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RaNA Therapeutics, Inc. and Santaris Pharma A/S announce agreement to develop RNA-targeted medicines that selectively activate



RaNA Therapeutics, Inc. and Santaris Pharma A/S announce agreement to develop
     RNA-targeted medicines that selectively activate protein expression

  PR Newswire

  HOERSHOLM, Denmark, SAN DIEGO and CAMBRIDGE, Massachusetts, July 8, 2013

-- RaNA Therapeutics licenses rights to utilize Santaris Pharma A/S
proprietary Locked Nucleic Acid (LNA) Drug Platform to discover and develop
RNA-targeted medicines that selectively activate protein expression against up
to 10 of RaNA's proprietary RNA targets for the treatment of human diseases

-- RaNA will apply single-stranded LNA-based technology to develop drug
candidates in the fields of rare genetic disorders, inflammation, oncology,
metabolic diseases and neurodegenerative diseases

HOERSHOLM, Denmark, SAN DIEGO and CAMBRIDGE, Massachusetts, July 8, 2013
/PRNewswire/ -- Santaris Pharma A/S and RaNA Therapeutics, Inc. announced
today that they have signed an agreement whereby RaNA gains access to
Santaris' Locked Nucleic Acid (LNA) technology to develop RNA-targeted
medicines. Under the terms of the agreement, Santaris will license its
proprietary Locked Nucleic Acid drug technology to RaNA for the development of
LNA-based drug candidates against up to 10 RaNA proprietary RNA targets. The
license granted by Santaris includes access to proprietary chemistry capable
of delivering potent single-stranded LNA-based drug compounds. RaNA will apply
the LNA chemistry along with its proprietary technologies for selective
protein activation to develop RNA-targeted medicines. Santaris will receive an
upfront payment and is eligible for target nomination payments, development
milestones and royalties on each product.

(Logo: http://photos.prnewswire.com/prnh/20100111/SPLOGO )

Henrik Oerum, CSO & VP Business Development of Santaris Pharma A/S, said
"Santaris Pharma's LNA drug platform provides the means to discover and
develop novel drugs that potently and specifically bind RNA-targets in
previously inaccessible clinical pathways. Usually, the objective is to
inhibit the target to avoid protein synthesis, but in RaNA's case the LNA
drugs will be developed to selectively activate protein expression. As such,
our agreement with RaNA is yet another example of how we seek to leverage the
broad applicability of our drug platform through partnerships with the
pharmaceutical industry."

"We are delighted to gain access to the Santaris LNA chemistry to augment our
drug development programs," said Arthur M. Krieg, MD, President and CEO of
RaNA Therapeutics. "We believe the LNA chemistry has important properties to
contribute to RaNA's proprietary approach to selectively express therapeutic
proteins."

About Santaris Pharma A/S Santaris Pharma A/S is a privately held
clinical-stage biopharmaceutical company focused on the discovery and
development of RNA-targeted therapies. The LNA Drug Platform and Drug
Discovery Engine developed by Santaris Pharma A/S combine the company's
proprietary LNA chemistries with its highly specialized and targeted drug
development capabilities to rapidly deliver potent single-stranded LNA-based
drug candidates across a multitude of disease states. The company's research
and development activities focus on infectious diseases and cardiometabolic
disorders, while partnerships with major pharmaceutical companies include a
range of therapeutic areas including cancer, cardiovascular disease,
infectious and inflammatory diseases, and rare genetic disorders. The company
has strategic partnerships with Bristol-Myers Squibb, miRagen Therapeutics,
Shire PLC, Pfizer, GlaxoSmithKline, and Enzon Pharmaceuticals. As part of its
broad patent estate, the company holds exclusive worldwide rights to
manufacture, have manufactured and sell products that contain LNA as an active
ingredient for studies performed with a view to obtaining marketing approval.
Santaris Pharma A/S, founded in 2003, is headquartered in Denmark with
operations in the United States. Please visit www.santaris.com for more
information.

About RaNA Therapeutics, Inc. RaNA Therapeutics is pioneering the discovery of
a new class of medicines that target RNA to selectively activate protein
expression within the body's own cells. RaNA's novel drugs work by targeting
specific genes to precisely upregulate the expression of beneficial proteins.
The company's leadership leverages preeminent founders and advisors in the
field, and the strength of its fundamental patents, technology, and know-how
that underlie the discovery and development of therapeutics that selectively
activate RNA. RaNA is applying its proprietary technology to rapidly advance a
pipeline of novel drugs for diverse therapeutic areas, with an initial
emphasis in the fields of rare genetic disorders, inflammation, oncology,
metabolic diseases and neurodegenerative diseases. For more information about
the company and its drug platform, please visit www.ranarx.com .

Website: http://www.santaris.com
Contact: Santaris, Henrik Stage, President and CEO, Cell: +45 4026 0900,
e-mail: hs@santaris.com, Henrik Oerum, CSO & VP Business Development, Cell:
+45 2834 6474, e-mail: hoe@santaris.com, RaNA Therapeutics, Art Krieg,
President and CEO, Office: 857-242-3421, e-mail: akrieg@ranarx.com, Rene
Castro, Chief Business Officer, Office: 857-242-3501, e-mail:
rcastro@ranarx.com
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