This segment focuses on the research and development of RNAi therapeutics for various liver diseases. Dicerna's GalXC platform is central to this segment, enabling the development of drugs targeting specific genes in liver cells. Research and development activities include preclinical studies, clinical trials, and regulatory submissions. The primary therapeutic areas include rare liver diseases, chronic liver diseases, and viral infections. The goal is to improve patient outcomes by providing targeted therapies with fewer side effects. Market positioning involves strategic collaborations with pharmaceutical partners like Novo Nordisk and Roche. Future opportunities include expanding the pipeline to address additional liver diseases and advancing clinical trials to later stages. Regulatory aspects involve obtaining FDA approval for new drug candidates. Partnerships with other companies are crucial for drug development and commercialization.

Dicerna's Rare Diseases segment concentrates on developing RNAi-based therapies for rare genetic disorders. The company utilizes its GalXC platform to design drugs that silence genes associated with these conditions. Research and development efforts include identifying therapeutic targets, conducting preclinical studies, and initiating clinical trials. The segment's focus is on diseases with high unmet medical needs, such as primary hyperoxaluria. The patient impact is significant, as these therapies aim to improve the quality of life and potentially extend the lifespan of patients. Market positioning involves collaborations with other pharmaceutical companies and seeking regulatory approvals. Future opportunities include expanding the pipeline to address additional rare diseases and advancing clinical trials. Regulatory aspects involve navigating the FDA approval process for orphan drugs. Partnerships are essential for funding research, conducting clinical trials, and commercializing approved therapies.

This segment is dedicated to the development of RNAi therapeutics for cardiometabolic diseases. Dicerna's GalXC platform is used to target genes involved in these complex conditions. Research and development activities include identifying therapeutic targets, conducting preclinical studies, and initiating clinical trials. The segment focuses on diseases such as those related to lipid metabolism and cardiovascular health. The goal is to improve patient outcomes by providing targeted therapies with fewer side effects. Market positioning involves strategic collaborations with pharmaceutical partners. Future opportunities include expanding the pipeline to address additional cardiometabolic diseases and advancing clinical trials to later stages. Regulatory aspects involve obtaining FDA approval for new drug candidates. Partnerships with other companies are crucial for drug development and commercialization.