Dicerna Pharmaceuticals

Dicerna Pharmaceuticals is an emerging pharmaceutical company developing RNA interference (RNAi)-based therapeutics against genetically-defined targets in multiple disease areas, including cancer. Dicerna’s promising therapeutic approach utilizes its patented Dicer Substrate siRNA (DsiRNA™) molecules and EnCore™ drug delivery technologies to silence previously undruggable disease targets, offering a potential new treatment option for patients.

RNAi is a naturally occurring biological process utilized by cells to regulate and silence gene expression at the RNA level. RNAi has potency that goes far beyond the potency of traditional drug technologies, enabling the effective use of oligonucleotide-based, gene-directed drugs in oncology for the first time. In addition, RNAi is gene specific, allowing maximal therapeutic effect and minimal off-target side effects.

Dicerna delivers its DsiRNA molecules to cancer cells using its EnCore lipid nanoparticle drug delivery technology. EnCore has been specifically engineered to accumulate in tumors and mediate efficient delivery of the DsiRNAs to the RNAi machinery in the cancer cell cytoplasm. Encore particles are well tolerated and manufactured using well-established unit operations to ensure commercial scalability.

Using DsiRNAs against key driver oncogenes, delivered via EnCore, Dicerna has achieved strong anti-tumor efficacy in multiple preclinical models. Dicerna’s lead program, DCR-M1711 is focused on Hepatocellular Carcinoma (HCC) and is directed at the MYC oncogene, a key regulator of oncogenic pathways that has been implicated in multiple cancer types. Dicerna expects to enter human clinical trials with its lead program in early 2014. The company is advancing its other preclinical programs in development.