Dicerna Announces Publication of Data Demonstrating Tumor Growth Inhibition from Knockdown of Key Oncogene Using Dicer Substrate RNAi Compounds

Dicerna Announces Publication of Data Demonstrating Tumor Growth Inhibition from Knockdown of Key Oncogene Using Dicer Substrate RNAi Compounds
Paper Highlights Ability of Dicerna's DsiRNA Molecules and EnCore™ Delivery Technology to Silence Key Genes in Hepatocellular Carcinoma
October 09, 2013 08:40 AM Eastern Daylight Time
WATERTOWN, Mass.--(BUSINESS WIRE)--Dicerna Pharmaceuticals, Inc., an emerging pharmaceutical company developing RNA interference (RNAi)-based therapeutics against genetically-defined targets in multiple disease areas, announced today the publication of a paper in Molecular Therapy: "Knockdown of β-catenin with Dicer Substrate siRNAs Reduces Liver Tumor Burden in Vivo." The data presented in Molecular Therapy demonstrate the potential therapeutic benefit achievable through the silencing of key oncogenes utilizing Dicerna's unique approach to RNA interference.

"These data provide important proof of concept for Dicerna's approach, through DsiRNAs, to silence traditionally undruggable cancer targets"

"These data provide important proof of concept for Dicerna's approach, through DsiRNAs, to silence traditionally undruggable cancer targets," commented Bob D. Brown, Ph.D., Chief Scientific Officer at Dicerna. "Challenges to realizing the potential of RNAi in oncology have long included the identification of the appropriate genes to target and achieving their sufficient knockdown in tumors. The paper demonstrates Dicerna's ability to overcome those challenges and to develop product candidates with high in vivo activity in established animal oncology models."

Dicerna has identified a series of oncogene targets to disrupt using its EnCore delivery system. β-catenin is a target of high value given its role in several difficult to treat tumors and its nature as a traditionally undruggable protein target. The Company has previously announced similar success with the KRAS and MYC oncogenes. Dicerna's most advanced oncology program, DCR-M1711, targets the MYC oncogene and is slated to enter clinical studies in 2014. These newly published results provide further data regarding Dicerna's ability to successfully target high value, formerly intractable oncogenes, utilizing DsiRNA in conjunction with the EnCore delivery system.

The data were published in the online Molecular Therapy cited HERE.

About Dicer Substrate RNAi (DsiRNA)

Dicerna's DsiRNA molecules are designed to target and silence genes that lead to serious diseases. DsiRNAs are uniquely structured and different from first generation siRNAs. Specifically, the company's DsiRNA molecules are 25 or more base pairs in length and are processed by the Dicer enzyme. The ability to engage the Dicer enzyme, which operates early in the gene silencing cascade, provides functional benefits over conventional, shorter siRNA molecules.

About Dicerna

Dicerna is a private, venture-backed biopharmaceutical company harnessing RNA interference (RNAi) to develop breakthrough, targeted therapeutics. Utilizing its Dicer Substrate Technology™ platform and EnCore™ delivery technology, Dicerna is developing a pipeline of programs to reach previously undruggable intracellular disease targets.

The company has collaborations and alliances with global pharmaceutical companies and will continue to build on this momentum to advance its research and development efforts. Dicerna is based in Watertown, Mass. For more information, please visit www.dicerna.com.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward looking statements, including statements regarding the future of Dicerna and its potential pharmaceutical products and systems, that are subject to significant risks and uncertainty. Dicerna is an early stage biopharmaceutical company with all of its potential drug candidates in laboratory or pre-clinical studies. Most early stage drug candidates fail to become commercially available drugs due to, among other reasons, clinical failure, regulatory problems, or the inability to raise the significant funds required for drug development. RNAi is a novel area of therapeutics and there are no approved RNAi-based drugs. As an early stage company, Dicerna incurs and expects to continue to incur significant operating losses.

Contacts
Dicerna Pharmaceuticals
Jim Weissman, 617-612-6214
Chief Business Officer
or
MacDougall Biomedical Communications
Chris Erdman, 781-235-3060

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