Dicerna Pharmaceuticals Announces Second DsiRNA Therapeutic Oncology Candidate, Achieves Development Milestone

  Dicerna Pharmaceuticals Announces Second DsiRNA Therapeutic Oncology
  Candidate, Achieves Development Milestone

          Company Receives Milestone Payment from Kyowa Hakko Kirin

Business Wire

WATERTOWN, Mass. -- August 08, 2012

Dicerna Pharmaceuticals Inc., a second generation RNA interference (RNAi)
company developing novel therapeutics utilizing its proprietary Dicer
Substrate Technology™ and Dicer Substrate siRNA (DsiRNA) molecules, announced
today that Kyowa Hakko Kirin Co., Ltd. (TSE: 4151) (KHK) has recognized the
successful achievement of an in vivo activity milestone in the development of
its second collaborative therapeutic oncology candidate. This achievement
triggers an undisclosed milestone payment to Dicerna.

Dicerna has a research collaboration and license agreement with KHK for the
research, development and commercialization of DsiRNA pharmaceuticals and drug
delivery systems for therapeutic targets in oncology. In December 2011,
Dicerna announced that their first oncology target was moving from the
research stage into development and that a second oncology program had been
initiated with an additional gene target.

“At the end of last year, we were very pleased to announce an important
validation of the in vivo efficacy of our DsiRNA technology ,” said Douglas M.
Fambrough, Ph.D., chief executive officer of Dicerna. “This work demonstrated
its significant potential in the development of cancer therapeutics by
targeting a gene that had been of field-wide interest for many years and
considered to be among the classical undruggable pharmacology targets. This
latest achievement shows the excellent progress that has been made in
targeting yet another oncology related gene. Our partnership with KHK has been
very fruitful, resulting in two promising announced programs and additional
programs in earlier stages.”

“We were particularly pleased with the speed with which this milestone was
reached for our second candidate,” said Etsuo Ohshima, Ph.D., managing officer
and vice president, head, research division at KHK. “It reflects the
collaborative activity of our respective teams, and it also represents a
unique feature of RNAi technology and its facility in bringing forward highly
potent therapeutic candidates. As has been the case throughout this
collaboration, this in vivo success was achieved utilizing Dicerna’s DsiRNA
molecule in combination with our proprietary drug delivery system.”

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
www.dicerna.com.

About Kyowa Hakko Kirin

Kyowa Hakko Kirin Co., Ltd. is a leading biopharmaceutical company in Japan.
It had a new start in October 2008 following the merger of Kyowa Hakko Kogyo
Co., Ltd. and Kirin Pharma Company, Limited, with the aim of becoming a global
specialty pharmaceutical company that creates innovative new drugs. Kyowa
Hakko Kirin is focusing its core business areas of oncology, nephrology and
immunology to create new value in the life sciences and to contribute to the
health and well-being of people around the world. For more information, visit
www.kyowa-kirin.co.jp/english/.

Contact:

Dicerna Pharmaceuticals
Douglas M. Fambrough, Ph.D., 617-612-6205
CEO
or
Pure Communications
Michele Rozen, 617-730-8284
 
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