Dicerna Announces Patent Issuance for DsiRNAs Directed at the KRAS Gene, a Key Cancer Regulator

  Dicerna Announces Patent Issuance for DsiRNAs Directed at the KRAS Gene, a
  Key Cancer Regulator

 New Claims Expand Dicerna’s Patent Estate for the Dicer Substrate Technology

Business Wire

WATERTOWN, Mass. -- March 19, 2013

Dicerna Pharmaceuticals, Inc. (Dicerna), a second generation RNA interference
(RNAi) company developing novel therapeutics utilizing its proprietary Dicer
Substrate Technology™ and Dicer Substrate siRNA (DsiRNA) molecules, today
announced that the United States Patent and Trademark Office (USPTO) has
issued patent claims specific to double-stranded RNAs directed against the
important oncology gene target KRAS. U.S. patent 8,372,816, titled “Methods
and compositions for the specific inhibition of KRAS by asymmetric
double-stranded RNA” was recently issued to Dicerna. This issuance continues
the expansion of Dicerna’s patent portfolio around RNAi-related technology,
including the DsiRNA class defining claims of U.S. patent 8,084,599, titled
“Methods and compositions for the specific inhibition of gene expression by
double-stranded RNA.”

“We are extremely pleased the USPTO has granted these most recent patent
claims specific to the KRAS gene, further adding to our strong patent estate
and allowing for broad therapeutic use claims in the RNAi field,” said Douglas
Fambrough, chief executive officer of Dicerna. “The KRAS gene has been
recognized as a target of great therapeutic potential for decades, and yet has
remained undruggable. The successful application of Dicer Substrate Technology
against KRAS is a key advancement that could lead to important new
therapeutics for cancer treatment.”

On February 26, 2013, scientists from Kyowa Hakko Kirin Co., Ltd. (KHK)
presented a talk entitled “Development of Lipid Nanoparticle (LNP) Based
Delivery System of siRNA Targeting Extrahepatic Tumor,” at Asia TIDES:
Oligonucleotide and Peptide Research, Technology and Product Development. The
presentation highlighted in vivo tumor efficacy data obtained with DsiRNA
targeting the KRAS gene, one of the programs from the research and development
collaboration between Dicerna and KHK. The partners had previously announced
in December 2011 that KHK had elected to advance this first collaborative
therapeutic oncology candidate from the research stage into formal development
studies, triggering a $5 million milestone payment for Dicerna.

About Dicer Substrate RNAi

Dicer is a critical enzyme involved in the RNAi gene silencing cascade and
acts as the natural initiation point for this pathway by processing
double-stranded RNA so that it can be used for gene silencing. Dicer then
delivers these modified small RNA molecules to the mature gene silencing
complex. Dicerna’s synthetic Dicer Substrate siRNA (DsiRNA) molecules are 25
or more base pairs in length and are processed by Dicer. By utilizing this
distinct early entry point into the pathway, DsiRNA molecules have greater
potency and longer duration of action than other RNAi approaches. In addition,
DsiRNA molecules have enhanced delivery potential because their structure
creates a natural conjugation point for cellular targeting agents.

About Dicerna Pharmaceuticals

Dicerna Pharmaceuticals is a private, venture-backed RNAi-focused
biopharmaceutical company developing novel therapeutic agents and related drug
delivery systems in oncology and other disease areas based on its proprietary
Dicer Substrate Technology™ platform and Dicer Substrate siRNA (DsiRNA)
molecules. Dicer Substrate Technology™ is a second generation RNAi approach
that results in greater potency, longer duration of action and enhanced
delivery potential, differentiating it from other RNAi approaches. Dicerna has
a major alliance with Kyowa Hakko Kirin for DsiRNA pharmaceuticals and drug
delivery systems focused in oncology, immunology and inflammation. The company
also has a partnership with Ipsen to research and develop novel DsiRNA
therapeutics with targeted delivery in oncology and endocrinology. Dicerna is
based in Watertown, Massachusetts. For more information, please visit

About Kyowa Hakko Kirin

Kyowa Hakko Kirin is a leading biopharmaceutical company in Japan focusing on
its core business area of oncology, nephrology and immunology/allergy. Kyowa
Hakko Kirin leverages antibody-related leading-edge technologies to discover
and develop innovative new drugs aiming to become a global specialty
pharmaceutical company which contributes to the health and well-being of
people around the world. For more information, visit


Dicerna Pharmaceuticals
Jim Weissman, Chief Business Officer
Michele Rozen, 617-730-8284
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