RNAi Firm Raises Funds to Develop Drugs That Turn Off Disease-Causing Genes

First published on MedCityNews.com. Just a few years ago, several big companies abandoned their RNAi drug development efforts, finding it difficult to deliver drugs that use the RNAi method for silencing genes.

First published on MedCityNews.com. Just a few years ago, several big companies abandoned their RNAi drug development efforts, finding it difficult to deliver drugs that use the RNAi method for silencing genes. Recently, though, the market seems to be on the rebound.

A San Diego startup developing a siRNA drug delivery system for the orphan disease market is further evidence of that. To expand its team and work toward choosing a lead clinical candidate next year, Arcturus Therapeutics has just rounded up $5 million from angel investors in Canada, Japan and the United States.

RNA interference is a technique that uses small snippets of RNA to silence a cell’s expression of genes and viruses that cause disease. Arcturus is developing a lipid nanoparticle drug delivery system that leverages small interfering RNA, which can bind to specific messenger RNA molecules and increase or decrease their activity. It’s using that system to develop a pipeline of new therapeutics for orphan diseases, beginning with a rare liver disease.

Since its $1.3 million seed round closed early this summer, the company has licensed a portfolio of Marina Biotech’s unlocked nucleic acid technology.

Two former employees of Nitto Denko, Joseph Payne and Pad Chivukula, started Arcturus earlier this year. It’s one of 18 life sciences companies located at Janssen Labs, an innovation center in San Diego that provides startups access to wet labs and research and administrative equipment.

Global Industry Analysts estimates that the market for RNAi technologies, with leaders like Alnylam Pharmaceuticals and Silence Therapeutics, will reach $4 billion by 2017.