progression, or improving the vision can significantly impact patients’ quality of life.

“The improvements in BCVA, LLVA and MLMT in RHO patients—a disease affecting more than 10,000 people in the U.S. alone—supports the gene-agnostic mechanism of action for OCU400,” said Dr. Musunuri.

In October 2023, OCU500, was selected by the NIAID Project NextGen for inclusion in clinical trials. OCU500 will be tested via two different mucosal routes, inhalation into the lungs and as a nasal spray. Currently used injected vaccines, including mRNA vaccines, are not effective in preventing infection and spread although effective against severe infection. Generating mucosal immunity in nasal and respiratory airways could help block the infection at its origin, thus limiting spread.

“NIAID support for our mucosal vaccine platform is the result of many months of hard work and dedicated effort by our Ocugen team and is a first step in potentially expanding the platform to flu and other respiratory viral diseases and infections,” said Dr. Musunuri. “Additionally, this funding makes it possible to focus the majority of Ocugen’s R&D and clinical resources on our first-in-class gene and cell therapies.”

As Ocugen prepares to start Phase 3 for OCU400, begin dosing patients for OCU410 and OCU410ST, and initiate the Phase 1 trial for OCU500 in collaboration with NIAID, the company is making meaningful progress toward its long-term strategy and delivering on each of its scientific platforms in the near-term.

Ophthalmic Gene Therapies—First-in-class

• OCU400 – Completed dosing adult RP patients in the dose-escalation and dose-expansion portions of the trial; completed dosing three LCA patients including a pediatric patient. Phase 3 clinical trial for the treatment of RP to be initiated in early 2024 following FDA concurrence on study design. Subsequently, the Company is expecting to expand the OCU400 Phase 3 clinical trial for LCA patients in the second half of 2024 based on Phase 1/2 study results in LCA patients and alignment with the FDA.
• OCU410 and OCU410ST – IND applications to initiate Phase 1/2 trials for both OCU410 and OCU410ST were cleared by the FDA and the Company intends to dose patients in Phase 1/2 trials by the end of 2023.

Ophthalmic Biologic Product

• OCU200 – Continuing to work on the Company’s response to the FDA regarding the IND application and expect to initiate the Phase 1 clinical trial in the first half of 2024, contingent on the lift of the FDA hold and adequate availability of funding.

Regenerative Cell Therapies—First-in-class

• NeoCart^® – Ocugen’s autologous regenerative cell therapy (using patients’ own cartilage cells) remains on track to begin its Phase 3 clinical trial in the second half of 2024. A cGMP facility for manufacturing NeoCart is expected to be completed at the end of 2023 and qualification is expected in the first half of 2024.  

Vaccines Portfolio—First-in-class

• Mucosal Vaccine Platform – The Company is collaborating with NIAID to initiate clinical trials of OCU500 in early 2024.

Third Quarter 2023 Financial Results

• The Company’s cash, cash equivalents, and investments totaled $53.5 million as of September 30, 2023, compared to $90.9 million as of December 31, 2022. The Company had 256.5 million shares of common stock outstanding as of September 30, 2023.
• Total operating expenses for the three months ended September 30, 2023 were $15.4 million and included research and development expenses of $6.3 million and general and administrative expenses of $9.1 million. This compares to total operating expenses for the three months ended September 30, 2022 of $23.1 million that included research and development expenses of $15.6 million and general and administrative expenses of $7.5 million.
• Ocugen reported a $0.06 net loss per common share for the three months ended September 30, 2023 compared to a $0.10 net loss per common share for the three months ended September 30, 2022.

Conference Call and Webcast Details

Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s senior management team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call or webcast using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 1787631
Webcast: Available on the events section of the Ocugen investor site

A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.

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, 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 include, but are not limited to, statements regarding the Company’s clinical development activities and related anticipated timelines; strategy, business plans and objectives for its clinical stage programs; plans and timelines for the preclinical and clinical development of its product candidates, including the therapeutic potential, clinical benefits and safety thereof; expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials; the ability to initiate new clinical programs; and Ocugen’s financial condition. 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, as well as discussions of potential risks, uncertainties, and other important factors in Ocugen’s subsequent filings with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. 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 2, 2023

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MALVERN, Pa., Nov. 02, 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 it will host a conference call and live webcast to discuss the Company’s third quarter 2023 financial results and provide a business update at 8:30 a.m. ET on Thursday, November 9, 2023.

Ocugen will issue a pre-market earnings announcement on the same day. Attendees are invited to participate on the call using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 1787631
Webcast: Available on the events section of the Ocugen investor site

A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.

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, 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

October 10, 2023

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• NIAID is conducting early phase clinical trials on select next generation vaccine candidates with the intent to identify the most effective platforms and delivery routes
• OCU500 will be tested as both inhaled and intranasal vaccine candidates
• Clinical trials scheduled to start in early 2024

MALVERN, Pa., Oct. 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 National Institute of Allergy and Infectious Diseases (NIAID), a part of the National Institutes of Health, will conduct a trial comparing the administration of Ocugen’s mucosal vaccine candidate, OCU500, via two different mucosal routes, inhalation into the lungs and as a nasal spray.

Ocugen is developing a novel anti-viral mucosal vaccine platform initially targeting COVID-19 and influenza (flu). The intent is to provide protection against severe disease, increase durability and prevent transmission of viral threats. OCU500 is based on a novel chimpanzee adenovirus-vectored (ChAd) technology. Earlier clinical studies to prevent COVID-19 employing a similar vector administered via inhalation demonstrated increased mucosal antibodies, systemic antibodies, and durable immune response up to one year using one fifth (1/5) of the dose compared to the same vaccine given via intramuscular administration. Additionally, Ocugen believes that this vaccine can be rapidly scaled-up as new variants emerge.

“We believe our novel mucosal vaccine platform technology has the potential to prevent infection and spread of COVID-19, and improve durability for an annualized vaccine similar to flu,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This is the first vaccine candidate using our inhaled platform technology, which we hope to expand in order to address multiple respiratory threats, including flu. We have benefited from a strong collaborative relationship with NIAID and BARDA since the start of Project NextGen and we look forward to participating in this initiative.”

NIAID will be conducting clinical trials to evaluate several early stage vaccine candidates. The study involving Ocugen’s vaccine will be administered via both intranasal and inhaled routes and is designed to help answer an important question – does an inhaled COVID-19 vaccine provide greater immune response than the same vaccine administered through a nasal spray. Upon completion of the trial NIAID and Ocugen will assess the results and determine next steps for OCU500.

Project NextGen is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID, with funding from Project NextGen, will cover the full cost of the clinical trials, including operations and related analysis. Ocugen will be responsible for providing clinical trial materials and upon completion will have full right of reference to the findings, which Ocugen believes will provide clinical evidence to support the further development of the Company’s lead mucosal vaccine candidate.

The announced collaboration comes at a time when COVID-19 infection rates are rising with the emergence of new variants. Durability of existing vaccines continues to be of concern with antibody protection waning several months following vaccination while vaccine compliance rates have declined since the initial wave. According to a recent Harris poll, 66% of Americans would prefer to have more vaccine options. The poll also found that 52% of Americans would be more open to getting an intranasal or inhaled, versus injectable COVID-19 vaccine.

Ocugen looks forward to this important next step in the development of its novel mucosal vaccine platform and further supporting the Company’s commitment to advancing public health.

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 Twitter 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, 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

October 4, 2023

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MALVERN, Pa., Oct. 04, 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 Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen will present at the 2023 Cell & Gene Meeting on the Mesa being held October 10-12, 2023 in Carlsbad, CA at the Park Hyatt Aviara Resort.

Dr. Musunuri will provide an overview of Ocugen’s clinical development programs, including the business strategy across its unique gene and cell therapy platforms, anticipated milestones, and more detail about the Company’s recent positive Phase 1/2 OCU400 data results.

Details of the presentation are as follows:

Event:	2023 Cell & Gene Meeting on the Mesa
Date & Time:	October 11, 2023 at 5:15 PM, PDT
Location:	Park Hyatt Aviara Resort, Carlsbad, CA, Aseptic Technologies Ballroom

“The Cell & Gene Meeting on the Mesa convenes leading industry professionals confronting major scientific, logistic, policy, and economic questions in cell and gene therapy,” said Dr. Musunuri. “I am pleased to join this impressive group and share the latest gene therapy news from Ocugen—compelling positive results from our Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA)—as well as our progress toward initiating the Phase 3 trial for the Company’s novel cell therapy product candidate for cartilage repair in the second half of 2024.”

Virtual attendance is available, which includes a livestream of Ocugen’s presentation and on-demand viewing of all conference sessions. Please visit https://meetingonthemesa.com for full information including registration.

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 Twitter 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

September 20, 2023

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MALVERN, Pa., Sept. 20, 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 Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen will participate in a panel at the annual Cantor Fitzgerald Global Healthcare Conference taking place from September 26-28, 2023 in NYC.

On the panel, Dr. Musunuri will join the CEO of Tarsier Pharma to discuss their companies’ respective work toward developing novel therapies for hard-to-treat ocular diseases. In addition, members of Ocugen’s executive team will conduct one-on-one meetings with registered investors, to showcase the Company’s business and clinical development strategy across its unique gene therapy, cell therapy, and vaccine platforms, and highlight recent corporate achievements and anticipated milestones.

Details of the presentation are as follows:

Date: Tuesday, September 26, 2023
Presentation Time: 1 p.m. ET
Location: Track 1, InterContinental Barclay Hotel, New York City

A live video webcast of the fireside chat will be available on the events page of the Ocugen investor site.

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 Twitter 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 
IR@ocugen.com  

Research News and Market Data on OCGN

September 13, 2023

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• Favorable safety and tolerability profile of OCU400 investigational drug product in RP and LCA subjects to date
• Clinical study update suggests continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes
• 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or LLVA or MLMT scores from baseline
• Notably, 86% (6/7) of RHO mutation subjects experienced either stabilization of or increase in MLMT scores from baseline including a subset of 29% (2/7) that demonstrated a 3 Lux luminance level improvement

MALVERN, Pa., Sept. 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 a clinical study update for Retinitis Pigmentosa (RP) participants treated in the Phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber congenital amaurosis (LCA) with mutation(s) in the CEP290 gene. This clinical study update is an extension of results provided by Ocugen on April 14, 2023, and includes additional subjects from the high dose group. The Company believes that OCU400—Ocugen’s therapeutic approach, utilizing a proprietary modifier gene therapy platform—has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration.

“This clinical study update supports our vision to help change the lives of patients suffering from inherited retinal diseases,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We remain dedicated to our mission of pioneering breakthroughs in biotechnology and believe that OCU400 has the potential to have an impact on the future treatment of patients with RP and LCA.” 

This Phase 1/2 trial is a multicenter, open-label, dose ranging study. A total of 18 subjects with vision impairment due to RP associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose (1.66 x 10¹⁰ vg/mL), medium dose (3.33 x 10¹⁰ vg/mL), or high dose (1.66 x 10¹¹ vg/mL) of OCU400. The study profile included a diverse group of subjects aged 18-77 years old, with varied disease stages, racial and ethnic profiles, medical histories, and mutation subgroups. Ocugen further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.

Inherited retinal diseases (IRDs) such as RP and LCA encompass a group of genetic disorders that affect the retina, the light-sensitive tissue at the back of the eye. These diseases often lead to a gradual loss of vision over time and can ultimately result in blindness. Stabilization of vision is crucial for patients with IRDs due to the progressive and degenerative nature of these conditions.

Preserving remaining vision, slowing disease progression, or improving the vision can significantly impact patients’ quality of life. It not only enhances the quality of life for affected individuals but also provides hope for future treatments that may ultimately lead to vision restoration. Comprehensive care, early diagnosis, and access to emerging therapies are essential components of a strategy to stabilize vision in IRD patients.

“I am gratified to see the progress we have made in our pursuit of developing a novel gene-agnostic therapy for RP and LCA. Our team’s unwavering dedication to advancing modifier gene therapy research demonstrated positive preliminary clinical results that offer renewed hope to patients and their families. We remain resolute in our mission and vision to bring a bright future to those with inherited or age-related retinal diseases through courageous innovation and unwavering determination,” said Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research, Development and Medical at Ocugen.

This clinical study update is based on the currently available data from Phase 1 (dose-escalation: Cohort 1, 2 and 3) and the Phase 2 (open enrollment) portion of the study. The exploratory efficacy update includes data for 12 subjects who have completed a minimum of 6-month follow up. The data set comprised of 2 subjects [Cohort 1] with 12-month follow-up, 5 subjects [N=2 from Cohort 1 and N=3 from Cohort 2] with 9-month follow-up, and 5 subjects [N=2 from Cohort 3 and N=3 from Open Enrollment/Phase 2] with 6-month follow-up.

“It is an important steppingstone for Ocugen and its mission to help the nearly 1.6 million patients affected by RP and LCA worldwide. For those suffering from these IRDs, this clinical trial update provides hope. It is encouraging to see a favorable safety and tolerability profile and positive efficacy readout for OCU400 in RP patients,” said Dr. Lejla Vajzovic, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine and leader in gene-therapy research.

Key efficacy outcomes from 12 subjects demonstrated:
BCVA:

• 83% (10/12) of subjects demonstrated stabilization or improvements in treated eyes in BCVA scores from baseline
• 42% (5/12) of OCU400 treated eyes experienced 4-letter improvement and 33% (4/12) treated eyes experienced 7-letter improvement in BCVA from baseline
• 57% (4/7) of RHO subjects’ treated eyes experienced 4-letter improvement and 43% (3/7) treated eyes experienced 7-letter improvement in BCVA scores from baseline

LLVA:

• 83% (10/12) of subjects demonstrated stabilization or improvement in treated eyes in LLVA scores from baseline
• 42% (5/12) of OCU400 treated eyes experienced 5-letter improvement (1 line) in LLVA from baseline, with 25% (3/7) increasing by 10 letters (2 lines)
• 43% (3/7) of RHO subjects experienced 5-letter improvement (1 line) in treated eyes in LLVA scores from baseline, among which 29% (2/7) increased by 10 letters (2 lines)

MLMT:

• 75% (9/12) of subjects demonstrated stabilization or improvement in treated eyes in MLMT scores from baseline
• 33% (4/12) of subjects in the low, medium, and high dose cohorts experienced at least 1 Lux luminance level improvement from baseline in treated eyes, among which 17% (2/12) increased by 3 Lux luminance levels
• 86% (6/7) of RHO subjects experienced either stabilization or increases in MLMT scores from baseline, among which 29% (2/7) improved by 3 lux levels

“The RHO mutation affects more than 10,000 people in the US,” said Dr. David Birch, Scientific Director, Retina Foundation of the Southwest and Principal investigator of the study. “In my view, the clinical study update supports the gene-agnostic mechanism of action of OCU400 in RHO patients. The improvements in BCVA, LLVA and MLMT in this patient population are very exciting and encouraging because stabilization alone could be considered as a treatment benefit.”

The clinical study update from the Phase 1/2 clinical trial demonstrated that OCU400 continued to be generally safe and well-tolerated in subjects across different mutations and dose levels. There were no serious adverse events (SAEs) related to the investigational product in the low and medium-dose cohorts. In the high-dose and open-enrollment cohorts, SAEs were reported for two subjects. Adverse events were mostly deemed related to the surgical procedure and resolved within a few days to weeks.

“The clinical study update released by Ocugen appears to have a tangible biological impact on Retinitis Pigmentosa associated with NR2E3 and RHO mutations,” said Dr. David Boyer, Clinical Professor of Ophthalmology USC/Keck School of Medicine Los Angeles, CA and Partner, Retina Vitreous Associates Medical Group. “These findings may indicate a huge step forward in the way we approach and treat this condition. We remain optimistic and eager to continue the trial and understand the full potential of OCU400.”

Ocugen will continue to monitor long-term safety and efficacy data from the treated patients and provide additional updates.

A webcast and conference call will take place today at 8:30 a.m. ET:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 7803227

Webcast: Available on the events section of the Ocugen investor site

About Modifier Gene Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, LCA, and Stargardt disease, as well as dry AMD. Our modifier gene therapy platform is based on the use of Nuclear hormone receptors (NHRs), master gene regulators, which have the potential to restore homeostasis — the basic biological processes in the retina. Unlike single-gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks. Currently Ocugen has three modifier gene therapy programs OCU400 (RP, LCA), OCU410 (dry AMD), OCU410ST (Stargardt disease).

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 the potential to improve retinal health and function in patients with inherited retinal diseases.

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 Twitter 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, which are subject to risks and uncertainties, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trial results, and anticipated timing of clinical trial updates and regulatory interactions. 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 the Phase 1/2 clinical trial 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
Tiffany.Hamilton@ocugen.com