Dicerna Pharmaceuticals
Jan 12, 2015

Dicerna Announces Issuance of US Patent Providing Broad Coverage of DsiRNA-EX RNAi Payloads

Claims of US Patent No. 8,927,705 Cover Composition of Matter of Dicerna's Next-Generation Proprietary Payloads for RNAi Therapeutics and Self-Targeting Conjugates

WATERTOWN, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA), a leader in the development of RNAi therapeutics, today announced that the United States Patent and Trademark Office (USPTO) issued to Dicerna Patent No. 8,927,705, "Single Stranded Extended Dicer Substrate Agents and Methods for the Specific Inhibition of Gene Expression," on January 6, 2015.

The patent provides broad composition of matter claims that describe double-stranded RNA (dsRNA) molecules, called extended Dicer substrate short interfering RNAs (DsiRNA-EX), possessing single-stranded extensions of variable length on their 5' and/or 3' ends. The single-stranded features provide multiple functional attributes, enhancing the therapeutic potential of these DsiRNA-EX molecules and improving their chemical and biological properties. Notably, the extensions enable the development of subcutaneously delivered molecules that are self-targeting and possess enhanced biological stability. These and other favorable features are introduced into the dsRNA while the molecules nonetheless maintain extremely high RNAi activity. This issued patent complements previously issued US Patent No. 8,349,809, which describes the methods of use of such extension-containing dsRNA molecules. Dicerna will identify this RNAi platform as its DsiRNA-EX platform.

"These composition claims further expand the issued Dicerna patent portfolio and reinforce our unique position in the RNAi space," said Bob D. Brown, Ph.D., Dicerna's chief scientific officer and the inventor of the technology. "The DsiRNA-EX technology offers unique biological activities and pharmaceutical enhancements over older-generation siRNA technologies. The patent announced today allows Dicerna to apply our DsiRNA-EX technology to develop a wide range of therapies, carving out additional space in which Dicerna is free to operate while securing broad IP on these extended molecules."

Several of Dicerna's development programs already employ the DsiRNA-EX technology, which supports self-delivering molecules that can be administered both subcutaneously and by intravenous injection, as clinical circumstances dictate.

About Dicerna

Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company focused on the discovery and development of innovative treatments for rare, inherited diseases involving the liver and for cancers that are genetically defined. The company is using its proprietary RNA interference technology platform to build a broad pipeline in these therapeutic areas. In both rare diseases and oncology, Dicerna is pursuing targets that have been difficult to address using conventional approaches, but where connections between targets and diseases are well understood and documented. The company intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q filing and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

Westwicke Partners
Peter Vozzo, 443-213-0505
Alex Van Rees, 973-442-1555 ext. 111

Source: Dicerna Pharmaceuticals, Inc.

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