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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) announces participation of CEO Geoff McDonough, M.D., in a panel discussion at the Wedbush PacGrow Healthcare Conference 2021 on August 10 at 9:10 a.m. ET. The event will focus on the company's innovations in genetic medicines for rare and prevalent diseases. A live webcast will be available on the company's investor website, with a replay accessible for 30 days post-event. Generation Bio's unique platform, utilizing closed-ended DNA and advanced lipid nanoparticle delivery, aims to provide durable treatments and scalable manufacturing for genetic therapies.
Generation Bio (Nasdaq: GBIO) announced a transition to rapid enzymatic synthesis (RES) for its closed-ended DNA manufacturing across all programs, enhancing quality, scale, and speed. A lease agreement was signed for a cGMP manufacturing facility in Waltham, MA, expected to be operational in 2023. The company anticipates submitting an IND application for hemophilia A by year-end 2023, with factor VIII expression data anticipated in late 2021. Despite incurring $104 million in lease costs and $45 million in facility investments, the company expects its cash runway to remain sufficient until 2024.
Generation Bio, a leader in genetic medicines, announced that CEO Geoff McDonough will present at the William Blair Biotech Focus Conference on July 15, 2021, at 9:00 a.m. ET. The event will feature a live webcast available on their investor website, with a replay accessible for 30 days. Generation Bio focuses on developing non-viral gene therapies aimed at providing durable treatments for various diseases. Their innovative platform utilizes closed-ended DNA and a specialized delivery system to target specific tissues for effective therapy.
Generation Bio Co. (Nasdaq: GBIO) announced that CEO Geoff McDonough will present at the JMP Securities Life Sciences Conference on June 16 at 11:30 a.m. ET. A live webcast of the presentation will be available on the company's investor website, with a replay available for 30 days. The company specializes in non-viral gene therapies aimed at providing durable treatments for various diseases, utilizing a unique platform that combines high-capacity DNA constructs and targeted delivery systems. Generation Bio is committed to broadening access to gene therapy worldwide.
Generation Bio Co. (Nasdaq: GBIO) has announced that its CEO, Geoff McDonough, will be presenting at the Jefferies Virtual Healthcare Conference on June 3, 2021, at 11:30 a.m. ET. The presentation will be accessible via a live webcast on the company’s investor website, with a replay available for 30 days after.
Generation Bio specializes in non-viral gene therapies aimed at providing durable treatments for various diseases. Their innovative platform employs closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle system to enhance gene delivery and efficacy.
Generation Bio Co. (Nasdaq: GBIO) reported significant progress in its non-viral gene therapy platform during the 24th ASGCT Annual Meeting, showcasing the production of anti-SARS-CoV-2 antibodies in mice and advancements in manufacturing efficiency. The first quarter results revealed a cash position of $451.1 million and R&D expenses of $18.8 million. However, the company recorded a net loss of $25.6 million, widening from the previous year. Generation Bio aims to leverage these developments to address a range of diseases and drive future growth.
Generation Bio Co (Nasdaq: GBIO) has announced three digital presentations for the American Society of Gene & Cell Therapy (ASGCT) 2021 Virtual Annual Meeting from May 11-14. These presentations will showcase data on their innovative non-viral gene therapy platform, focusing on antibody gene therapy, hemophilia A programs, and ceDNA construct engineering. Chief Scientific Officer Matthew Stanton noted that the hemophilia A program is progressing well, with additional data expected by mid-year. Registered attendees can access the presentations beginning May 11.
Generation Bio Co. (Nasdaq: GBIO), a genetic medicines company, announced that its CEO, Geoff McDonough, will join a fireside chat at the 20th Annual Needham Virtual Healthcare Conference on April 15, 2021, at 3:00 p.m. ET. A live webcast will be accessible via the company's investor website, with a replay available for 30 days post-event. Generation Bio focuses on non-viral gene therapies, utilizing innovative technology such as closed-ended DNA (ceDNA) and cell-targeted lipid nanoparticles (ctLNP) to provide solutions for rare diseases.
Generation Bio Co. (Nasdaq: GBIO) reported strong financials for 2020, ending the year with a cash balance of $262.3 million, bolstered by $211.3 million raised in early 2021. Notably, the company has a successful follow-on offering, raising $225.4 million in January 2021. The company also announced promising preclinical data for its hemophilia A program, showing potential for significant factor VIII expression. Despite a net loss of $80.5 million in 2020, the company remains focused on advancing its pipeline through planned milestones in 2021.
Generation Bio (Nasdaq: GBIO) announced the appointment of Ron Cooper to its Board of Directors, effective March 1, 2021. Cooper, a seasoned biopharmaceutical executive, has led Albireo Pharma since June 2015 and has a strong background with Bristol Myers Squibb. His experience in commercializing products and working in rare diseases is expected to bolster Generation Bio during its critical growth phase. The company focuses on innovative non-viral gene therapies for rare diseases, aiming to enhance treatment durability and scalability.
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