Retinitis Pigmentosa

PHOENIX, Oct. 30, 2024 (GLOBE NEWSWIRE) -- A Race Against Blindness, a nonprofit organization dedicated to funding sight-saving research for children, is pleased to announce a $1 million grant to Axovia Therapeutics to support the company’s lead program, AXV-101. This marks an important step toward developing a treatment to address blindness and obesity associated with Bardet-Biedl Syndrome 1 (BBS1), a rare genetic disorder with no current curative options. The funding from A Race Against Blindness will directly support Axovia's near-term milestones associated with the initiation of the clinical study and initial patient enrollment in the United Kingdom in mid-2025 to treat blindness caused by BBS1 mutations. Axovia Therapeutics’ future plans include initiation of a clinical trial in the United States and progression of the program to also treat obesity. BBS is a devastating genetic disorder associated with primary cilia dysfunction. One of the hallmarks is retinal degeneration commonly presenting as retinitis pigmentosa. The other hallmark is hyperphagia resulting in morbid obesity, among other complex clinical manifestations. Of the many genes known to be associated with BBS, pathogenic variants in the BBS1 gene are most commonly encountered. Axovia is developing a genetic therapy for those harboring two mutations in the BBS1 gene. There is no cure for BBS, and its prevalence ranges between one in 70,000 and one in 100,000 in Europe and North America, with up to 10 times this prevalence in the Middle East. “Our $1 million grant to Axovia is a significant step for the BBS community, and we are optimistic about Axovia’s groundbreaking clinical research. However, the path to treating RP/BBS is challenging and there is still much work to be done. A Race Against Blindness is committed to supporting the effort to raise the necessary funds to ensure the execution of the AXV-101 clinical trial and potentially bring a new therapeutic option to BBS patients like our son, Luke,” said Dr. Stephen Johnston, Co-Founder and President. “We are advancing the AXV-101 program and preparing to initiate the first clinical study in mid-2025. We are immensely grateful for this funding from A Race Against Blindness,” said Professor Phil Beales, Chief Executive Officer and Co-Founder at Axovia Therapeutics. “By utilizing gene therapy to address the loss of sight and obesity experienced by patients with BBS1, we believe our approach will have a profound and dramatic impact on the health and well-being of this community. We look forward to generating clinical data for this program.” With the goal of initiating clinical studies in mid-2025, A Race Against Blindness will continue aggressive efforts to fundraise, engage the scientific community, and raise global awareness of BBS. Media Contact:Stephen Johnston, M.D., Co-Founder and PresidentSteve@ARaceAgainstBlindness.org (520) 548 - 1807

Progress on OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and new data from Phase 1/2Preliminary safety and efficacy update on OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA)Clinical update on OCU410ST Phase 1/2 GARDian clinical trial for Stargardt diseaseFeaturing patients, investigators and thought leaders MALVERN, Pa., Oct. 28, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that it will host an in-person Clinical Showcase on Tuesday, November 12, 2024. The event will take place from 10 a.m.-noon ET at the Nasdaq MarketSite in Times Square, New York City. The event will focus on encouraging updates from Ocugen’s ongoing gene therapy trials, including: Clinical update on Phase 3 liMeliGhT clinical trial for retinitis pigmentosa along with data updates from Phase 1/2 RP and Leber congenital amaurosis (LCA)Preliminary safety and efficacy data from Phase 1/2 OCU410 ArMaDa clinical trial for GAClinical trial progress from Phase 1/2 OCU410ST GARDian study for Stargardt disease In addition, background on Ocugen’s biologic candidate, OCU200 for diabetic macular edema, will be presented. The Company is planning to initiate the OCU200 Phase I clinical trial this quarter. Ocugen presenters will include: Dr. Shankar Musunuri, Chairman, CEO & Co-founderDr. Huma Qamar, Chief Medical OfficerDr. Arun Upadhyay, Chief Scientific Officer & Head of R&DMike Shine, Senior Vice President, Commercial Joining the Ocugen team will be study investigators: Dr. Benjamin Bakall, Director of Clinical Research at Associated Retina Consultants (ARC) and Clinical Assistant Professor at University of Arizona, College of Medicine – PhoenixDr. Lejla Vajzovic, Professor of Ophthalmology, Pediatrics, and Biomedical Engineering with Tenure at Duke Eye Center and Duke University School of MedicineDr. Syed M. Shah, Vice Chair for Research and Digital Health, Director of Retina Service at Gundersen Health – La Crosse, Wisconsin The program will conclude with a patient panel representing participants in Ocugen’s ongoing clinical trials. Advance registration is required and can be done by contacting Tiffany Hamilton at Tiffany.Hamilton@ocugen.com. About Ocugen, Inc.Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on 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, 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; 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 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 HamiltonHead of CommunicationsTiffany.Hamilton@ocugen.com

MILAN, Oct. 28, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, is pleased to congratulate Professor Alberto Auricchio, the Company’s Founder and Chief Scientific Officer (CSO), on his appointment as President and Board member of the European Society of Gene and Cell Therapy (ESGCT). The ESGCT Board is composed of researchers and clinicians representing a broad range of European Countries. Members are elected by nomination and vote at the Annual General Meeting. Election to the Board of ESGCT is a great honor and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. As well as being CSO of AAVantgarde, Professor Auricchio is Scientific Director of TIGEM and Professor of Medical Genetics at University “Federico II” in Naples, Italy. He is co-author of over 140 peer-reviewed publications and inventor of several patents related to the use of viral vectors for gene therapy and has received the Outstanding New Investigator Award of the American Society of Gene Therapy and the International Prize for Scientific Research “Arrigo Recordati”. Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde said, “Congratulations to Alberto on this important and well-deserved recognition of his outstanding scientific contributions to the field of gene therapy. We are privileged and honoured to work with him as Founder and Chief Scientific Officer of AAVantgarde.” About AAVantgarde BioAAVantgarde Bio is a clinical stage, international biotechnology company that has developed two proprietary Adeno-Associated Viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms could be used to deliver large genes to ocular and non-ocular tissues. AAVantgarde is initially validating its platforms in two inherited retinal diseases with clear unmet need, with its lead program in Usher syndrome type 1B already in the clinic. For more information, please visit: www.aavantgarde.com Contact:Dr. Magda Blanco – Head of Corporate Development, AAVantgardeEmail: info@aavantgarde.com