Arcturus Therapeutics is an American biotech company focused on the discovery, development and commercialization of therapeutics for rare diseases with focus on RNA. Arcturus has developed a novel, potent and safe RNA therapeutics platform called LUNAR®, a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, gene editing, DNA, antisense, and microRNA oligotherapeutics. The company’s pipeline includes RNA therapeutics towards rare diseases such as ornithine transcarbamylase deficiency, and respiratory diseases such as cystic fibrosis. Vaccine medicines include a vaccine candidate for COVID-19.
Company history
Founded in 2013 by Joseph Payne and Dr. Pad Chivukula, Arcturus Therapeutics is headquartered in San Diego, California, USA. The company’s core technology platform is a proprietary lipid nanoparticle-mediated delivery system able to deliver therapeutic RNA or DNA to target cells inside a patients body. The company has a portfolio of at least 10 RNA-based therapeutics in development. On June 27th 2019, the FDA granted Orphan Drug Designation for the Company’s lead product candidate ARCT-810 to treat an inherited metabolic disorder ornithine transcarbamylase deficiency. OTCD is the most common urea cycle disorder, which impacts an individual’s ability to remove toxic waste products from the body. The orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. Drug products approved by the FDA that have orphan drug status receive up to 7 years of marketing exclusivity. On April 13, 2020, it was announced that the company’s investigational new drug application for Phase 1b study in patients with OTCD was allowed to proceed by the U.S. FDA. Another additional Clinical Trial Application for a Phase 1 study in healthy volunteers was approved by the New Zealand Medicines and Medical Devices Safety Authority. On June 5, 2020 Arcturus Therapeutics announced it dosed its first healthy volunteer in a Phase 1 study with ARCT-810.
Platform
Arcturus Therapeutics' primary technology platform for RNA therapeutics is called LUNAR® a novel Lipid-enabled nanoparticle. LUNAR is a multi-component drug delivery system that enables scientists to target specific cells inside the body and deliver a payload of RNA into the cells cytosol. Once release of the RNA into the cytosol occurs, the normal translational machinery of the cell can interact with the RNA to make functional protein that delivers a therapeutic effect. ARCT-810, the company’s lead product utilizes Arcturus' LUNAR® lipid-mediated delivery platform intended to safely and effectively deliver OTC messenger RNA to liver cells. LUNAR® mediated delivery to the liver has been confirmed in collaboration with the Salk Institute for Biological Studies in La Jolla, California which demonstrated the successful LUNAR®-mediated delivery of a Factor IX messenger RNA to the liver as reported in a publication in PNAS, where it was confirmed be efficacious. LUNAR® is an effective and reproducible LNP mRNA delivery platform to potentially treat diseases requiring protein replacement. Researchers at the MD Anderson Cancer Center also showed the unique application of microRNAs delivered in nanoparticles using the LUNAR platform as novel immune therapeutics for the treatment of malignancy. STARRTM The STARRTM technology platform combines self-replicating mRNA with LUNAR® creating the ability to generate a protective immune response against infectious pathogens or increasing therapeutic protein expression to potentially prevent and treat a variety of diseases. Arcturus is developing a vaccine to protect against COVID-19 utilizing STARRTM technology.
Products in development
The company has at least 10 programs in development for novel RNA therapeutics, both wholly owned and in collaboration with other Pharmaceutical companies and Academic Labs.
LUNAR-OTC
The company’s lead development program: an mRNA medicine to treat ornithine transcarbamylase deficiency, a life-threatening genetic disease caused by lack of the OTC enzyme in liver cells. OTC can cause seizures, coma, and death in untreated patients. There is currently no cure for OTC deficiency. In 2019, LUNAR OTC received FDA Orphan Drug Status. Lunar OTC clinicals trials in humans began June 2020.
