Genomics Therapy Company Secures $60M Venture Financing

(NIST.gov)

Dicerna Pharmaceuticals Inc., in Watertown, Massachusetts, closed its third round of venture financing after start-up with $60 million in additional capital from new and current investors. Dicerna is a six year-old biotechnology company developing therapies harnessing genomic material to silence genes causing diseases.

Dicerna’s technology is based on ribonucleic acid or RNA interference (RNAi), a natural process to silence the expression of genes causing disease. RNA is genetic material related to DNA that the body uses to transmit genetic information to cells and synthesize proteins. RNA interference targets specific genes, making it a potentionally powerful therapeutic technique, while minimizing damage to other genes, thus limiting side effects.

The company’s technology is based on research by its scientific co-founders John Rossi and Mark Behlke, who serve on Dicerna’s scientific advisory board. Their research highlighted the key role of an enzyme, known as the Dicer enzyme, that processes the double-stranded RNA, and serves as the starting point for gene silencing and an agent for organizing components of a functioning RNAi pathway. Rossi is a molecular geneticist with City of Hope medical center in Duarte, California, and Behlke is chief scientist at Integrated DNA Technologies in Coralville, Iowa, a collaborator with Dicerna.

To transmit RNAi molecules in the body, Dicerna is also developing a drug delivery mechanism using lipid nanoparticles. The company says this technique, brand-named EnCore, makes it possible to deliver the treatment directly to tumor tissue and promote the release and accumulation of therapeutics inside the cell. The EnCore delivery also promotes binding and internalization of the therapeutic with targeted cancer cells.

The company’s lead product, code-named DCR-M1711, is an RNAi therapy delivered by EnCore to treat hepatocellular carcinoma that accounts for the vast majority (85 to 90%) of liver cancers, and is the third leading cause of cancer deaths. DCR-M1711 targets the MYC gene that regulates pathways associated with multiple forms of cancer, including cancer of the liver. The company says preclinical studies show DCR-M1711 able to prevent growth of hepatocellular carcinoma tumors.

The financing round was led by new investor RA Capital, with participation by new investors Brookside Capital, Deerfield, and Omega Funds, as well as current institutional investors Abingworth Management, Domain Associates, Oxford Bioscience Partners, Skyline Ventures, and SR One. Dicerna says it expects to use the proceeds of the round to advance its current pipeline therapies into clinical studies.

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