Tag: OCGN

Research News and Market Data on OCGN

August 13, 2025

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MALVERN, Pa., Aug. 13, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for Stargardt disease and provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application (MAA).

EMA provided this opinion based on safety and tolerability that OCU410ST demonstrated in the Phase 1 GARDian trial, including 48% slower lesion growth and statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up in evaluable treated eyes compared to untreated eyes. The Phase 2/3 study will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The unique adaptive design of this trial includes a masked interim analysis of 24 subjects in the study (16 in treatment group and 8 in control group) at 8 months. The primary objective of the trial is to evaluate the reduction in atrophic lesion size. Key secondary endpoints include improvements in BCVA and low luminance visual acuity (LLVA), compared to controls. Data from the one-year follow-up will be used to support the Company’s planned Biologics License Application (BLA) and MAA in the EU.

“This positive opinion endorses a single trial as the basis for both BLA and MAA submissions and brings us closer to providing a one-time, modifier gene therapy to approximately 100,000 Stargardt patients in the U.S. and Europe combined,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “We are very encouraged about the prospect of addressing the unmet medical need that exists for these patients who currently have no approved treatment options available to them.”

The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. Alignment with the EMA follows recent important milestones for the OCU410ST program, including Rare Pediatric Disease Designation (RPDD) in May, IND clearance in June, and first patient dosing in July. With enrollment scheduled to be complete in the first quarter of 2026 the Company remains on track for a BLA filing in the first half of 2027, aligned with its goal of three BLAs in the next three years.

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 pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

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 therapies to address major blindness diseases 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 address significant unmet medical need for large patient populations through our gene-agnostic approach. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU410ST to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Research News and Market Data on OCGN

August 12, 2025

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MALVERN, Pa., Aug. 12, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the closing of its previously announced registered direct offering pursuant to a securities purchase agreement with Janus Henderson Investors, a global asset management firm, for the purchase and sale of 20,000,000 shares of common stock and warrants to purchase up to an aggregate of 20,000,000 shares of common stock at a purchase price of $1.00 per share (closing price on August 7, 2025) and accompanying warrant. The warrants have an exercise price of $1.50 per share, are exercisable immediately upon issuance, and will expire two years following the date of issuance. The warrants are callable by the Company when the VWAP of the Company’s common stock exceeds $2.50 per share for at least five of a trailing 30 trading day period. 

Noble Capital Markets, Inc. acted as the sole placement agent in connection with the offering. Maxim Group LLC and Titan Partners Group, a division of American Capital Partners, acted as independent financial advisors in connection with the offering.

The gross proceeds to the Company were approximately $20 million before deducting the placement agent fees and other estimated offering expenses, which the Company anticipates will extend the Company’s cash runway into the second quarter of 2026. The Company may receive up to $30 million of additional gross proceeds if the warrants are exercised in full. The Company anticipates that combined offering proceeds of $50 million with warrant exercise will extend the Company’s cash runway into the first quarter of 2027. The offering was made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-278774) previously filed with the U.S. Securities and Exchange Commission (“SEC”), which was declared effective on May 1, 2024. The offering was made only by means of a prospectus forming a part of the effective registration statement relating to the offering. A prospectus supplement relating to the shares of common stock and warrants has been filed by the Company with the SEC. Copies of the prospectus supplement relating to the registered direct offering, together with the accompanying prospectus, can be obtained at the SEC’s website at www.sec.gov or from Noble Capital Markets, Inc., 150 East Palmetto Park Rd., Suite 110, Boca Raton, FL 33432.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About Ocugen, Inc.

Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Statement Regarding 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. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate, including the Company’s expected cash runway, whether the warrants will be exercised and various other factors. 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.

Ocugen Contact:

Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@Ocugen.com

Research News and Market Data on OCGN

August 8, 2025

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MALVERN, Pa., Aug. 08, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that it has entered into a securities purchase agreement with Janus Henderson Investors, a global asset management firm, to purchase 20,000,000 shares of common stock and warrants to purchase up to an aggregate of 20,000,000 shares of common stock at a purchase price of $1.00 per share (closing price on August 7, 2025) and accompanying warrant in a registered direct offering. The warrants have an exercise price of $1.50 per share, are exercisable immediately upon issuance, and will expire two years following the date of issuance. The warrants are callable by the Company when the VWAP of the Company’s common stock exceeds $2.50 per share for at least five of a trailing 30 trading day period. 

Noble Capital Markets, Inc. acting as the sole placement agent in connection with the offering.

The gross proceeds to the Company are expected to be approximately $20 million before deducting the placement agent fees and other estimated offering expenses. The Company may receive up to $30 million of additional gross proceeds if the warrants are exercised in full. The offering is expected to close on or about August 11, 2025, subject to the satisfaction of customary closing conditions. The offering is being made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-278774) previously filed with the U.S. Securities and Exchange Commission (“SEC”), which was declared effective on May 1, 2024. The offering is made only by means of a prospectus forming a part of the effective registration statement relating to the offering. A prospectus supplement relating to the shares of common stock and warrants will be filed by the Company with the SEC. When available, copies of the prospectus supplement relating to the registered direct offering, together with the accompanying prospectus, can be obtained at the SEC’s website at www.sec.gov or from Noble Capital Markets, Inc., 150 East Palmetto Park Rd., Suite 110 Boca Raton, FL 33432.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About Ocugen, Inc.

Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Statement Regarding 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. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate, including the satisfaction of customary closing conditions related to the offering, completion of the offering, whether the warrants will be exercised and various other factors. 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.

Ocugen Contact:

Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@Ocugen.com

Research News and Market Data on OCGN

August 1, 2025

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

• Initiated dosing in OCU410ST Phase 2/3 GARDian3 pivotal confirmatory clinical trial
• Actively dosing patients in OCU400 Phase 3 liMeliGhT clinical trial and on track for 2026 BLA filing
• OrthoCellix reverse merger intended to unlock the value of NeoCart/regenerative cell therapies and enable the Company to focus capital on modifier gene therapy platform
• Signed binding term sheet for exclusive Korean rights to OCU400 with upfront fees and near-term development milestone payments totaling up to $11 million

MALVERN, Pa., Aug. 01, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported second quarter 2025 financial results along with a business update.

“While our modifier gene therapy clinical trials advance—now with two in late-stage—we are securing strategic partnerships and evolving the business to support three successful Biologics License Application (BLA) filings over the next three years,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “We have also made important appointments to our Board of Directors, Retina Scientific Advisory Board, and Leadership Team to provide the Company with scientific and strategic know-how to bring us closer to delivering paradigm-changing gene therapies to millions of people with blindness diseases.”

In June, the Company announced a proposed reverse merger with OrthoCellix, a wholly-owned subsidiary, and Carisma Therapeutics, Inc. to create a Nasdaq-listed, late clinical-stage regenerative cell therapy company with a first-in-class technology platform, focused on orthopedic diseases. The combined company will focus on the development of OrthoCellix’s NeoCart^® technology for the treatment of articular knee cartilage defects. Previously, NeoCart^® received Regenerative Medicine Advanced Therapy (RMAT) designation and concurrence from the U.S. Food and Drug Administration (FDA) on a single, confirmatory Phase 3 clinical trial to enable submission of a BLA.

Aligned with Ocugen’s business development strategy to pursue regional partnerships for OCU400, the Company signed a binding term sheet to negotiate and enter into a licensing agreement with a well-established leader in the pharmaceutical and healthcare sector in Korea for exclusive Korean rights to OCU400. Pursuant to the term sheet, under the license agreement, in addition to the upfront and milestone fees, the Company will be entitled to sales milestones of $1 million for every $15 million of net sales in Korea in addition to a royalty of 25% on net sales of OCU400 generated by Ocugen’s partner. Ocugen will manufacture commercial supply of OCU400 under terms of a supply agreement. A regional approach preserves Ocugen’s rights to larger geographies to maximize total patient reach while also generating return for shareholders.

Following the FDA’s agreement to proceed with a Phase 2/3 GARDian3 pivotal confirmatory trial for OCU410ST for Stargardt disease, the agency granted Rare Pediatric Disease Designation (RPDD) to OCU410ST in May. This designation underscores the urgent need to address Stargardt disease, which remains a significant unmet medical need. Stargardt disease is an inherited retinal disorder that typically presents in childhood and affects approximately 100,000 people in the U.S. and Europe combined, and approximately 1 million globally. Currently, there is no FDA-approved treatment available for Stargardt disease.

The OCU410ST Phase 2/3 GARDian3 clinical trial is progressing well with the first patient dosed in July after FDA clearance in June. The GARDian3 clinical trial builds upon encouraging results and positive data from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow-up in evaluable treated eyes compared to untreated eyes. Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up when compared to untreated eyes.

Positive preliminary efficacy and safety data from the OCU410 Phase 1 ArMaDa clinical trial at 12 months demonstrated no drug-related serious adverse events (SAEs), 23% slower geographic atrophy (GA) lesion growth in treated eyes versus fellow eyes after a single injection, and 2-line/10-letter gain in visual acuity in treated eyes when compared to untreated fellow eyes. Preliminary results from ongoing Phase 2 clinical trial (N=31), 6-month interim analysis, demonstrated a 27% slower lesion growth and preservation of retinal tissue. These data support the potential for OCU410 to provide a one-time treatment for life for the 2-3 million people in the U.S. & EU combined who suffer from GA.

Patients are actively being recruited in the United States and Canada for the OCU400 Phase 3 liMeliGhT clinical trial, which remains on track for BLA and MAA submissions in 2026. This is the only broad retinitis pigmentosa (RP) gene-agnostic trial to address multiple genetic mutations with a single therapeutic approach. In addition, the European Medicines Agency has granted eligibility to submit the OCU400 Marketing Authorization Application (MAA) through the centralized procedure, based on the current study design and statistical analysis plan.

Regarding the Company’s inhaled vaccines portfolio, the National Institute of Allergy and Infectious Diseases (NIAID) intends to initiate the Phase 1 clinical trial for OCU500 in the third quarter of 2025.

In addition to the notable leadership appointments, Ocugen welcomed the National Security Commission on Emerging Biotechnology (NSCEB) and U.S. Rep. Chrissy Houlahan to its manufacturing facility as part of the NSCEB’s Biotech Across America events, highlighting biotech innovation in Pennsylvania. Rep. Houlahan subsequently announced the bipartisan BIOTech Caucus to build greater awareness and understanding of biotechnology among lawmakers and support transformative advances in healthcare. Dr. Musunuri supports the formation of this very important bipartisan BIOTech Caucus that includes senior congressional leaders such as Rep. Pete Sessions in addition to local leaders, which will prioritize biotechnology at the national level to ensure U.S. leadership globally.

“The meaningful progress Ocugen is making across its novel modifier gene therapy platform, along with strategic leadership changes and significant external alliances are evidence of a strong first half of 2025,” said Dr. Musunuri. “We look forward to providing critical program updates and data in the coming months.”

Modifier Gene Therapy Platform—a Novel First-in-Class Platform

• OCU400 for RP – On track to complete enrollment in support of BLA/MAA filings in 2026. Data and Safety Monitoring Board (DSMB) convened and found no SAEs related to OCU400 and recommended to continue study dosing as planned.
• OCU410ST for Stargardt Disease – FDA granted RPDD for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. FDA cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST and dosing has been initiated.
• OCU410 for GA – Phase 1 data at 12 months demonstrates reduced lesion growth, preservation of retinal tissue, and—most importantly—a positive effect on the functional visual measure of low luminance visual acuity (LLVA). Interim Phase 2 data at 6 months demonstrated very encouraging results consistent with Phase 1 data.

Ophthalmic Biologic Product

• OCU200 – DSMB approved continuation of dosing in the third cohort and the Company intends to complete the Phase 1 clinical trial in the second half of 2025.

Second Quarter 2025 Financial Results

• The Company’s cash, cash equivalents, and restricted cash totaled $27.3 million as of June 30, 2025, compared to $58.8 million as of December 31, 2024, providing cash runway into the first quarter of 2026. The Company had 292.2 million shares of common stock outstanding as of June 30, 2025.
• Total operating expenses for the three months ended June 30, 2025 were $15.2 million and included research and development expenses of $8.4 million and general and administrative expenses of $6.8 million. This compares to total operating expenses for the three months ended June 30, 2024 of $16.6 million that included research and development expenses of $8.9 million and general and administrative expenses of $7.7 million.
• Ocugen reported a $0.05 net loss per common share for the three months ended June 30, 2025, compared to a $0.06 net loss per common share for the three months ended June 30, 2024.

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 executive leadership team will host the call, which will be open to all listeners. There also will be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call or webcast:
Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 9627149
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 pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. 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, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s 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; Ocugen’s financial condition and expected cash runway into the first quarter of 2026, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, and Ocugen’s negotiations regarding the license agreement with a Korean partner and Ocugen’s potential merger transaction regarding the OrthoCellix business, 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; that a definitive agreement for the license with a Korean partner will be delayed or not executed at all, or that, if executed, it may not be on terms anticipated; that the OrthoCellix merger transaction may not close or, if closed, may not result in the benefits anticipated. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

View full release here.

Research News and Market Data on OCGN

July 21, 2025

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MALVERN, Pa., July 21, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced updates to its Retina Scientific Advisory Board (SAB) and Executive Leadership Team to enhance external guidance from key opinion leaders and strengthen the Company’s internal expertise in critical functions and as it pursues its goal of three BLAs in the next three years.

“As our novel modifier gene therapy programs continue to progress and demonstrate positive data in the clinic, it is now more important than ever to solidify an optimized Retina SAB reflecting the absolute best guidance in this space as we move closer to commercialization,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Additionally, it’s imperative that we ensure our leadership team includes top talent to most effectively execute our corporate strategy.”

Three renowned retinal surgeons who are at the forefront of research and cutting-edge advancements for retinal disease are joining the Retina SAB to help the Company bring meaningful, innovative therapeutic options for patients living with serious retina diseases.

New SAB members include:

Jeffrey S. Heier, MD, Director of the Vitreoretinal Service, and Director of Retina Research at Ophthalmic Consultants of Boston. Dr. Heier is one of the leading retinal clinical researchers in the country for new treatments in exudative and non-exudative macular degeneration, diabetic macular edema, venous occlusive disease, vitreoretinal surgical techniques and instrumentation, and diagnostic imaging of the retina. Dr. Heier has contributed extensively to the ophthalmic literature, having authored or co-authored many of the landmark publications in the retinal literature, including the New England Journal of Medicine, Lancet, and numerous other journals.

Peter K Kaiser, MD, Chaney Family Endowed Chair in Ophthalmology Research, Cole Eye Institute, Cleveland Clinic and Professor of Ophthalmology Case Western Reserve School of Medicine. Dr. Kaiser is the director of the Center for Ocular Research and Evaluation (CORE) and is a major contributor to medical literature having authored seven textbooks, 30 book chapters, and more than 400 peer-reviewed manuscripts. He is Associate Editor of International Ophthalmology Clinics and serves on the editorial boards of American Journal of Ophthalmology, Retina, Retina Today, and Ocular Surgery News.

Arshad M. Khanani, MD, MA, FASRS, Managing Partner, Director of Clinical Research, and Director of Fellowship at Sierra Eye Associates and Clinical Professor at the University of Nevada, Reno School of Medicine. In 2021, Dr. Khanani founded the Clinical Trials at the Summit meeting to foster discussion on clinical trial design and data. Dr. Khanani has been recognized among the top 10 researchers globally on The Ophthalmologist Power List 2025 and has received numerous prestigious awards, including the Macula Society’s Lawrence J. Singerman Medal in 2025 and the American Society of Retina Specialists Presidential Award.

Drs. Heier, Kaiser, and Khanani join SAB chair, Lejla Vajzovic, MD, FASRS, Director of the Duke Surgical Vitreoretinal Fellowship Program and Professor of Ophthalmology, Pediatrics, and Biomedical Engineering with Tenure at Duke University Eye Center; and existing SAB members David S. Boyer, MD, Senior Partner at Retina-Vitreous Associates Medical Group and Adjunct Clinical Professor of Ophthalmology at the University of Southern California/Keck School of Medicine, and Carl D. Regillo, MD, FACS, Professor of Ophthalmology at the Sidney Kimmel Medical College at Thomas Jefferson University, Chief of the Retina Service at Wills Eye Hospital, and founder and former director of the Wills Eye Clinical Retina Research Unit.

To optimize Ocugen’s R&D and clinical efforts and build upon positive momentum pursuing strategic partnerships and developing commercial strategy, the Company has made notable leadership appointments.

Vijay Tammara, PhD, has joined Ocugen in the newly created position of Chief Development Officer and brings over 32 years of global regulatory leadership with deep expertise in biotechnology, biosimilars, 505(b)(2), and complex regulatory submissions. Dr. Tammara has made significant contributions to the approval of nine Marketing Authorizations (MAs)—Biologics License Applications and New Drug Applications (NDAs), two biosimilar MAs in emerging markets, three Abbreviated New Drug Applications, 12 Orphan Drug Designations, seven Qualified Infectious Drug Product Designations, and filed over 62 investigational NDAs with clearance in the first review cycle with no clinical holds. He has successfully led regulatory strategy and operations across the U.S., EU, Latin America, and Asia—including Japan, China, and South Korea. Prior to joining Ocugen, he held senior advisory and leadership roles at the FDA and life sciences companies including Sanofi, Wyeth/Pfizer, and Merck, in addition to leading his own consulting firm.

Abhi Gupta, MBA, has been named Executive Vice President, Commercial and Business Development, following the retirement of Mike Shine. Abhi has more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry. He has led commercialization planning for transformative rAAV gene therapies and played a pivotal role in building Pfizer’s $5.5B gene therapy portfolio across neuromuscular, hematologic, and cardiovascular indications. Before joining Ocugen, Mr. Gupta served as SVP and Head of Cell and Gene Therapies at Syneos Health and previously held leadership roles at Pfizer, Regeneron, and Johnson & Johnson, with a track record of successful product launches, strategic partnerships, and business development initiatives. 

Ocugen is honored to partner with this distinguished group of advisors and delighted to welcome Dr. Tammara and Mr. Gupta as the Company sharpens its patient-centric focus to potentially deliver paradigm-changing gene therapies.

About Ocugen, Inc.
Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Research News and Market Data on OCGN

July 18, 2025

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MALVERN, Pa., July 18, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies).

“Dosing the first patient is an especially significant milestone and brings us closer to our goal of addressing the unmet medical need that exists for all Stargardt patients—100,000 in the U.S. and Europe and 1 million worldwide,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Progressing our second modifier gene therapy candidate into a registration clinical trial is a pivotal step in potentially providing a one-time therapy for life for the millions of patients affected by inherited retinal diseases.”

The Phase 2/3 clinical trial for OCU410ST builds upon encouraging results and positive data from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow up in evaluable treated eyes compared to untreated eyes. Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line gain in best corrected visual acuity (BCVA) at 12-month follow-up when compared to untreated eyes.

“Initiating dosing in this pivotal Phase 2/3 study is an important advancement for Ocugen and more importantly for the Stargardt community,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “The adaptive design of this trial, including a masked interim analysis at 8 months on 24 subjects, enables us to efficiently evaluate early signals of efficacy and safety while optimizing study conduct. This ensures we generate robust and meaningful data to support our regulatory submissions for approvals.”

“Treating the first patient with this novel gene therapy in the GARDian3 trial is a proud and hopeful moment for our team and for families affected by Stargardt disease,” said Victor H. Gonzalez, MD, Principal Investigator and retinal surgeon at Valley Retina Institute, McAllen, Texas. “For decades, patients have faced the progressive loss of central vision with no approved treatment options. The encouraging Phase 1 results give us confidence that OCU410ST could meaningfully slow disease progression and help preserve vision. This trial brings us closer to the possibility of a one-time gene therapy that could transform patients’ quality of life for years to come.”

OCU410ST maintains a favorable safety and tolerability profile with no serious adverse events or adverse events of special interest, including ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization.

The Phase 2/3 study will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The primary objective of the trial is to evaluate the reduction in atrophic lesion size. Key secondary endpoints include improvements in BCVA and low luminance visual acuity (LLVA), compared to controls. Data from the one-year follow-up will be used to support the company’s planned Biologics License Application (BLA).

The OCU410ST Phase 2/3 pivotal confirmatory trial represents Ocugen’s second late-stage clinical program. Ocugen plans to submit a BLA for OCU410ST in 2027 in alignment with its strategic goal of filing three BLAs over the next three years.

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 pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

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 therapies to address major blindness diseases 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 address significant unmet medical need for large patient populations through our gene-agnostic approach. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU410ST to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Research News and Market Data on OCGN

July 17, 2025

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MALVERN, Pa., July 17, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that it will host a conference call and live webcast to discuss the Company’s second quarter 2025 financial results and provide a business update at 8:30 a.m. ET on Friday, August 1, 2025.

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: 9627149
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 pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Research News and Market Data on OCGN

June 23, 2025

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• Proposed reverse merger with OrthoCellix, a wholly-owned subsidiary of Ocugen, to create Nasdaq-listed, late clinical-stage regenerative cell therapy company with a first-in-class technology platform, focused on orthopedic diseases
• OrthoCellix is developing the Phase 3-ready NeoCart® as an autologous cartilage implant technology utilizing patient cells to repair articular cartilage defects of the knee

PHILADELPHIA and MALVERN, Pa., June 23, 2025 (GLOBE NEWSWIRE) — Carisma Therapeutics Inc. (Nasdaq: CARM) (Carisma) and OrthoCellix, Inc. (OrthoCellix), a wholly-owned subsidiary of Ocugen, Inc. (Nasdaq: OCGN) (Ocugen), a clinical-stage company developing regenerative cell therapies for orthopedic diseases, today jointly announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on the development of OrthoCellix’s NeoCart® technology for the treatment of knee articular cartilage defects and plans to initiate a U.S. Food and Drug Administration (FDA)-endorsed Phase 3 clinical trial for NeoCart®.

“We believe merging OrthoCellix with Carisma will allow us to create a publicly-traded company focused on the development of NeoCart® and provide value for both Ocugen and Carisma stockholders while unlocking true market potential of NeoCart®,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-founder of Ocugen. “We believe NeoCart® has tremendous potential to deliver a truly transformative approach to cartilage repair, and we’ve established OrthoCellix with dedicated resources to bring this revolutionary technology to the patients who desperately need it.”

“Carisma evaluated a range of strategic alternatives, and we believe this proposed transaction represents an opportunity to deliver significant value to our stockholders,” said Steven Kelly, President and Chief Executive Officer, of Carisma. “OrthoCellix is strongly positioned with its NeoCart® platform, a dedication to developing regenerative cell therapies, and a well-credentialed management team to lead the combined company.”

About OrthoCellix’s NeoCart® Portfolio

OrthoCellix is developing NeoCart® as an autologous cartilage implant technology utilizing patient cells to repair articular cartilage defects of the knee. The novel platform merges a fortified 3D scaffold and patented bioprocessing technology to grow chondrocytes—the cells responsible for maintaining cartilage health—to produce adolescent-like cartilage at the time of implant. NeoCart® has the potential to accelerate healing and reduce pain by creating a similar, functional joint surface to help patients return to normal activities and prevent complications associated with articular cartilage damage.

OrthoCellix anticipates launching its Phase 3 clinical trial by the end of 2025. Previously, NeoCart® received Regenerative Medicine Advanced Therapy (RMAT) designation and concurrence from the FDA on a single, confirmatory Phase 3 clinical trial to enable submission of a Biologics License Application.

About the Proposed Transactions

Under the terms of the merger agreement, OrthoCellix will merge with and into a wholly-owned subsidiary of Carisma, with OrthoCellix continuing as a wholly-owned subsidiary of Carisma and the surviving company of the Merger. Carisma will issue to the pre-merger OrthoCellix stockholder shares of Carisma common stock as merger consideration in exchange for the cancellation of shares of capital stock of OrthoCellix. Carisma also expects to enter into subscription agreements for a private financing with Ocugen and other select investors, which is expected to close concurrently with the completion of the merger, to enable the combined company to complete the Phase 3 trial of NeoCart® without any additional cost or investment from Ocugen. In connection with the closing of the proposed transactions, Carisma stockholders will be issued contingent value rights representing the right to receive certain payments from proceeds received by the combined company, if any, related to Carisma’s pre-transaction legacy assets.

Under the terms of the merger agreement, upon the closing of the proposed transactions and after giving effect to the contemplated $25.0 million concurrent financing, OrthoCellix’s stockholder and the other participants in the concurrent financing are expected to own approximately 90% of the combined company, and existing Carisma stockholders are expected to own approximately 10% of the combined company, each on a fully diluted basis. The percentage of the combined company that each company’s former stockholders will own after completion of the merger is subject to adjustment based on Carisma’s net cash at the closing and the proceeds from the concurrent financing, among other adjustments, in each case as described in the merger agreement.

Upon the closing of the proposed transactions, “Carisma Therapeutics Inc.” is expected to be renamed “OrthoCellix, Inc.” and trade on the Nasdaq Capital Market under the ticker symbol ‘OCLX.’

The transaction has been unanimously approved by the board of directors of both companies and is expected to close in the second half of 2025, subject to customary closing conditions, including approvals by the stockholders of each company and the effectiveness of a registration statement to be filed with the Securities and Exchange Commission (the “SEC”) to register the shares of Carisma common stock to be issued in connection with the merger. In connection with the companies’ entry into the merger agreement, directors and officers of Carisma and OrthoCellix’s stockholder have executed support agreements, pursuant to which they have agreed to vote all of their shares of capital stock in favor of the merger or the issuance of Carisma equity in the merger, as applicable.

Advisors

Wilmer Cutler Pickering Hale and Dorr LLP is serving as legal counsel to Carisma and Lucid Capital Markets, LLC is providing a fairness opinion to Carisma’s board of directors. Chardan Capital Markets LLC is serving as M&A advisor and co-placement agent to OrthoCellix and Ocugen. Lake Street Capital Markets, LLC is co-placement agent to OrthoCellix, as a subsidiary of Ocugen, Goodwin Procter LLP is serving as legal counsel to Ocugen and OrthoCellix, and Paul Hastings LLP is serving as legal counsel to the placement agents.

About OrthoCellix

OrthoCellix is a regenerative cell therapy company dedicated to developing a first-in-class technology platform focused on cartilage defects and other orthopedic diseases to address considerable unmet medical needs. The lead program within OrthoCellix is NeoCart® with revolutionary 3D cell therapy technology designed to repair and restore articular cartilage defects in the knee. The Company has a pipeline of additional treatments based on its proprietary scaffold bioreactor and adhesive. OrthoCellix will utilize the Good Manufacturing Practice facility established by Ocugen to support OrthoCellix’s initial development of NeoCart®.

About Carisma Therapeutics

Carisma Therapeutics is a biotechnology company pioneering macrophage engineering to develop groundbreaking therapies for fibrosis and cancer. With a strong commitment to patient-centric innovation, Carisma aims to deliver scalable, next-generation solutions that transform treatment paradigms. Carisma is headquartered in Philadelphia, PA. For more information, please visit www.Carismatx.com.

Cautionary Note on Forward- Looking Statements

Certain statements in this communication, other than purely historical information, may constitute “forward-looking statements” within the meaning of the federal securities laws, including for purposes of the “safe harbor” provisions under the Private Securities Litigation Reform Act of 1995, concerning Carisma, OrthoCellix, the proposed financing and the proposed merger between Carisma and OrthoCellix (collectively, the “Proposed Transactions”) and other matters. These forward-looking statements include, but are not limited to, express or implied statements relating to Carisma’s and OrthoCellix’s management teams’ expectations, hopes, beliefs, intentions or strategies regarding the future including, without limitation, statements regarding: the structure, timing and completion of the proposed merger by and between Carisma and OrthoCellix; the Proposed Transactions and the expected effects, perceived benefits or opportunities of the Proposed Transactions; the combined company’s listing on Nasdaq after the closing of the Proposed Transactions; expectations regarding the structure, timing and completion of a concurrent financing, including investment amounts from investors, timing of closing of the Proposed Transactions, expected proceeds, expectations regarding the use of proceeds, and impact on ownership structure; the anticipated timing of the closing; the expected executive officers and directors of the combined company; each company’s and the combined company’s expected cash position at the closing and cash runway of the combined company following the proposed merger and any private financing; the future operations of the combined company, including research and development activities; the nature, strategy and focus of the combined company; the development and commercial potential and potential benefits of any product candidates of the combined company, including expectations around market exclusivity and intellectual property protection; anticipated clinical drug development activities and related timelines, including the expected timing for announcement of data and other clinical results; expectations regarding or plans for discovery, preclinical studies, clinical trials and research and development programs, in particular with respect to NeoCart®, and any developments or results in connection therewith, including the target product profile of NeoCart®; the anticipated timing of the commencement of and results from those studies and trials; the sufficiency of post-transaction resources to support the advancement of OrthoCellix’s pipeline through certain milestones and the time period over which OrthoCellix’s post-transaction capital resources will be sufficient to fund its anticipated operations; the cash balance of the combined entity at closing; expectations related to the anticipated timing of the closing of the Proposed Transactions (the “Closing”); the expectations regarding the ownership structure of the combined company; the expected trading of the combined company’s stock on Nasdaq under the ticker symbol “OCLX” after the Closing; and other statements that are not historical fact. All statements other than statements of historical fact contained in this communication are forward-looking statements. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “opportunity,” “potential,” “milestones,” “pipeline,” “can,” “goal,” “strategy,” “target,” “anticipate,” “achieve,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “plan,” “possible,” “project,” “should,” “will,” “would” and similar expressions (including the negatives of these terms or variations of them) may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements are made based on current expectations, estimates, forecasts, and projections, as well as the beliefs and assumptions of management, concerning future developments and their potential effects. There can be no assurance that future developments affecting Carisma, OrthoCellix, or the Proposed Transactions will be those that have been anticipated.

These forward-looking statements involve a number of risks and uncertainties, some of which are beyond Carisma’s or OrthoCellix’s control, or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the conditions to the Closing or consummation of the Proposed Transactions are not satisfied, including the failure to timely obtain approval of the proposed reverse stock split from Carisma’s stockholders and the proposed merger from both Carisma’s and OrthoCellix’s stockholders, if at all; the risk that the proposed concurrent financing is not completed in a timely manner, if at all; uncertainties as to the timing of the consummation of the Proposed Transactions and the ability of each of Carisma and OrthoCellix to consummate the Proposed Transactions; risks related to Carisma’s continued listing on Nasdaq until closing of the Proposed Transactions and the combined company’s ability to remain listed following the Closing; risks related to Carisma’s and OrthoCellix’s ability to correctly estimate their respective operating expenses and their respective expenses associated with the Proposed Transactions, as applicable, pending the Closing, as well as uncertainties regarding the impact any delay in the Closing would have on the anticipated cash resources of the combined company, and other events and unanticipated spending and costs that could reduce the combined company’s cash resources; risks related to the failure or delay in obtaining required approvals from any governmental or quasi-governmental entity necessary to consummate the Proposed Transactions; the occurrence of any event, change or other circumstance or condition that could give rise to the termination of the merger agreement; the effect of the announcement or pendency of the merger on Carisma’s or OrthoCellix’s business relationships, operating results and business generally; costs related to the merger; the risk that as a result of adjustments to the exchange ratio, OrthoCellix stockholders and Carisma stockholders could own more or less of the combined company than is currently anticipated; risks related to the market price of Carisma’s common stock relative to the value suggested by the exchange ratio; the uncertainties associated with OrthoCellix’s NeoCart® portfolio, as well as risks associated with the clinical development and regulatory approval of product candidates, including potential delays in the completion of clinical trials; risks related to the inability of the combined company to obtain sufficient additional capital to continue to advance these product candidates; uncertainties in obtaining successful clinical results for product candidates and unexpected costs that may result therefrom; risks related to the failure to realize any value from product candidates being developed and anticipated to be developed in light of inherent risks and difficulties involved in successfully bringing product candidates to market; the outcome of any legal proceedings that may be instituted against Carisma, OrthoCellix or any of their respective directors or officers related to the Proposed Transactions; the ability of Carisma and OrthoCellix to obtain, maintain, and protect their respective intellectual property rights; competitive responses to the Proposed Transactions; costs of the Proposed Transactions and unexpected costs, charges or expenses resulting from the Proposed Transactions; potential adverse reactions or changes to business relationships, operating results, and business generally, resulting from the announcement or completion of the Proposed Transactions; changes in regulatory requirements and government incentives; risks associated with the possible failure to realize, or that it may take longer to realize than expected, certain anticipated benefits of the Proposed Transactions, including with respect to future financial and operating results, legislative, regulatory, political and economic developments, and those uncertainties and factors; and the risk of involvement in litigation, including securities class action litigation, that could divert the attention of the management of Carisma or the combined company, harm the combined company’s business and may not be sufficient for insurance coverage to cover all costs and damages, among others. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties. These and other risks and uncertainties are more fully described in periodic filings with the SEC, including the factors described in the section titled “Risk Factors” in Carisma’s Annual Report on Form 10-K for the year ended December 31, 2024, which was originally filed with the SEC on March 31, 2025, as amended by Amendment No. 1 to the Annual Report on Form 10-K/A, which was filed with the SEC on April 29, 2025, subsequent Quarterly Reports on Form 10-Q filed with the SEC, and in other filings that Carisma makes and will make with the SEC in connection with the Proposed Transactions, including the Form S-4 and Proxy Statement described below under “Additional Information and Where to Find It”, as well as discussions of potential risks, uncertainties, and other important factors included in other filings by Carisma from time to time, any risk factors related to Carisma or OrthoCellix made available to you in connection with the Proposed Transactions, as well as risk factors associated with companies, such as OrthoCellix, that operate in the biopharma industry. Should one or more of these risks or uncertainties materialize, or should any of Carisma’s or OrthoCellix’s assumptions prove incorrect, actual results may vary in material respects from those projected in these forward-looking statements. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements in this communication, which speak only as of the date they are made and are qualified in their entirety by reference to the cautionary statements herein. Neither Carisma nor OrthoCellix undertakes or accepts any duty to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based, except as required by law. This communication does not purport to summarize all of the conditions, risks and other attributes of an investment in Carisma or OrthoCellix.

No Offer or Solicitation

This communication and the information contained herein is not intended to and does not constitute (i) a solicitation of a proxy, consent or approval with respect to any securities or in respect of the Proposed Transactions or (ii) an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities pursuant to the Proposed Transactions or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended, and otherwise in accordance with applicable law, or an exemption therefrom. Subject to certain exceptions to be approved by the relevant regulators or certain facts to be ascertained, the public offer will not be made directly or indirectly, in or into any jurisdiction where to do so would constitute a violation of the laws of such jurisdiction, or by use of the mails or by any means or instrumentality (including without limitation, facsimile transmission, telephone and the internet) of interstate or foreign commerce, or any facility of a national securities exchange, of any such jurisdiction.

NEITHER THE SEC NOR ANY STATE SECURITIES COMMISSION HAS APPROVED OR DISAPPROVED OF THE SECURITIES OR DETERMINED IF THIS COMMUNICATION IS TRUTHFUL OR COMPLETE.

Important Additional Information about the Proposed Transactions Will be Filed with the SEC

This communication relates to the proposed merger involving Carisma and OrthoCellix and may be deemed to be solicitation material in respect of the proposed merger. In connection with the Proposed Transactions, Carisma intends to file relevant materials with the SEC, including a registration statement on Form S-4 (the “Form S-4”) that will contain a proxy statement (the “Proxy Statement”) and prospectus. This communication is not a substitute for the Form S-4, the Proxy Statement or for any other document that Carisma may file with the SEC and/or send to Carisma’s stockholders in connection with the proposed merger. CARISMA URGES, BEFORE MAKING ANY VOTING DECISION, INVESTORS AND STOCKHOLDERS TO READ THE FORM S-4, THE PROXY STATEMENT AND ANY OTHER RELEVANT DOCUMENTS THAT MAY BE FILED WITH THE SEC, AS WELL AS ANY AMENDMENTS OR SUPPLEMENTS TO THESE DOCUMENTS, CAREFULLY AND IN THEIR ENTIRETY IF AND WHEN THEY BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT CARISMA, ORTHOCELLIX, THE PROPOSED TRANSACTIONS AND RELATED MATTERS.

Investors and stockholders will be able to obtain free copies of the Form S-4, the Proxy Statement and other documents filed by Carisma with the SEC (when they become available) through the website maintained by the SEC at www.sec.gov. Copies of the documents filed by Carisma with the SEC will also be available free of charge on Carisma’s website at www.carismatx.com, or by contacting Carisma’s Investor Relations at investors@Carismatx.com. In addition, investors and stockholders should note that Carisma communicates with investors and the public using its website at https://ir.Carismatx.com/.

Participants in the Solicitation

Carisma, OrthoCellix, and their respective directors and certain of their executive officers and other members of management may be deemed to be participants in the solicitation of proxies from Carisma’s stockholders in connection with the Proposed Transactions under the rules of the SEC. Information about Carisma’s directors and executive officers, including a description of their interests in Carisma, is included in Carisma’s most recent Annual Report on Form 10-K for the year ended December 31, 2024, which was filed with the SEC on March 31, 2025, as amended by Amendment No. 1 to the Annual Report on Form 10-K, which was filed with the SEC on April 29, 2025. Additional information regarding the persons who may be deemed participants in the proxy solicitations, including about the directors and executive officers of OrthoCellix, and a description of their direct and indirect interests, by security holdings or otherwise, will also be included in the Form S-4, the Proxy Statement and other relevant materials to be filed with the SEC when they become available. These documents can be obtained free of charge from the sources indicated above.

Research News and Market Data on OCGN

June 16, 2025

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MALVERN, Pa., June 16, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies). The FDA previously granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

“We have had a highly productive and collaborative engagement with the FDA’s Center for Biologics Evaluation and Research (CBER) in establishing the pivotal confirmatory trial for OCU410ST,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “It’s evident that there is a real sense of urgency by the agency in providing treatment options for patients who currently have nothing available to them. As we initiate the Phase 2/3 registration trial, we are expediting the clinical development of OCU410ST by two to three years and potentially providing an innovative gene therapy to patients desperate for a treatment option.”

Positive data from the Phase 1 GARDian trial for OCU410ST demonstrated:

• A favorable safety and tolerability profile with no serious adverse events related to OCU410ST, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization and no adverse events of special interest
• Considerably slower lesion growth—48% at 12-month follow up in evaluable treated eyes when compared to untreated eyes
• Statistically significant (p=0.031) improvement with clinically meaningful, nearly 2-line gain in visual function (BCVA) at 12-month follow-up in evaluable treated eyes when compared to untreated eyes

The Phase 2/3 clinical trial for OCU410ST will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The primary objective of the trial is to evaluate the reduction in atrophic lesion size. Key secondary endpoints include improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA), compared to controls. Data from the one-year follow-up will be used to support the company’s Biologics License Application (BLA).

“The initiation of this pivotal Phase 2/3 study represents a significant milestone in our commitment to bringing transformative genetic therapies to individuals affected by Stargardt disease—a progressive and debilitating condition,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “The recent RPDD granted by the FDA for this program further underscores the urgent need for innovative treatment options for children living with Stargardt disease. OCU410ST, developed through our proprietary modifier gene therapy platform, is designed to target the underlying biological mechanisms of the disease.”

Approximately 100,000 patients in U.S. and Europe combined and 1 million patients globally live with Stargardt disease. Stargardt and ABCA4-associated retinopathies are genetically complex, involving more than 1,200 known mutations and addressing this condition with traditional gene therapy or gene editing approaches remains highly challenging.

“Stargardt disease represents a significant unmet medical need, particularly among children and young adults,” said Lejla Vajzovic, MD, FASRS, Director of the Duke Surgical Vitreoretinal Fellowship Program and Professor of Ophthalmology, Pediatrics, and Biomedical Engineering with Tenure at Duke University Eye Center. “The Phase 2/3 study of OCU410ST is thoughtfully designed with scientific rigor and a patient-centered focus to evaluate both structural and functional outcomes. We are optimistic that this approach will move us closer to a meaningful therapeutic solution for affected families.”

The OCU410ST Phase 2/3 pivotal confirmatory trial represents a major advancement as Ocugen’s second late-stage clinical program. Ocugen plans to submit a BLA for OCU410ST in 2027 in alignment with its strategic goal of filing three BLAs over the next three years.

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 pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

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 therapies to address major blindness diseases 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 address significant unmet medical need for large patient populations through our gene-agnostic approach. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU410ST to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Research News and Market Data on OCGN

May 27, 2025

PDF Version

MALVERN, Pa., May 27, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the United States Food and Drug Administration (U.S. FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. Previously, OCU410ST received Orphan Drug designations for the treatment of ABCA4-associated retinopathies from the FDA and European Medicines Agency.

“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This inherited retinal disease presents itself most often in childhood—making Stargardt disease a diagnosis that not only affects the patient but impacts the entire family.”

The FDA grants RPDD for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S. There are approximately 100,000 people in the U.S. and Europe combined living with Stargardt disease.

With this designation for OCU410ST, Ocugen may be awarded a Priority Review Voucher (PRV), if the PRV program is reauthorized by the U.S. Congress. The PRV program is designed to incentivize drug development for serious rare pediatric diseases. If awarded, a PRV can be redeemed to receive priority review for a different product or sold to another sponsor and typically sells for about $100 million.

Ocugen is committed to advancing the OCU410ST program through clinical development and plans to initiate the Phase 2/3 pivotal confirmatory trial in the next few weeks with a target Biologics License Application (BLA) filing in 2027.

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 pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

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 pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. Discover more at www.ocugen.com and follow us X and LinkedIn.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU410ST to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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