Arcturus Therapeutics

Arcturus Therapeutics is an American RNA medicines biotechnology company focused on the discovery, development and commercialization of therapeutics for rare diseases and infectious diseases. Arcturus has developed a novel, potent, and safe RNA therapeutics platform called LUNAR, a proprietary lipid-enabled delivery system for nucleic acid medicines including small interfering RNA (siRNA), messenger RNA (mRNA), gene editing RNA, DNA, antisense oligonucleotides (ASO), and microRNA. 
The company's pipeline includes RNA therapeutics for the treatment of rare diseases such as ornithine transcarbamylase deficiency (OTCD), and respiratory diseases such as cystic fibrosis. Vaccine medicines include a vaccine candidate for COVID-19, currently co-developed with Singaporean scientists at Duke–NUS Medical School and an influenza vaccine in the preclinical development phase.
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 patient's body. The company has a portfolio of eight mRNA-based therapeutics and two mRNA-based vaccines 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). 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 (IND) application for Phase 1b study in patients with OTCD was allowed to proceed by the U.S. FDA. A Clinical Trial Application (CTA) for a Phase 1 study in healthy volunteers was approved by the New Zealand Medicines and Medical Devices Safety Authority (Medsafe). On June 5, 2020 Arcturus Therapeutics announced it dosed its first healthy volunteer in a Phase 1 study with ARCT-810. Following the success of Phase 1a, clinical phase progression continued in December 2020 with the dosing of the first patients in phase 1b of the Clinical Trial.
Platform.
LUNAR 
Arcturus Therapeutics' primary technology platform for RNA therapeutics is called LUNAR a novel lipid-mediated delivery system. 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 cell. Once release of the RNA into the cell occurs, the normal translational machinery of the cell can interact with the RNA to make a functional protein with 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 (FIX mRNA) 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.
STARR
STARR technology is a combination of self-replicating mRNA with the LUNAR delivery platform which increases the level and duration of expression of a therapeutic protein. The STARR technology platform is able to generate a protective immune response against infectious pathogens or increase the expression of therapeutic proteins to prevent and treat a variety of diseases. Arcturus is using the STARR technology to develop vaccines for both COVID-19 and, separately, influenza.
Products in development.
The company has programs in development for novel RNA therapeutics and mRNA vaccines, both wholly owned and in collaboration with other Pharmaceutical companies and Academic Labs.
LUNAR-COV19.
In response to the COVID-19 pandemic, Arcturus Therapeutics partnered with Singapore's Duke–NUS Medical School to develop a COVID-19 vaccine using STARR technology (STARR technology is a combination of self-replicating mRNA with the LUNAR delivery platform which increases the level and duration of expression of a therapeutic protein). The company also partnered with Catalent and Recipharm, contract development and manufacturing organizations, to manufacture multiple batches of Arcturus' COVID-19 mRNA vaccine candidate. LUNAR-COV19 clinical trials in healthy volunteers began in July 2020. Phase 2 clinical trials were approved in the United States and Singapore by the FDA and HSA, respectively in December 2020. 
Arcturus has entered into development and supply agreements with the Economic Development Board of Singapore and supply agreements with the Israel Ministry of Health for LUNAR-COV19.
LUNAR-OTC.
A wholly owned 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 clinical trials in patient began June 2020. In December 2020, clinical phase 1b of LUNAR-OTC began with dosing in patients.
LUNAR-CF
LUNAR-CF is a project in collaboration with the Cystic Fibrosis Foundation to treat cystic fibrosis.
LUNAR-FLU.
A wholly owned program to protect against the influenza virus. Arcturus is combining its self-replicating mRNA STARR technology with LUNAR to develop a prophylactic vaccine against influenza.
LUNAR-GSD.
LUNAR-GSD is a project in collaboration with Ultragenyx to treat glycogen storage disease type III. 
LUNAR-Rare.
LUNAR-Rare is a project in collaboration with Ultragenyx to develop therapeutic candidates for certain rare disease targets. 
LUNAR-HBV.
LUNAR-HBV is a project in collaboration with Janssen to develop medicines for the treatment of hepatitis B virus infection.
LUNAR-NASH.
LUNAR-NASH is a project in collaboration with Takeda to develop medicines for the treatment of non-alcoholic steatohepatitis (NASH) and other gastrointestinal disorders. 
LUNAR-RPL.
LUNAR-RPL is a project in collaboration with Synthetic Genomics to develop improved technology for vaccines and therapeutics.
LUNAR-AH.
LUNAR-AH is a project in collaboration with Synthetic Genomics to develop infectious disease prophylactic vaccines.