Arcturus Therapeutics

Arcturus Therapeutics develops novel ribonucleic acid (RNA) therapeutics focused on rare diseases. Its therapeutics are based on messenger RNA (mRNA), small interfering RNA (siRNA), gene editing RNA, DNA, antisense oligonucleotides, and microRNA.

The company’s proprietary lipid nanoparticle delivery system LUNAR can safely deliver therapeutic RNA or DNA to target cells inside a patient’s body. It also enables the direct delivery of gene-editing tools into target cells, facilitating the editing of disease-causing mutations. Arcturus also uses a technology, called STARR (Self-Transcribing and Replicating RNA) Technology, to combine self-replicating RNA with its proprietary LUNAR nanoparticle delivery system. 

As of March 2024, the company has a pipeline of drug candidates for mRNA medicines for the treatment of rare diseases, such as ornithine transcarbamylase deficiency and respiratory diseases, such as cystic fibrosis. The lead program of the company is hepatic LUNAR-OTC in Phase II, a treatment for ornithine transcarbamylase deficiency, a life-threatening genetic disease. The company additionally has five partner programs signed with CSL and the Biomedical Advanced Research and Development Authority (BARDA) for the development of therapeutics targeting Covid-19 and influenza. The most advanced partner program is its "ARCT-2303" candidate being developed in partnership with CSL for the treatment of Covid-19, the project is in Phase 3 of clinical testing.

Key customers and partnerships

Arcturus Therapeutics expanded its partnership with the Cystic Fibrosis Foundation in September 2023 (initially entered in May 2017) to advance the respiratory LUNAR-CF program, a potential treatment for cystic fibrosis. 

Funding and financials

Arcturus Therapeutics last raised funding in September 2023, when the company received USD 9 million in funding from The Cystic Fibrosis Foundation. The funding was provided so that Arcturus Therapeutics would to test an inhaled messenger RNA (mRNA) therapy that could treat the underlying cause of cystic fibrosis in all people living with the disease, including those with two copies of rare and nonsense mutations.