Arcturus Therapeutics Holdings Inc.

We are a messenger RNA medicines company focused on developing therapeutics for liver and respiratory rare diseases. We are conducting Phase 2 clinical studies for RNA therapeutic candidates targeting ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF). We developed KOSTAIVE®, the world's first approved self-amplifying messenger RNA (sa-mRNA) vaccine, in partnership with Seqirus, Inc. (CSL Seqirus), a part of CSL Limited. KOSTAIVE® is approved in Japan, the European Union, and the United Kingdom as a COVID-19 vaccine, with sales commencing in Japan in October 2024. We utilize key platform technologies to advance a pipeline of mRNA-based therapeutics for rare genetic disorders and vaccines for infectious diseases. Our mRNA medicines consist of messenger RNA (mRNA) constructs and lipid nanoparticles (LNP) for delivery. We specialize in designing and optimizing mRNA constructs, including self-amplifying mRNA (sa-mRNA). Our proprietary self-amplifying mRNA technology platform, STARR®, has shown to induce a robust, longer-lasting, and broader humoral immune response at lower doses compared to conventional mRNA-based vaccines. Our proprietary LNP delivery system, LUNAR®, aims to effectively and safely deliver RNA to target tissues, potentially enabling multiple nucleic acid medicines. We also possess expertise in developing and scaling manufacturing processes for nucleic acid medicines. Our internal pipeline includes RNA therapeutic candidates for ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF). In our vaccine program, we partner with CSL Seqirus on the development and commercialization of mRNA vaccines for COVID-19, influenza, and three other infectious diseases.