Arcturus rakes in $5M to back RNAi tech, nanoparticle delivery

San Diego RNAi specialist Arcturus Therapeutics pulled in $5 million this week to support its RNAi tech, which it acquired earlier this year from Marina Biotech ($MRNA), and its own nanoparticle delivery platform. The company expects to unveil its novel gene-silencing drugs with a candidate early next year.

Like many RNAi companies, Arcturus' technology is based around the silencing of gene expressions for diseases, although Arcturus is at a much earlier stage than some of the field's leaders, including Alnylam ($ALNY), which aims to have 5 treatments in clinical trials by 2015. But, according to CEO Joseph Payne, Arcturus' patent for its unlocked nucleobase analog (UNA), acquired from Marina for an undisclosed amount, has claims that are very broad in scale, including up to 40 nucleotides and acylic monomers, microRNA, siRNA and RNAi trigger techs, among others.

"We believe that once we go public with this early next year, it will command attention within the RNAi community, and the fact that we were able to achieve such potency at multi-doses gives us an edge," Payne told FierceDrugDelivery. "In the many years I've been in this field, I've never seen anything like it."

The UNA technology from Marina works with Arcturus' own lipid nanoparticle delivery system and is intended for the orphan disease market, the company says. It expects the "best-in-class" delivery tech will help expand its pipeline and bring RNA drugs into the mainstream. Its endosomal lipid technology, which is designed to disrupt the endosomal function of the cell, brings small bits of RNA (sRNA) into the cytoplasm, where it can then act on the specific part of the genetic code for a disease.

Founded in 2013, the company works out of Janssen Labs, owned by Johnson & Johnson ($JNJ). It pulled in $1.3 million in June seed funding before acquiring Marina's patents in August. As for the most recent cash, the $5 million series A round from private investors in Canada, the U.S. and Japan gives Arcturus enough to keep going through 2014 and get the candidate into the clinic by 2015.

"The focus is our lead program," Payne said of the company's plans for the funding. "We want to nominate a clinical candidate in 2014 and, in the very near term, hire some top-tier talent, including in business development."

RNAi drugs make up a field that Global Industry Analysts projects to reach over $4 billion by 2017, and Arcturus has high hopes for its product: "We plan to lead the next wave of RNAi technology," Payne said.

- here's the release