RARE Daily

Arcturus Therapeutics Raises $173 Million to Advance mRNA Pipeline

July 29, 2020

Rare Daily Staff

Arcturus Therapeutics has raised $173 million in a public offering of 3.3 million shares of its common stock at $53 a share.

The company will use the funding for working capital and to advance its Covid-19 vaccine candidate, and preclinical mRNA medicines for liver and respiratory rare diseases into clinical trials. 

Arcturus’ stock price has tripled since April as the race for a vaccine to stem the global Covid-19 pandemic has heated up, and was boosted when Arcurus received a go-ahead in mid-July to begin a phase 1/2 trial of its mRNA vaccine candidate, which is partnered with Duke-NUS Medical School in Singapore.

Arcturus has a number of mRNA medicine development programs that leverage its LUNAR 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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