Pharma

RNAi firm raises $5M to develop and deliver drugs that turn off disease-causing genes

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 […]

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.

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A Deep-dive Into Specialty Pharma

A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.

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.