Core Insights - Generation Bio Co. presented new data on its cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, highlighting advancements in genetic medicines for rare and prevalent diseases [1][2] Group 1: ctLNP Platform - A single intravenous dose of ctLNP successfully delivered mRNA encoding a reporter protein to a majority of circulating T cells in non-human primates, with a dose of 1 mg/kg transducing most T cells and showing balanced distribution to CD4+ and CD8+ cells [2] - Untargeted lipid nanoparticles (LNPs) did not achieve T cell transduction, indicating the specificity of the ctLNP platform [2] - The biodistribution of ctLNP-mRNA to other organs such as the liver, spleen, and lung was minimal, confirming improved results over previous murine studies [2] Group 2: iqDNA Platform - The iqDNA platform is a partially single-stranded DNA molecule designed to evade recognition by primary DNA sensors in both mice and non-human primates [3] - Recent research has focused on enhancing protein expression through methods such as encoding the promoter region as double-stranded and stabilizing iqDNA against degradation by exonucleases [3] - The company is pursuing targeted research areas to prepare iqDNA for the development of novel genetic medicines [3] Group 3: Company Overview - Generation Bio is focused on innovating non-viral genetic medicines aimed at providing durable and redosable treatments for patients with rare and prevalent diseases [5] - The company is developing two complementary platforms: the highly selective ctLNP delivery system and the novel iqDNA cargo produced through a scalable capsid-free manufacturing process [5] - The mission of Generation Bio is to extend the reach of genetic medicine to a broader patient population globally [5]
Generation Bio Announces the Presentation of Preclinical Data on ctLNP and iqDNA at the ESGCT 31st Annual Congress