Nanalyze

RNAi Developer Dicerna Pharmaceuticals to IPO

RNA Interference (RNAi) is a new approach in the development of specific targeted therapies against both rare diseases and cancer. Historically, the pharmaceutical industry has developed two fundamental classes of drugs that can address well-defined targets: small molecules and antibodies. Both of these drug classes have been very successful but are limited in the nature of the targets they can inhibit. RNAi offers the potential to overcome the key limitations of small molecules and antibodies by attacking targets such as transcription factors that are expressed exclusively inside cells. One company that is developing RNAi drugs and recently filed for IPO is Dicerna Pharmaceuticals.

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About
Founded in 2007, Massachusetts based Dicerna Pharmaceuticals has raised over $110 million in venture capital funding from the likes of Skyline Ventures, Omega Funds, Deerfield Capital, Oxford Bioscience Partners, Brookside Capital, and others. Dicerna has $54.7 million cash on hand with an accumulated deficit so far of $78.7 million. As of November 30, 2013, Dicerna’s patent estate included approximately 16 issued patents and approximately 64 pending patent applications licensed from City of Hope including the core DsiRNA patent (U.S. 8,084,599).  Dicenra’s co-founder CEO, Douglas M. Fambrough III, Ph.D., was a lead venture capital investor and board member of Sirna Therapeutics, an early RNAi company that was acquired by Merck & Co., Inc. in 2006 for $1.1 billion.

Pipeline
Dicerna has developed EnCore™ lipid nanoparticles, a proprietary and effective system for the delivery of DsiRNAs to the liver and to solid tumors. These delivery particles are highly potent, have low toxicity, and are amenable to manufacturing at scale. Dicerna’ is also developing Direct Targeted DsiRNAs to enable the targeted delivery of RNA interference sequences without the need for conventional lipid nanoparticles. Dicerna has partnered two of their oncology development programs with the global pharmaceutical company Kyowa Hakko Kirin Co., Ltd. Dicerna’s drug development pipeline can be seen below:
Dicerna_Pipeline

To date, the Company’s revenue has been primarily derived from their research collaboration and license agreement with KHK in which they have received total payments of $17.5 million. For each product candidate in their collaboration with KHK, Dicerna has the potential to receive clinical, regulatory and commercialization milestone payments of up to $110.0 million and royalties on net sales of each such product candidate. The Company expects to initiate clinical trials for DCR-M1711 in the first half of 2014 and for DCR-PH1 in 2015.

The Company has a number of competitors listed in their S-1 filing, most notably Arrowhead Research Coporation (NASDAQ:ARWR) which has substantially similar access to the same DsiRNA  patent rights granted to Dicerna with their City of Hope licenses. Arrowhead is developing RNA-based therapeutics for the treatment of diseases of the liver, which may directly compete with Dicerna’s product candidates. Dicerna aims to trade on the NASDAQ under the symbol “DRNA.”

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