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Generation Bio Co. (Nasdaq: GBIO) is a pioneering biotechnology company dedicated to developing innovative genetic medicines to treat rare and prevalent diseases. The company's mission is to deliver durable and redosable treatments through their groundbreaking platforms. Generation Bio focuses on liver and retina-related diseases and has built a robust pipeline leveraging two main proprietary technologies: the cell-targeted lipid nanoparticle (ctLNP) delivery system and immune-quiet DNA (iqDNA) cargo.
Using the ctLNP delivery system, Generation Bio can selectively target specific tissues and cell types, significantly reducing off-target effects. This system has shown promising results in both mouse models and non-human primates (NHPs), demonstrating effective delivery of genetic materials with minimal immunogenic response. The ctLNP platform is a cornerstone of their collaborative efforts with Moderna, aimed at developing highly specific in vivo delivery systems for T cells and other immune cells.
iqDNA, an optimized variant of the company's closed-ended DNA (ceDNA), stands out for its ability to evade the immune system while maintaining high levels of gene expression. This feature addresses a critical challenge in genetic therapies by allowing for repeated dosing without triggering adverse immune responses. Recent studies have shown that iqDNA has a tolerability profile comparable to chemically modified messenger RNA (mRNA) in both mice and NHPs.
Generation Bio's commitment to innovation is further highlighted by their rapid enzymatic synthesis (RES) manufacturing process. This proprietary method enables scalable and efficient production of their novel DNA constructs, ensuring high purity and consistency. The company believes that RES can support the manufacturing scale required to meet global clinical and commercial demands.
Recent announcements have emphasized the company's progress and strategic collaborations. For instance, their partnership with Moderna focuses on leveraging ctLNPs for targeted delivery of nucleic acid therapies to specific immune cell types. Additionally, Generation Bio's sustained financial health, supported by careful expense management and collaboration incomes, ensures a steady runway into 2026, enabling continued investment in their groundbreaking technologies.
On the business front, Generation Bio is ramping up efforts across their platforms, with ongoing programs targeting hemophilia A, sickle cell disease, and beta-thalassemia. The company is actively presenting their latest data at key industry conferences like the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, showcasing the advanced capabilities of their ctLNP and iqDNA platforms.
For more information about Generation Bio’s revolutionary work in genetic medicine, please visit their website at www.generationbio.com or follow their updates on Twitter (@lifetimegenetx).
Generation Bio Co. (Nasdaq: GBIO) closed its public offering of 9,200,000 shares at $24.50 each, generating approximately $225.4 million gross proceeds. This includes 1,200,000 shares from underwriters' option to purchase more. Joint bookrunning managers for the offering were J.P. Morgan, Jefferies, and Cowen. The funds raised will support the company's innovative gene therapy efforts aimed at treating both rare and prevalent diseases through its proprietary non-viral delivery platform.
Generation Bio Co. (Nasdaq: GBIO) has priced a public offering of 8,000,000 shares of its common stock at $24.50 per share. With a 30-day option for underwriters to purchase an additional 1,200,000 shares, the gross proceeds are estimated at approximately $196.0 million, pending closing on January 11, 2021. J.P. Morgan, Jefferies, and Cowen are leading the offering, with a focus on advancing their innovative gene therapy technology aimed at treating rare and prevalent diseases.
Generation Bio Co. (Nasdaq: GBIO) announced it will present at the 39th Annual J.P. Morgan Healthcare Conference on January 12, 2021. CEO Geoff McDonough will share insights on the company’s progress and upcoming milestones. Recently, Generation Bio achieved a key scientific milestone by demonstrating human factor VIII expression in hemophilia A mice, reaching 23% of normal levels. They also confirmed the successful translation of their liver-targeted delivery system into non-human primates, supporting further studies for hemophilia A treatments.
Generation Bio (NASDAQ: GBIO) has achieved significant progress in its non-viral gene therapy platform, particularly for hemophilia A treatments. Their recent study demonstrated a mean human factor VIII expression of 23% of normal in hemophilia A mice after a single dose of closed-ended DNA (ceDNA) delivered via cell-targeted lipid nanoparticles (ctLNPs). Notably, the therapy was well tolerated across all tested doses in both mice and non-human primates. Generation Bio is on track to select a final candidate for clinical development and begin IND-enabling studies this year.
Generation Bio Co. (Nasdaq: GBIO) announced progress in its non-viral gene therapy programs and third-quarter financial results. The company is on track to advance lead programs into IND-enabling preclinical development in 2021, backed by a strong cash position of $280.4 million as of September 30, 2020. R&D expenses rose to $15.3 million, while G&A expenses increased to $5.7 million, resulting in a net loss of $20.8 million. Key leadership appointments were also announced, including Tracy Zimmermann as Chief Development Officer and Doug Kerr as Chief Medical Officer, to enhance R&D capabilities.
Generation Bio Co. (Nasdaq: GBIO) announced that Phillip Samayoa will present a company overview at the Cell & Gene Meeting on the Mesa, virtually held from Oct. 12-16, 2020. The presentation will be available for on-demand viewing during the conference. Additionally, CEO Geoff McDonough, M.D., will participate in a panel discussion on gene delivery, accessible from Oct. 13. Generation Bio focuses on innovative, non-viral gene therapy for durable treatments of rare and prevalent diseases using proprietary technologies aimed at scalable and effective gene delivery.
Generation Bio Co. (Nasdaq: GBIO), a genetic medicines company, announced that CEO Geoff McDonough will present at the Jefferies 2020 Virtual Gene Therapy/Editing Summit on October 1 at 11:00 a.m. ET. A live webcast of the presentation will be available on the company's investor website and can be replayed for 90 days afterward.
The company's innovative platform focuses on non-viral gene therapy, utilizing proprietary closed-ended DNA and targeted delivery systems to enable durable treatments for various diseases.
Generation Bio, listed as GBIO, reported significant advances in its genetic medicine pipeline and second-quarter financial results. The company successfully completed a $230 million IPO on June 16, 2020, enhancing its robust cash position of $301.1 million as of June 30, 2020. R&D expenses rose to $13.5 million, while G&A expenses increased to $4.3 million. The net loss for the quarter was $17.7 million, up from $15.0 million in 2019. The leadership team was bolstered by the appointment of Dr. Matthew Norkunas as CFO.
Generation Bio (Nasdaq: GBIO), a leader in non-viral gene therapy, will present at the 2020 Wedbush PacGrow Healthcare Virtual Conference on August 12, 2020, at 10:20 a.m. ET. The presentation will highlight their innovative platforms, featuring closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP), aimed at providing long-lasting therapies for both rare and prevalent diseases. A live webcast will be accessible on their investor website, with an archive available for 60 days post-event.
Generation Bio Co (Nasdaq: GBIO) announced the appointment of Matthew Norkunas, M.D., MBA, as chief financial officer. Norkunas brings a wealth of experience, having previously served as CFO at SomaLogic and as a senior equity analyst at Marsico Capital Management. His diverse background in healthcare and finance is expected to bolster Generation Bio's strategic initiatives as it seeks to advance its non-viral gene therapy platform. This innovative approach aims to provide sustainable treatments for both rare and prevalent diseases, enhancing the company’s mission to broaden the accessibility of gene therapy.
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