NR2E3

Favorablesafetyandtolerabilitypro >ofOCU400investigational drug productin 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 luminancelevel 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 pro a diverse group of subjects aged 18-77 years old, with varied disease stages, racial and ethnic pro 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 pro 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 investorsite AboutModifierGeneTherapy 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). AboutOCU400 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. AboutOcugen,Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at and follow us on Twitter and LinkedIn. CautionaryNoteonForward-LookingStatements Thispressreleasecontainsforward-lookingstatementswithinthemeaningofThePrivateSecuritiesLitigationReformActof1995,whicharesubject torisksanduncertainties,including,butnotlimitedto,statementsregardingqualitative assessments of available data, potential benefits, expectations for ongoing clinical trial results, and anticipated timing of clinical trial updates and regulatory interactions.Wemay,insomecases,usetermssuchas“predicts,”“believes,”“potential,”“proposed,”“continue,”“estimates,”“anticipates,”“expects,” “plans,”“intends,”“may,”“could,”“might,”“will,”“should,”orotherwordsthatconveyuncertaintyoffutureeventsoroutcomestoidentifytheseforward- lookingstatements.Suchstatementsaresubjecttonumerousimportantfactors,risks,anduncertaintiesthatmaycauseactualeventsorresultsto differmateriallyfromourcurrentexpectations,including,butnotlimitedto, 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.TheseandotherrisksanduncertaintiesaremorefullydescribedinourperiodicfilingswiththeSecuritiesand ExchangeCommission(SEC),includingtheriskfactorsdescribedinthesectionentitled“RiskFactors”inthequarterlyandannualreportsthatwe theSEC.Anyforward-lookingstatementsthatwemakeinthispressreleasespeakonlyasofthedateofthispressrelease.Exceptasrequiredby 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

Phalguni Deswal @Phalguni_GD Ocugen announced that the manufacturing facility for its regenerative cell therapy for cartilage, NeoCart, is expected for completion by the end of 2023. Image Credit: Sergey Nivens / Shutterstock. Phase I/II trials for Ocugen ’s gene therapies, OCU410 and OCU410ST, are planned for initiation by the end of 2023 in geographic atrophy and Stargardt disease. The plans follow the US Food and Drug Administration’s (FDA) approval of the Investigational New Drug (IND) application for the two gene therapies. Recommended Reports Reports LOA and PTSR Model - Gene Therapy To Target Bcma and Cd19 in Waldenstrom Macroglobulinemia (Lymph... GlobalData Reports LOA and PTSR Model - Gene Therapy to Target BCMA and CS1 for Relapsed and Refractory Multiple Mye... GlobalData View all Companies Intelligence Ocugen Inc View all Ocugen is also planning for a Phase III trial in retinitis pigmentosa for OCU400 in Q4 2023/Q1 2024. The Phase III trial initiation is contingent on the FDA’s approval of trial plans and Phase I/II data. OCU410 is an adeno-associated virus containing a gene that encodes human retinoic acid receptor (RAR)-related orphan receptor alpha (RORα). It is planned to be investigated in geographic atrophy, which is the chronic degeneration of the macula during the late stages of age-related macular degeneration. OCU410ST was granted the orphan drug designation by the FDA for the treatment of Stargardt disease. Also known as ABCA4 retinopathy, the illness is a rare genetic disorder caused by pathogenic variations in the ABCA4 gene leading to fatty build-up on the macula and loss of vision. OCU400 is an adeno-associated virus serotype 5 capsid containing the gene for human nuclear hormone receptor NR2E3. It is being developed in partnership with China-based CanSinoBIO. It is currently being evaluated in an open-label Phase II trial (NCT05203939). An open-label Phase I trial (NCT05802329) for OCU200 in patients with diabetic macular oedema was initiated in Q2 2023. One of the cohorts in the trial will evaluate the combination therapy of OCU200 and Novartis ’ Lucentis (ranibizumab). Ocugen announced that the manufacturing facility for its regenerative cell therapy for cartilage, NeoCart, is expected for completion by the end of 2023, with a Phase III trial for the therapy planned for initiation in H2 2024. The US-based company is looking for government funding for its inhaled mucosal vaccine platform, with the IND application for the treatment expected to be filled in 2024. As part of their second quarter financials, Ocugen reported cash equivalents of $70.6m on 30 June 2023. The company also reported a loss of $0.10 per common share in Q2 2023. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

MALVERN, Pa., April 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 the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa and Leber congenital amaurosis, OCU410 for the treatment of dry age-related macular degeneration (dry AMD), and OCU410ST for the treatment of Stargardt disease; along with OCU200, a novel biologic candidate to treat diabetic macular edema (DME), at The Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting in New Orleans from April 23-27, 2023. “We are thrilled to share more detail on our unique modifier gene therapy platform, as well as our novel biologics ophthalmic product pipeline with the professional community at ARVO,” commented Arun Upadhyay, PhD, Chief Scientific Officer and Head of Research, Development and Medical at Ocugen. “It is especially exciting to be at ARVO just following positive preliminary safety and efficacy results from the Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa. We look forward to highlighting this most recent news along with the work we are doing across our ophthalmology portfolio to combat hard-to-treat blindness diseases affecting millions of patients globally,” Dr. Upadhyay concluded. Poster Presentation: Title: Modifier Gene Approach Using OCU410 for Dry AMD Therapy: One Gene—Multiple Targets Authors: Dinesh Singh, Mohamed Nsaibia, Sree Kattala, Subechhya Neupane, Matthew Ritts, Arun UpadhyayPresenter: Dinesh Singh, Associate Director, Discovery, Ocugen Presentation Type/Number: Poster Session, 755-C0356Location: Exhibit HallDate: Sunday, April 23, 2023Time: Noon – 1:45 p.m. CDT Exhibitor Presentations (Exhibitor Education Lounge): Title: Ocugen—OCU400—Modifier Gene Therapy for Treatment of Inherited Retinal Diseases: Retinitis Pigmentosa & Leber Congenital AmaurosisPresenter: Arun Upadhyay, PhD, CSO and Head of Research, Development and Medical, OcugenDate: Monday, April 24, 2023Time: 2 p.m. CDT Title: Ocugen—OCU410 & OCU410ST—Nuclear Receptor Gene RORA as a Potential Therapeutic for Dry AMD and Stargardt diseasePresenter: Dinesh Singh, Associate Director, Discovery, Ocugen Date: Tuesday, April 25, 2023Time: 2 p.m. CDT Title: Ocugen—OCU200—A Novel Biologic for the Treatment of DME, DR, and Wet AMDPresenter: Pushpendra Singh, Director, Cell and Gene Therapy, Ocugen Date: Wednesday, April 26, 2023Time: 2 p.m. CDT About OCU400OCU400 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 potential to improve retinal health and function in patients with inherited retinal diseases. These diseases, combined, account for approximately 125,000 cases in the U.S. About OCU410 and OCU410STOCU410 is a modifier gene therapy product candidate being developed for the treatment of dry AMD. OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. Various genes associated with AMD are regulated by RORA. The RORA protein plays an important role in lipid metabolism and demonstrates an anti-inflammatory role, which we believe could be a potential therapeutic candidate for dry AMD based on in-vitro and in-vivo (animal model) studies. Using the same technology as OCU410, Ocugen plans to submit an IND for Stargardt disease, an orphan eye disease, in Q2 2023. About OCU200OCU200 is a novel fusion protein consisting of human transferrin linked to human tumstatin. It exerts anti-proliferative, anti-inflammatory, and anti-oxidative effects by selective targeting to the retinal and choroidal tissues. OCU200 potentially showcases better bioavailability and tissue penetrance than tumstatin alone due to transferrin and provides distinct MOA binding through αVβ3 integrin pathways that can potentially reduce the number of injections for patients. OCU200 can potentially be used for the treatment of DME, diabetic retinopathy, and wet age-related macular degeneration. These diseases, combined, account for approximately 10 million cases in the U.S. 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 Twitter and LinkedIn. Cautionary Note on Forward-Looking StatementsThis 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 HamiltonHead of CommunicationsTiffany.Hamilton@ocugen.com