This segment focuses on the research and development of mRNA-based therapeutics for cystic fibrosis (CF) lung disease. Arcturus Therapeutics utilizes its proprietary LUNAR delivery platform to develop mRNA constructs targeting the CFTR gene, aiming to restore functional CFTR protein expression in lung cells. Research and development activities include preclinical studies, clinical trials, and formulation optimization for inhaled delivery. The segment's technologies and methodologies center around mRNA design, lipid nanoparticle (LNP) formulation, and pulmonary delivery systems. The therapeutic area is cystic fibrosis, addressing the underlying genetic defect that causes the disease. The patient impact is significant, as successful therapies could improve lung function, reduce exacerbations, and extend the lifespan of CF patients. Market positioning involves competing with other CF therapies, including CFTR modulators and gene therapies. Future opportunities include expanding the LUNAR-CF program to address different CFTR mutations and exploring combination therapies. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials to demonstrate safety and efficacy. Partnerships and collaborations may include collaborations with patient advocacy groups and other pharmaceutical companies to accelerate development and commercialization.

This segment focuses on the development of mRNA vaccines for infectious diseases, including COVID-19, influenza, and potential pandemic threats like H5N1 flu. Arcturus Therapeutics leverages its LUNAR delivery platform to design and develop mRNA vaccines that elicit robust immune responses. Research and development activities include vaccine design, preclinical studies, clinical trials, and manufacturing process development. The technologies and methodologies employed involve mRNA synthesis, LNP formulation, and various delivery methods. The therapeutic areas covered are infectious diseases, with a focus on preventing and controlling outbreaks. The patient impact is substantial, as effective vaccines can protect individuals and communities from severe illness and death. Market positioning involves competing with established vaccine manufacturers and other mRNA vaccine developers. Future opportunities include expanding the vaccine portfolio to address emerging infectious diseases and developing next-generation vaccine technologies. Regulatory and clinical aspects involve obtaining regulatory approvals from health authorities and conducting clinical trials to demonstrate safety and efficacy. Partnerships and collaborations are crucial for manufacturing, clinical trials, and commercialization, as evidenced by collaborations with Vinbiocare Biotechnology Joint Stock Company and CSL.

This segment is dedicated to the development of mRNA-based therapeutics for rare diseases, with a current focus on ornithine transcarbamylase (OTC) deficiency. Arcturus Therapeutics utilizes its LUNAR delivery platform to develop mRNA therapies that address the underlying genetic causes of these rare conditions. Research and development activities include preclinical studies, clinical trials, and formulation optimization. The technologies and methodologies employed involve mRNA design, LNP formulation, and targeted delivery systems. The therapeutic area is rare diseases, specifically targeting genetic disorders with high unmet medical needs. The patient impact is significant, as successful therapies can improve the quality of life and potentially extend the lifespan of patients with rare diseases. Market positioning involves competing with other rare disease therapies, including gene therapies and small molecule drugs. Future opportunities include expanding the rare disease pipeline to address other genetic disorders and exploring combination therapies. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials to demonstrate safety and efficacy. Partnerships and collaborations may include collaborations with patient advocacy groups and other pharmaceutical companies to accelerate development and commercialization.