Arcturus Therapeutics Holdings Inc. is a biotechnology company focused on RNA-based medicines, developing a pipeline of vaccines for infectious diseases alongside treatments for rare liver and respiratory conditions across the United States. Its notable development programs encompass LUNAR-OTC, designed for ornithine transcarbamylase (OTC) deficiency, and LUNAR-CF, targeting cystic fibrosis lung disease caused by specific CFTR gene mutations. The company also advances vaccine initiatives such as LUNAR-COV19 and LUNAR-FLU. Arcturus has cultivated a range of strategic collaborations, including partnerships with Vinbiocare Biotechnology Joint Stock Company for manufacturing COVID-19 vaccines; Janssen Pharmaceuticals, Inc., to create nucleic acid-based therapies for hepatitis B virus; Ultragenyx Pharmaceutical, Inc., for developing mRNA therapeutics aimed at rare disease targets; CureVac AG, to progress mRNA therapeutic and vaccine candidates across various indications; the Singapore Economic Development Board and Duke-NUS Medical School, specifically for the LUNAR-COV19 vaccine; and Millennium Pharmaceuticals, Inc., focused on discovering siRNA medicines for non-alcoholic steatohepatitis (NASH). Founded in 2013, Arcturus Therapeutics maintains its corporate headquarters in San Diego, California.
Arcturus Therapeutics Announces Interim OTC Deficiency Phase 2 Results and Introduces LUNAR 2.0™ Next-Generation mRNA Delivery Platform
Arcturus announced interim Phase 2 results showing ARCT-810 was generally safe and well tolerated in OTC deficiency, reducing and/or maintaining fasting ammonia within normal range and lowering glutamine, with some participants achieving normal glutamine on the 0.5 mg/kg dose.
The company introduced LUNAR 2.0, a next-generation mRNA delivery platform demonstrating greater than 30-fold higher protein expression than LUNAR 1.0 in non-human primate studies, and ARCT-2601 built on it will be dosed near year-end 2026 in the ongoing Phase 2 study following favorable FDA Type C meeting feedback.
Arcturus also agreed to acquire AI discovery company myNEO, expected to close in October, to improve mRNA design quality and target identification. New pipeline programs in phenylketonuria and gout were announced alongside the platform.