This segment focuses on the research and development of genetic medicines for liver diseases. Generation Bio utilizes its proprietary ctLNP and iqDNA platforms to deliver gene therapies to the liver, aiming to provide durable and redosable treatments. Research and development activities include preclinical studies, clinical trials, and the optimization of lipid nanoparticles for targeted delivery. The company is working on therapies for both rare and prevalent liver diseases, with the goal of improving patient outcomes and quality of life. Market positioning involves competing with other gene therapy companies and traditional pharmaceutical approaches. Future opportunities include expanding the pipeline to address additional liver disorders and securing regulatory approvals for its lead product candidates. Partnerships with other companies, such as Moderna, are also key to this segment.

This segment is dedicated to developing genetic medicines for retinal diseases. Generation Bio's research and development efforts involve the application of its ctLNP and iqDNA platforms to deliver therapeutic genes to the retina. The company is focused on creating durable and redosable treatments for various retinal disorders, with the aim of preserving or restoring vision. Research activities include preclinical studies, clinical trials, and the optimization of delivery systems to ensure efficient and targeted gene expression in the eye. The company's approach involves addressing both rare and prevalent retinal diseases, with the goal of improving patient outcomes and quality of life. Market positioning involves competing with other gene therapy companies and established treatments. Future opportunities include expanding the pipeline to address additional retinal disorders and securing regulatory approvals for its lead product candidates. Partnerships and collaborations are also important for this segment.

This segment focuses on the development of siRNA therapeutics for T cell-driven autoimmune diseases. Generation Bio is applying its T cell-selective lipid nanoparticle (ctLNP) technology to develop these therapies. Research and development activities include preclinical studies, clinical trials, and the optimization of ctLNP for targeted delivery to T cells. The company aims to address autoimmune diseases by modulating the activity of T cells, with the goal of improving patient outcomes and quality of life. Market positioning involves competing with other companies developing treatments for autoimmune diseases. Future opportunities include expanding the pipeline to address additional autoimmune disorders and securing regulatory approvals for its lead product candidates. Partnerships and collaborations are also key to this segment.