New Data Published in Nature Genetics Demonstrate That Tiny LNA-Based Compounds Developed by Santaris Pharma A/S Inhibit Entire Disease-Associated microRNA FamiliesBy Santaris Pharma As, PRNE
Sunday, March 20, 2011
Tiny Locked Nucleic Acid (LNA) based compounds, which are 8-mer LNA oligonucleotides, successfully inhibit entire microRNA families, providing potential new approach for treating a variety of diseases, including cancer, viral infections, cardiovascular and muscle diseases
HOERSHOLM, Denmark and SAN DIEGO, March 21, 2011 - A study published online in this week's Nature Genetics demonstrates that
tiny Locked Nucleic Acid (LNA)-based compounds developed by Santaris Pharma
A/S can inhibit entire disease-associated microRNA families. This provides a
potential new approach for treating a variety of diseases including cancer,
viral infections, cardiovascular and muscle diseases (1).
Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused
on the research and development of mRNA and microRNA targeted therapies,
developed the tiny LNA-based compounds, which are 8-mer LNA oligonucleotides,
using its proprietary LNA Drug Platform. The high affinity and target
specificity of tiny LNA-based compounds enabled functional inhibition of both
single microRNAs and entire microRNA families in a range of tissues in vivo
without off-target effects.
MicroRNAs have emerged as an important class of small regulatory RNAs
encoded in the genome. They act to control the expression of sets of genes
and entire pathways and are thus thought of as master regulators of gene
expression associated with many diseases. Because they dictate the expression
of fundamental regulatory pathways, microRNAs represent potential drug
targets in the treatment of many disease processes.
"Using tiny LNA-based compounds to successfully inhibit entire
disease-associated microRNA families provides a new range of opportunities to
develop novel microRNA-targeted drugs for both in-house drug discovery
programs, as well as with our partners," said Henrik Oerum, Ph.D., Vice
President and Chief Scientific Officer of Santaris Pharma A/S. "The
versatility of our proprietary LNA Drug Platform has the potential to develop
new modalities to target a broad range of diseases, including cardiometabolic
disorders, infectious and inflammatory diseases, and cancer by targeting
microRNAs, entire microRNA families or messengerRNAs."
The study published in Nature Genetics was carried out by Santaris Pharma
A/S scientists and collaborators at Cold Spring Harbor Laboratory, New York.
In this study, scientists demonstrated that tiny LNA-based compounds
inhibited both single microRNAs and entire microRNA families in cultured
cells, as well as in vivo in several mice tissues and in a mouse breast tumor
model. The tiny LNA-based compounds were well tolerated by the mice and could
be delivered without the use of complex delivery vehicles.
The Santaris Pharma A/S LNA Drug Platform is the only RNA technology with
both mRNA and microRNA targeted drugs in clinical trials, demonstrating the
broad utility of the proprietary platform. In September 2010, Santaris Pharma
A/S successfully advanced miravirsen, a lead microRNA drug candidate
targeting miR-122, into Phase 2 studies for the treatment of patients
infected with the Hepatitis C virus. In addition, Santaris Pharma A/S is
advancing two mRNA-targeted drugs, SPC5001 targeting PCSK9 and SPC4955
targeting apoB, for the treatment of high cholesterol into Phase 1 in the
first half of 2011.
Santaris Pharma A/S also has a robust product pipeline with its partners
consisting of mRNA and microRNA drug discovery and development
collaborations. These include partnerships with Pfizer, Inc. (delivery of
lead candidates against up to 20 targets), miRagen Therapeutics
(cardiovascular diseases), Shire plc (rare genetic disorders),
GlaxoSmithKline (four viral disease drug candidates) and Enzon
Pharmaceuticals (eight cancer targets successfully delivered - three are now
in Phase 1 clinical studies).
About Locked Nucleic Acid (LNA) Drug Platform
The LNA Drug Platform and Drug Discovery Engine developed by Santaris
Pharma A/S combines the Company's proprietary LNA chemistry with its highly
specialized and targeted drug development capabilities to rapidly deliver
LNA-based drug candidates against RNA targets, both mRNA and microRNA, for a
range of diseases including cardiometabolic disorders, infectious and
inflammatory diseases, cancer and rare genetic disorders. LNA-based drugs are
a promising new class of therapeutics that are enabling scientists to develop
drug candidates to work through previously inaccessible clinical pathways.
The LNA Drug Platform overcomes the limitations of earlier antisense and
siRNA technologies to deliver potent single-stranded LNA-based drug
candidates across a multitude of disease states. The unique combination of
small size and very high affinity allows this new class of drugs candidates
to potently and specifically inhibit RNA targets in many different tissues
without the need for complex delivery vehicles. The most important features
of LNA-based drugs include excellent specificity providing optimal targeting;
increased affinity to targets providing improved potency; and favorable
pharmacokinetic and tissue-penetrating properties that allow systemic
delivery of these drugs without complex and potentially troublesome delivery
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 Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery Engine
developed by Santaris Pharma A/S combine the Company's proprietary LNA
chemistry 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 miRagen Therapeutics, Shire plc, Pfizer,
GlaxoSmithKline, and Enzon Pharmaceuticals. As part of its broad patent
estate, the Company holds exclusive worldwide rights to all therapeutic uses
of LNA. 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.
(1) Obad, dos Santos, Petri, Heidenblad, Broom, Ruse, Fu, Lindow,
Stenvang, Straarup, Hansen, Koch, Pappin, Hannon and Kauppinen. 2011.
Silencing of microRNA families by seed-targeting tiny LNAs. Nature Genetics
Navjot Rai, Director, Global Communications of Santaris Pharma A/S, office, +1-858-764-7064, ext. 206, cell, +1-619-723-5450, navjot.rai at santaris.com
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