Latest news with #Vitalgen
Associated Press
4 days ago
- Business
- Associated Press
Vitalgen Completes Phase III Enrollment for VGR-R01 Gene Therapy Targeting Bietti Crystalline Dystrophy
Company on track for BLA submission in H2 2026 for potential first-in-class treatment 'Completing enrollment for our Phase III trial represents a pivotal milestone in bringing the first potential treatment to BCD patients who currently have no therapeutic options'— Dr. David Wu, Business Development Director SHANGHAI, SHANGHAI, CHINA, May 27, 2025 / / -- Vitalgen BioPharma Co., Ltd., a clinical-stage gene therapy company, today announced the completion of patient enrollment in March 2025 for its Phase III pivotal trial (VGR-R01-301) evaluating VGR-R01, an investigational AAV-based gene therapy for Bietti Crystalline Dystrophy (BCD). The multicenter trial, conducted across five leading clinical institutions in China, will evaluate the efficacy and safety of VGR-R01 in BCD patients. The therapy has received Breakthrough Therapy Designation from China's Center for Drug Evaluation (CDE) and Orphan Drug Designation from the U.S. FDA. With these regulatory designations, VGR-R01 is positioned to potentially become both a first-in-class and best-in-class treatment, with a Biologics License Application (BLA) submission targeted for the second half of 2026. 'Completing enrollment for our Phase III trial represents a pivotal milestone in bringing the first potential treatment to BCD patients who currently have no therapeutic options,' said Dr. David Wu, Business Development Director at Vitalgen. 'The strong safety profile and efficacy demonstrated in our Phase I/II trials, combined with our regulatory designations, underscore VGR-R01's potential to transform the treatment landscape for this devastating disease.' Positive Phase I/II Results Support Advancement Phase I/II clinical data demonstrated that VGR-R01 improved best-corrected visual acuity (BCVA) and functional vision in low-luminance conditions. The therapy showed benefits in patients with disease duration exceeding 10 years and reported no VGR-R01-related serious adverse events. VGR-R01 delivers a functional copy of the CYP4V2 gene to retinal pigment epithelial cells, addressing the underlying metabolic dysfunction that causes progressive vision loss in BCD patients. By restoring lipid metabolism in the retina, the therapy aims to prevent structural and functional deterioration, offering hope for halting or slowing disease progression. About Bietti Crystalline Dystrophy BCD is a rare inherited retinal disorder primarily affecting East Asian populations, particularly in China, Japan, and Korea, with an estimated 80,000+ patients globally. The disease causes progressive vision loss, night blindness, and visual field constriction. No approved treatments currently exist, representing a significant unmet medical need. About Vitalgen Vitalgen BioPharma Co., Ltd. is a clinical-stage biotechnology company developing gene therapies for rare and common diseases. Founded in 2020 and headquartered in Shanghai, the company operates GMP-compliant facilities meeting both Chinese and U.S. regulatory standards. For more information, visit Contact: Business Development Email: [email protected] Forward-Looking Statements This press release contains forward-looking statements regarding VGR-R01's development, including expectations for regulatory submissions and the therapy's potential benefits. These statements are based on current expectations and are subject to risks and uncertainties that could cause actual results to differ materially. Factors include, but are not limited to, clinical trial outcomes, regulatory review processes, and manufacturing capabilities. Vitalgen undertakes no obligation to update these forward-looking statements except as required by law. David Wu Vitalgen BioPharma email us here Legal Disclaimer: EIN Presswire provides this news content 'as is' without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.
Associated Press
15-04-2025
- Business
- Associated Press
Vitalgen's Cutting-Edge Gene Editing and Delivery Platforms to be Featured at AACR Annual Meeting 2025
Collaborative research with Grit Biotechnology showcases Vitalgen's proprietary platform and delivery systems in oncology applications 'These AACR presentations showcase how our proprietary platforms can enable breakthrough therapies across multiple therapeutic areas through strategic collaborations'— Dr. Xiaoping Zhao, Ph.D., CEO CHICAGO, IL, UNITED STATES, April 15, 2025 / / -- Vitalgen BioPharma Co., Ltd. ('Vitalgen'), a pioneer in transformative gene therapy technologies, today announced its collaborative research with Grit Biotechnology Co., Ltd. ('Grit Bio') will be featured in two presentations at the American Association for Cancer Research (AACR) Annual Meeting 2025 in Chicago from April 25th to 30th. The presentations highlight the versatility and efficacy of Vitalgen's proprietary technology platforms in oncology applications, demonstrating the company's ability to form productive partnerships across therapeutic areas. 'These AACR presentations showcase how our proprietary platforms can enable breakthrough therapies across multiple therapeutic areas through strategic collaborations,' said Dr. Xiaoping Zhao, Ph.D., CEO and Co-founder of Vitalgen. 'While we maintain our internal focus, our technology platforms—particularly our ViCas® CRISPR gene-editing platform and ViLNP® delivery systems—have proven valuable in advancing development of novel cancer therapies through partnerships.' The collaborative research being presented includes advancements in gene-edited tumor-infiltrating lymphocyte (TIL) therapy and next-generation neoantigen cancer vaccines. Specifically, Grit Bio will present data on their development a next-generation dual-knockout TIL product, GT300, which utilizes Vitalgen's proprietary AaCas12bMax gene-editing technology, showing superior editing safety profile and enhanced expansion and cell fitness compared to their SpCas9-edited counterparts. Moreover, a novel neoantigen cancer vaccine GT600 approach combining Grit Bio's NEOvigator™ neoantigen identification platform with Vitalgen's APC-targeted LNP delivery system Dakini™ will be presented. Both GT300 and GT600 are under clinical assessment. 'The AaCas12bMax system represents a significant leap forward in gene-editing technology, offering potent on-target editing efficiency with a proven superior safety profile compared to the conventional editors,' said Dr. Xi Zhu, Ph.D., Head of Translational Research at Vitalgen. 'Precise editing fidelity combined with good cellular fitness and expansion capacity post editing are critical determinants for the development of engineered cell therapies. Our collaboration with Grit Bio validates the translational potential of AaCas12bMax in advancing the development of next-generation gene-editing products with enhanced safety profile and therapeutic potential.' Vitalgen's technology platforms featured in the presentations include: 1. ViCas® CRISPR DNA Editing Platform: -Featuring the AaCas12bMax system, which offers efficiency gene editing with negligible off-target gene editing -Enhanced cellular fitness and expansion capacity post AaCas12bMax editing during manufacture 2. ViLNP® Dakini: An Antigen Presenting Cell (APC)-specific delivery LNP with efficiency of antigen-specific T cell induction exceeding comparable technologies by more than 10-fold without detectable hepatocyte delivery following systemic administration. Since its founding in March 2020, Vitalgen has raised $160 million through Series B+ funding and developed a robust pipeline with two programs in Phase III, two in Phase I/II, and four in IIT studies. The company has built comprehensive infrastructure including 2,500 m² of R&D facilities and 8,500 m² of GMP-compliant manufacturing space in Shanghai. About Vitalgen Vitalgen BioPharma Co., Ltd. is a patient-focused, innovation-driven biotechnology company developing transformative gene therapies. With vertically integrated proprietary platforms, Vitalgen focuses on rare and common diseases while enabling partners to leverage its technologies across multiple therapeutic areas. Founded in 2020 and headquartered in Shanghai, China, Vitalgen operates state-of-the-art facilities compliant with both China GMP and US cGMP standards. For more information, visit About Grit Bio GRIT Bio was founded in 2019 as an innovative biopharmaceutical company, focused on immune cell treatments for oncology and characterized by a R&D pipeline in TIL therapies. GRIT Bio has completed multiple rounds of equity financing and is backed by renowned venture capital funds. GT101, a proprietary injectable developed by GRIT Bio, is the first TIL therapy that entered registrational clinical trial. GT 101 is currently in pivotal Phase II clinical study. GRIT Bio's GT201 injectable, the first TIL therapy with membrane-bound IL-15 complex, has cleared IND in both US and China. Core R&D platforms of the company include StemTexp® stemness TIL expansion platform, StaViral® stably virus transfected cell lines, ImmuT Finder® immune modulator target discovery platform, and KOReTIL® high-efficiency gene knock-out system. Based on the platforms, GRIT Bio generated a series of next-generation gene-edited TIL therapies. With internationally advanced technology reserve and industry resources, GRIT Bio aims to develop breakthrough therapies for solid tumors and bring new hope to cancer patients. For further information, please visit: Forward-Looking Statements This press release contains forward-looking statements about Vitalgen BioPharma Co., Ltd. based on management's current expectations, which are subject to known and unknown uncertainties and risks. Words such as 'anticipated,' 'expect,' 'intend,' 'plan,' 'believe,' 'seek,' 'estimate,' 'may,' 'will,' and variations of these words or similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding our expectations for our technology platforms, gene therapy candidates in development, collaborations, and plans for commercialization. Our actual results could differ materially from those discussed due to a number of factors, including, but not limited to, the risk inherent in developing pharmaceutical product candidates, conducting successful clinical trials, and obtaining regulatory approvals. Except as required by law, Vitalgen assumes no obligation to update any forward-looking statements contained herein as a result of new information, future events, or otherwise. David Wu Vitalgen BioPharma email us here Legal Disclaimer: EIN Presswire provides this news content 'as is' without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. 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