RARE Daily

Arcturus Therapeutics Raises $70 Million to Advance mRNA Medicines Pipeline

April 16, 2020

Rare Daily Staff

Arcturus Therapeutics has raised $70 million in a public offering of 4.1 million shares of its common stock priced at $17 a share. The company will use the funding for working capital and to advance its mRNA therapies for rare diseases and vaccines. 

Arcturus has a number of mRNA medicine development programs that leverage its proprietary delivery system and mRNA design and production capabilities. It’s lead clinical candidate is an experimental mRNA therapeutic to treat ornithine transcarbamylase (OTC) deficiency, a urea cycle disorder in which a lack of the OTC enzyme in liver cells results in high blood ammonia levels and can cause seizures, coma, and death in untreated patients. There is currently no cure for OTC deficiency.

Other candidates include a recent grant program with the Cystic Fibrosis Foundation to develop a novel mRNA therapeutic candidate to treat cystic fibrosis, and R&D programs targeting glycogen storage disease type 3, hepatitis B, non-alcoholic steatohepatitis (NASH), and a self-replicating mRNA vaccine for SARS-CoV-2.

Arcturus’ RNA platform can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics.

In addition to its collaboration with the Cystic Fibrosis Foundation, Arcturus has struck deals to use its mRNA capabilities to discover and develop RNA therapeutics with several pharmaceutical and biotech companies including Janssen Pharmaceuticals, Ultragenyx Pharmaceutical, Takeda Pharmaceutical, CureVac, and Synthetic Genomics.


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