Generation Bio

Generation Bio is at the forefront of genetic medicine, focusing on creating durable and redosable treatments for individuals with rare and prevalent diseases. The company operates in the biopharmaceutical sector, leveraging its proprietary non-viral genetic medicine platform. This platform features iqDNA, an immune-quiet DNA, and ctLNP, a cell-targeted lipid nanoparticle delivery system. Additionally, Generation Bio employs a scalable, capsid-free manufacturing process known as rapid enzymatic synthesis (RES) to produce iqDNA. This method aims to provide multi-year durability from a single dose, deliver large genetic payloads to specific tissues and cell types, and allow for titration and redosing to adjust expression levels in patients. The RES process has the potential to scale production to hundreds of millions of doses, supporting the company's mission to make genetic medicine accessible to a broader population. Generation Bio's business model revolves around developing and commercializing these genetic treatments, targeting both rare and common diseases, thereby addressing a significant unmet medical need.

Keywords: genetic medicine, iqDNA, ctLNP, RES, biopharmaceutical, rare diseases, prevalent diseases, scalable manufacturing, cell-targeted delivery, redosable treatments.