Latest news with #Stargardt
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]
Malaysian Reserve
13-06-2025
- Business
- Malaysian Reserve
Foundation CEO Provides Expert Testimony to U.S. Senate as Outside Witness on Vision Research Funding
Jason Menzo Urges Robust FY2026 Funding for National Eye Institute and Protection of Its Independent Status COLUMBIA, Md., June 13, 2025 /PRNewswire/ — The Foundation Fighting Blindness, the driving force in the global development of treatments and cures for blinding diseases, today submitted written testimony to the U.S. Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies through its chief executive officer, Jason Menzo, who served as an expert outside witness. In the testimony, Menzo urged the Subcommittee to deliver robust and sustained funding for the National Eye Institute (NEI) in Fiscal Year 2026 and to preserve NEI's status as an independent institute within the National Institutes of Health (NIH). 'NEI is the only federal institute dedicated exclusively to vision research,' said Jason Menzo, CEO of the Foundation Fighting Blindness. 'Its work is restoring sight, advancing precision medicine, and delivering hope to millions of Americans living with retinal diseases. Continued investment is critical to maintaining momentum and achieving breakthroughs for conditions that currently have no cure.' The Foundation's testimony highlights how NEI-supported research has led to transformative discoveries, including the first FDA-approved gene therapy for an inherited disease, and emphasizes NEI's strategic role in public-private collaborations that accelerate treatment development. The Foundation Fighting Blindness and NEI have long shared a close, collaborative partnership to advance high-impact scientific discovery and bring new treatments to patients faster. Vision loss from diseases such as retinitis pigmentosa, macular degeneration, and Stargardt disease currently affects tens of millions and costs the U.S. economy an estimated $134.2 billion annually. The Foundation emphasized that inconsistent funding stalls clinical trials, closes labs, and risks losing top scientific talent—consequences that are difficult and costly to reverse. The testimony also noted that for every dollar invested in NIH-funded research, $2.56 in new economic activity is generated, making this one of the highest-return investments Congress can make. 'We're not just talking about restoring sight—we're talking about protecting America's leadership in biomedical innovation and supporting economic growth in communities nationwide,' added Menzo. The Foundation Fighting Blindness urges Congress to: Provide strong, sustained funding for NEI in the FY2026 Labor-HHS Appropriations bill; and Preserve NEI's status as an independent institute within NIH to ensure focused, strategic investment in vision science. The full testimony is available at About the Foundation Fighting BlindnessEstablished in 1971, the Foundation Fighting Blindness is the world's leading private source of funding for research on retinal degenerative diseases. The Foundation has raised over $954 million toward its mission to prevent, treat, and cure blinding diseases such as retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and more. Visit for more information. Media Contacts:Chris AdamsVice President, Marketing & CommunicationsCAdams@ 423-0585
Yahoo
13-06-2025
- Business
- Yahoo
Foundation CEO Provides Expert Testimony to U.S. Senate as Outside Witness on Vision Research Funding
Jason Menzo Urges Robust FY2026 Funding for National Eye Institute and Protection of Its Independent Status COLUMBIA, Md., June 13, 2025 /PRNewswire/ -- The Foundation Fighting Blindness, the driving force in the global development of treatments and cures for blinding diseases, today submitted written testimony to the U.S. Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies through its chief executive officer, Jason Menzo, who served as an expert outside witness. In the testimony, Menzo urged the Subcommittee to deliver robust and sustained funding for the National Eye Institute (NEI) in Fiscal Year 2026 and to preserve NEI's status as an independent institute within the National Institutes of Health (NIH). "NEI is the only federal institute dedicated exclusively to vision research," said Jason Menzo, CEO of the Foundation Fighting Blindness. "Its work is restoring sight, advancing precision medicine, and delivering hope to millions of Americans living with retinal diseases. Continued investment is critical to maintaining momentum and achieving breakthroughs for conditions that currently have no cure." The Foundation's testimony highlights how NEI-supported research has led to transformative discoveries, including the first FDA-approved gene therapy for an inherited disease, and emphasizes NEI's strategic role in public-private collaborations that accelerate treatment development. The Foundation Fighting Blindness and NEI have long shared a close, collaborative partnership to advance high-impact scientific discovery and bring new treatments to patients faster. Vision loss from diseases such as retinitis pigmentosa, macular degeneration, and Stargardt disease currently affects tens of millions and costs the U.S. economy an estimated $134.2 billion annually. The Foundation emphasized that inconsistent funding stalls clinical trials, closes labs, and risks losing top scientific talent—consequences that are difficult and costly to reverse. The testimony also noted that for every dollar invested in NIH-funded research, $2.56 in new economic activity is generated, making this one of the highest-return investments Congress can make. "We're not just talking about restoring sight—we're talking about protecting America's leadership in biomedical innovation and supporting economic growth in communities nationwide," added Menzo. The Foundation Fighting Blindness urges Congress to: Provide strong, sustained funding for NEI in the FY2026 Labor-HHS Appropriations bill; and Preserve NEI's status as an independent institute within NIH to ensure focused, strategic investment in vision science. The full testimony is available at About the Foundation Fighting BlindnessEstablished in 1971, the Foundation Fighting Blindness is the world's leading private source of funding for research on retinal degenerative diseases. The Foundation has raised over $954 million toward its mission to prevent, treat, and cure blinding diseases such as retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and more. Visit for more information. Media Contacts:Chris AdamsVice President, Marketing & CommunicationsCAdams@ 423-0585 View original content to download multimedia: SOURCE Foundation Fighting Blindness
Yahoo
12-06-2025
- Business
- Yahoo
SpliceBio secures $135m for gene therapy development
SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007, its gene therapy candidate for Stargardt disease. Jointly led by Sanofi Ventures and EQT Life Sciences, the funding round also saw participation from Roche Venture Fund and current investors including Novartis Venture Fund, UCB Ventures, New Enterprise Associates and Ysios Capital. The Series B financing will support the ongoing interventional Phase I/II ASTRA trial and the observational POLARIS study of SB-007. The funds will expedite the development of SpliceBio's AAV gene therapy programme pipeline across fields including neurology, ophthalmology and other undisclosed indications, leveraging the company's protein splicing platform. Stargardt disease is a genetic retinal condition that occurs due to mutations in the ATP-binding cassette subfamily A member 4 (ABCA4) gene, resulting in progressive loss of vision and eventual blindness. SB-007 has received clearance from the Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) to enter clinical development. It aims to deliver a functional copy of the full-length ABCA4 protein, potentially treating individuals with Stargardt disease, irrespective of their specific ABCA4 mutation. Three new members will join the company's board of directors: EQT Life Sciences managing director Daniela Begolo, Sanofi Ventures partner Laia Crespo and Roche Venture Fund head Carole Nuechterlein. SpliceBio co-founder and CEO Miquel Vila-Perelló stated: 'This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our pipeline across ophthalmology, neurology and beyond. 'The support from such high-quality investors underscores the strength of our programmes and our unique protein splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today.' In 2023, SpliceBio signed a licensing agreement with Spark Therapeutics, allowing the latter to use its protein splicing platform and develop a gene therapy for an undisclosed inherited renal disease. "SpliceBio secures $135m for gene therapy development" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
11-06-2025
- Business
- Yahoo
SpliceBio lands $135M for a new kind of eye gene therapy
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. SpliceBio, a Spanish biotechnology company working on next-generation gene therapies, said Wednesday it raised $135 million in a Series B round to advance its lead program in Stargardt disease into further clinical testing. The startup is built around a technology that's meant to overcome a key limitation of current gene therapies. Scores of gene therapy developers use adeno-associated viruses, or AAVs, to deliver genetic cargo into the body. But because of their small size, AAVs can't carry larger corrective genes, leaving many diseases out of reach or forcing companies to come up with creative solutions. One example is in Duchenne muscular dystrophy. Because of the large size of the gene coding for the muscle-protecting protein dystrophin Duchenne patients lack, several companies, like Sarepta Therapeutics, have developed gene therapies that make a shortened form. The approach comes with drawbacks, however, as a truncated version of the protein might not work as well as the original. SpliceBio has a different workaround. The company is using two separate AAVs to send pieces of a large gene into cells. Once inside, specially engineered splicing molecules help reassemble their cargo into full-length proteins. Stargardt disease, for example, is caused by mutations to the ABCA4 gene, which is too large to stuff into an AAV. SpliceBio says its method can help the body produce a functional version of the protein that gene produces. Its lead program, SB-007, is in a Phase 1/2 trial. SpliceBio isn't the only biotech pursuing this type of approach to treat Stargardt, an inherited eye condition that causes progressive vision loss. Another European company, AAVantgarde, has a similar type of program in early-stage testing. SpliceBio's technology is based on research conducted at Princeton University, where co-founder and CEO Miquel Vila-Perelló was studying protein design and engineering. Launched in 2014 under the name ProteoDesign, the biotech is also working on additional programs in ophthalmology, neurology and other undisclosed therapeutic areas. Its Series B round was co-led by EQT Life Sciences and Sanofi Ventures, and involved seven other backers including Roche Venture Fund, New Enterprise Associates and Novartis Venture Fund. "The support from such high-quality investors underscores the strength of our programs and our unique protein splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today,' Vila-Perelló said in a statement. The company raised €50 million in a Series A round in 2022. Gene and cell therapy startups backed by the roughly two dozen investment firms BioPharma Dive tracks have secured a little over $1.1 billion in venture dollars so far in 2025. Half of those fundings have come in the form of 'megarounds' exceeding $100 million apiece, continuing an ongoing trend. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
