Navigation Links
New data published in Nature Genetics demonstrate that tiny LNA-based compounds developed by Santaris Pharma A/S inhibit entire disease-associated microRNA families
Date:3/20/2011

  • 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

  • Data show high affinity and target specificity of tiny LNA-based compounds enabled functional inhibition of entire microRNA families in a range of tissues without off-target effects

  • Tiny LNA-based compounds are well tolerated in preclinical studies and can be delivered without the use of complex delivery vehicles

  • Versatility of Santaris Pharma A/S proprietary LNA Drug Platform allows LNA-based drugs to inhibit single microRNA targets or entire microRNA families, opening up development of new modalities to target a broad range of diseases

Hoersholm, Denmark/San Diego, California, 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 rum, 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).


'/>"/>

Contact: Navjot Rai
navjot.rai@santaris.com
858-764-7064 x206
Edelman PR
Source:Eurekalert

Related biology news :

1. High school students paper published in prestigious college math journal
2. Results of the third school nutrition dietary assessment study published
3. Special issue Comparative Cognition in Context now published
4. Nutrigenomics -- developing personalized diets for disease prevention -- part 2 just published in OMICS
5. Major aspergillus genomics supplement published by journal Fungal Genetics and Biology
6. Ambati study published in PNAS
7. First comprehensive guidelines for managing medullary thyroid carcinoma published in Thyroid journal
8. Data published in the New England Journal of Medicine support use of raxibacumab (ABthrax) for the treatment of inhalation anthrax
9. University of Hawaii at Manoa professor published in science journal
10. New Report Just Published: World Solid-State, Fiber, Gas and Dye Lasers Market Report
11. Reviews of microbial gene language published in special issue of Trends in Microbiology
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/11/2017)... 11, 2017 Crossmatch®, a globally-recognized leader ... today announced that it has been awarded a ... Activity (IARPA) to develop next-generation Presentation Attack Detection ... "Innovation has been a driving force within Crossmatch ... allow us to innovate and develop new technologies ...
(Date:4/11/2017)... Florida , April 11, 2017 ... a security technology company, announces the appointment of independent Directors ... Bendheim to its Board of Directors, furthering the company,s ... ... of NXT-ID, we look forward to their guidance and benefiting ...
(Date:4/5/2017)... , April 5, 2017 Today HYPR ... that the server component of the HYPR platform is ... providing the end-to-end security architecture that empowers biometric authentication ... HYPR has already secured over 15 million users across ... manufacturers of connected home product suites and physical access ...
Breaking Biology News(10 mins):
(Date:7/27/2017)... N.C., July 27, 2017 United Therapeutics Corporation ... financial results for the second quarter ended June 30, ... second quarter total net revenues reached $445 million, ... Martine Rothblatt, Ph.D., United Therapeutics Chairman and Chief ... is well-positioned given the large and growing number ...
(Date:7/26/2017)... ... 26, 2017 , ... Franz Inc ., an early ... for Knowledge Graphs, today announced Gruff v7.0 , the industry’s leading Graph ... and graph experts the ability to visually build queries and explore connections as ...
(Date:7/26/2017)... ... July 25, 2017 , ... Proscia Inc. , a software solutions ... featured panelist at 2017 MedCity CONVERGE. His talk, “The Davids vs. the Cancer Goliath,” ... and emerging technology (AI, VR, Big Data) sectors are taking aim at cancer diagnosis ...
(Date:7/26/2017)... ... July 26, 2017 , ... ... complex problems we face every day. This unique capability combines high resolution ... chemists, and manufacturers to deliver unprecedented datasets for chemical analysis, quality control, and ...
Breaking Biology Technology: