Latest news with #OCU410ST
Business Upturn
18-07-2025
- Business
- Business Upturn
Ocugen, Inc. Announces First Patient Dosed in Phase 2/3 GARDian3 Pivotal Confirmatory Trial for OCU410ST—Novel Modifier Gene Therapy Candidate for Stargardt Disease
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 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 HamiltonAVP, Head of Communications [email protected]
Yahoo
16-06-2025
- Business
- Yahoo
Ocugen, Inc. Announces U.S. FDA Clearance of Investigational New Drug Amendment to Initiate Phase 2/3 Pivotal Confirmatory Clinical Trial of OCU410ST—Modifier Gene Therapy Candidate for Stargardt Disease
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 OCU410STOCU410ST 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 Stargardt DiseaseStargardt 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 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 and follow us on X 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, 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 HamiltonAVP, Head of
Associated Press
29-04-2025
- Business
- Associated Press
Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress
MALVERN, Pa., April 29, 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 Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410ST for the treatment of Stargardt disease (Phase 2/3 pivotal confirmatory clinical trial), and OCU410 for the treatment of geographic atrophy (Phase 2 ArMaDa clinical trial), at The Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting at the Calvin L. Rampton Salt Palace Convention Center in Salt Lake City, Utah from May 4-8, 2025, and Retina World Congress at the Marriott Harbor Beach Resort in Ft. Lauderdale, Florida from May 8-11, 2025. 'We look forward to sharing more about the potential of our modifier gene therapy platform and the meaningful results we are seeing in the clinic during these two important meetings for the retina community,' said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. 'Ocugen remains on track to deliver on our commitment to file three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) in the next three years—potentially addressing significant unmet medical need for large patient populations through our gene-agnostic approach.' The ARVO Annual Meeting is a premiere gathering for eye and vision scientists from across the globe, students, and those in affiliated fields to share the latest research findings and collaborate on innovative solutions. Retina World Congress brings together leading retina specialists from every continent to achieve a global scientific and clinical exchange in retinal health. Ocugen's presence in Utah kicks off with the Company Showcase at Eyecelerator, presented by Dr. Huma Qamar, Chief Medical Officer at Ocugen, and continues through presentations and thought leadership engagement at ARVO. Eyecelerator @ Park City 2025 Session: Retina—Gene Therapy and Novel Mechanisms of Action Showcase Location: Grand Hyatt Deer Valley, Strawberry Ballroom, Park City, UT Date: Friday, May 2, 2025 Time: 2:06 p.m. MDT Presenter: Dr. Huma Qamar ARVO Exhibitor Education Forum Two-Year Follow-Up of a Phase 1/2 Clinical Trial for the Safety and Efficacy of OCU400 Novel Modifier Gene Therapy for Retinitis Pigmentosa Location: Exhibitor Floor, Section 1037 Date: Monday, May 5, 2025 Time: 2 p.m. MDT Presenter: Benjamin Bakall, MD, Ph.D., Assistant Clinical Professor, University of Arizona, College of Medicine–Phoenix, and Director for Clinical Research, Director for The Inherited Retinal Disease and Visual Function Clinic, Associated Retina Consultants Safety and Efficacy of OCU410ST: A Phase 1/2 Trial of a Novel Modifier Gene Therapy for Stargardt Disease (GARDian) Location: Exhibitor Floor, Section 1037 Date: Tuesday, May 6, 2025 Time: 2 p.m. MDT Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin An Evaluation of the Safety and Efficacy of Novel Modifier Gene Therapy OCU410 for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration Location: Exhibitor Floor, Section 1037 Date: Wednesday, May 7, 2025 Time: 2 p.m. MDT Presenter: Neena Haider, Ph.D., Faculty Harvard Medical School and Founder, CEO, Shifa Precision Paper Session Preliminary Safety and Efficacy of OCU410 for Treatment of Geographic Atrophy: Phase 1/2 OCU410: The Age-related Macular Degeneration (ArMaDa) Study Update Presentation Number: 3675 Session Number and Title: 358/AMD Clinical research II Location: Ballroom J Date: Tuesday, May 6, 2025 Time: 4:15 p.m. MDT Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin Poster Session A0513: Safety and Efficacy of OCU410ST for the Treatment of Stargardt Disease: Phase 1/2 Study Update Location: Hall A-E Date: Thursday, May 8, 2025 Time: 2 p.m. MDT Presenter: Ramiro Maldonado, MD, Duke Center for Ophthalmic Genetics, Duke Pediatric Retina, Adult vitreo-Retinal diseases Dr. Qamar will represent Ocugen at Retina World Congress to share the Company presentation and serve alongside notable retinal surgeons and industry peers during a panel discussion. Retina World Congress Retina Unplugged Inherited and Rare Retinal Diseases Session Moderators: Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD Location: Grand Ballroom Date: Thursday, May 8, 2025 Time: 10:35 am – 11:12 a.m. EDT Ocugen is committed to bringing game-changing therapies to treat inherited retinal diseases as well as blindness diseases affecting millions to market and working even harder to provide access to patients globally. 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 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 Contact: Tiffany Hamilton AVP, Head of Communications [email protected]
