Research Published in the New England Journal of Medicine Demonstrates Marked and Long-Lasting Antiviral Activity Against HCV for Santaris Pharma A/S’ Miravirsen, the First MicroRNA-Targeted Drug to Enter Clinical Trials

Santaris Pharma LogoHOERSHOLM, Denmark and SAN DIEGO, March 27, 2013 /PRNewswire/ — Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, today announced the publication of study results online in the New England Journal of Medicine (NEJM). The publication highlights the potential benefits of miravirsen, a host-targeted, pan-HCV genotype anti-viral agent and the first microRNA-targeted drug to enter clinical trials for the treatment of Hepatitis C virus (HCV). In the study, miravirsen, given as a four-week monotherapy treatment, provided robust dose-dependent antiviral activity with a mean reduction of 2 to 3 logs from baseline in HCV RNA (log10 IU/mL). The effect was sustained well beyond the end of therapy.

Clinical data from the Phase 2a study demonstrated the following:

  • Miravirsen was safe, well tolerated and provided prolonged antiviral activity well after the last dose of miravirsen monotherapy (x5 weekly injections)
  • There were no signs of viral resistance
  • Adverse events were infrequent, mild and did not lead to study drug discontinuation
  • There were no dose limiting toxicities or discontinuations due to adverse events
  • Miravirsen was associated with dose-dependent reductions in [click to continue…]

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ORTHOPAEDIC RESEARCH SOCIETYROSEMONT, Ill., Jan. 28, 2013 /PRNewswire-USNewswire/ — Currently there is no effective treatment used in clinical practice for patients with an injured spinal cord.  However, a group of orthopaedic scientists have recently discovered that the administration of microRNA-210 could be an effective treatment for an injured spinal cord by promoting regeneration following injury.

Previously, microRNA-210 has been studied as an effective treatment for cancer and other diseases.  Scientists noted that there was an absence of this particular gene in cancerous tumors, but it was found in abundance in healthy tissue. By delivering what was missing directly to the diseased tumor, scientists were able to stop the progression of the disease.  Other scientists, however, noted that microRNA has the possibility to actually promote the growth of certain cancers.

Dr. Satoshi Ujigo from Hiroshima University in Japan and his colleagues applied this same theory to injured spinal cords hoping for similar results.  His work was recently [click to continue…]


Institutional Investment Is One of the Largest Ever for a Texas Biotechnology Company

miRNA Therapeutics Inc. LogoAustin, TX – October 24, 2012
Mirna Therapeutics Inc. (Mirna), a biopharmaceutical company focused on the development and commercialization of microRNA (miRNA) therapeutics in cancer, announced today the completion of a $34.5 million Series C financing.

The company’s first institutional financing was led by Sofinnova Ventures, with participation from new investors, New Enterprise Associates (NEA), Pfizer Ventures, Osage University Partners, Correlation Ventures, and existing investors. The funding will enable Mirna to advance its lead microRNA-based therapeutic product candidate MRX34 through human proof of concept studies. As a result of the financing, Michael Powell Ph.D., General Partner, Sofinnova Ventures, Ed Mathers, Partner at NEA, and Elaine Jones, Ph.D., Executive Director, Venture Capital-Worldwide Business Development, Pfizer Inc., will join the Board of Directors.

“This significant round of financing is an important validation of the therapeutic and commercial potential of our differentiated miRNA replacement approach and underscores the promise of our pipeline of miRNA-based product candidates,” said Paul Lammers, M.D., Mirna’s President and Chief Executive Officer. “Armed with a robust preclinical data package, Mirna is now [click to continue…]


-New study published in Science Translational Medicine demonstrates microRNA-21 contributes to fibrogenesis in the kidney
-Regulus, in partnership with Sanofi, developing novel anti-fibrotic therapies targeting microRNAs

B.N. Chau et al.
“MicroRNA-21 Promotes Fibrosis of the Kidney by Silencing Metabolic Pathways”
Sci Transl Med 15 February 2012:
Vol. 4, Issue 121, p. 121ra18
Sci. Transl. Med. DOI: 10.1126/scitranslmed.3003205

LA JOLLA, Calif., Feb. 16, 2012  /PRNewswire/ — Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that new preclinical data investigating the role of microRNA-21 (miR-21) in the treatment of kidney fibrosis has been published in the journal Science Translational Medicine. Regulus’ lead program for fibrosis targets miR-21, which is up-regulated in fibrotic tissues of humans. Previous preclinical studies by Regulus scientists and collaborators have shown that therapeutic oligonucleotides targeting miR-21 (anti-miR-21) can decrease fibrosis in preclinical models by reducing the expression of extracellular matrix proteins.  Despite the current burden of fibrosis-related human disease, there are few therapies that can specifically treat this devastating disease.

“We are pleased with the published results demonstrating that targeting miR-21 with proprietary anti-miR oligonucleotides is effective at preventing kidney fibrosis in preclinical models,” said Neil W. Gibson, Ph.D., Regulus’ Chief Scientific Officer.  “We plan to select an anti-miR-21 development candidate [click to continue…]


Patent Protects Use of UNAs in Multiple Nucleic Acid Constructs Including siRNAs, microRNA Mimics, Antagomirs and Single-Stranded Constructs

Marina Biotech LogoBOTHELL, WA–(Marketwire – Jan 23, 2012) – Marina Biotech, Inc. (NASDAQ: MRNAD), a leading oligonucleotide-based drug discovery and development company today announced that the Intellectual Property Office of New Zealand (IPONZ) has issued a Notice of Acceptance for patent application 580712. The claims broadly cover multiple sequence independent and length independent, nucleic acid constructs having one or more unlocked nucleobase analogs (UNAs). The nucleic acid constructs of the patent include both RISC and dicer length siRNAs, both microRNA mimetics and microRNA antagomirs as well as single-stranded oligonucleotides.

“The company continues to deliver on a patent strategy which expands and protects our broad oligonucleotide therapeutics platform,” stated J. Michael French, President and CEO at Marina Biotech. “This allowed patent is part of our global UNA patent portfolio providing broad and comprehensive protection for multiple, distinct UNA containing nucleic acid constructs all of which can modulate gene expression through distinct cellular mechanisms including RNAi, mRNA translational inhibition, steric blocking or microRNA pathways. This patent allowance reinforces our belief that we will continue to obtain patent protection for our UNA technology in other countries thereby strengthening the company’s intellectual property position for our broad oligonucleotide drug discovery platform.”

UNA are non-nucleotide, acyclic monomers which provide greater structural flexibility in a nucleic acid strand. Their value has been demonstrated in Marina Biotech’s proprietary UsiRNA constructs which are double-stranded small interfering RNA (siRNA) incorporating at least one UNA monomer and are distinct from the standard siRNA constructs used by others in the industry. UsiRNAs are specifically designed to provide greater specificity for RNAi-based therapeutics. Substitution with UNA [click to continue…]


Silence Therapeutics Signs Collaboration with miRagen Therapeutics to Evaluate Delivery of Novel microRNA-based Therapeutics

January 9, 2012

LONDON, January 9, 2012/PRNewswire-FirstCall/ — – Third collaboration to investigate the potential application of Silence’s proprietary RNAi delivery technologies in the development of novel microRNA-based therapeutics Silence Therapeutics plc (AIM: SLN) (“Silence”), a leading RNA interference (RNAi) therapeutics company, announces that it has signed an agreement with miRagen Therapeutics, Inc. (“miRagen”), to assess the delivery […]

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Regulus Therapeutics Initiates an Orphan and Rare Disease microRNA Therapeutic Effort in Brain Cancer

December 12, 2011

-Regulus, Accelerate Brain Cancer Cure (ABC2) and Samsung Medical Center to Advance microRNA Therapeutics for Glioblastoma – LA JOLLA, Calif., Dec. 12, 2011 /PRNewswire/ — Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced the initiation of a new discovery effort in microRNA therapeutics for the treatment […]

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Servier and Miragen Sign $352 Million Partnership Agreement for the Research, Development and Commercialization of MicroRNA-targeting Drugs for Cardiovascular Disease

October 18, 2011

Miragen retains all U.S. and Japanese commercialization rights; Servier gains rights to all other global markets Servier to fund multi-year research and development plan on various targets and all preclinical and clinical development costs through Phase II for the compounds Miragen retains option to co-sponsor Phase III or opt in after completion and refund part […]

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miRNAs’ Therapeutic Potential

March 31, 2010

Scientists Scrutinize Promising Molecules as Potential Drug Targets and Biomarkers Genetic Engineering News – Patricia F. Dimond, Ph.D. MicroRNAs (miRNAs) finely regulate gene expression and play an important role in various cellular processes, including cell growth, differentiation, proliferation, and apoptosis. To date, more than 5,000 of these endogenous, noncoding single-stranded RNAs have been identified. miRNAs […]

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Number of Companies working on RNAi Based Therapeutics is Exploding!

November 3, 2009

AiRNA Pharmaceuticals Allergan Alnylam Pharmaceuticals Benitec BioCandell Therapeutics Calando Pharmaceuticals Cenix BioScience Cequent Pharmaceuticals Copernicus Therapeutics Dicerna Pharmaceuticals Expression Genetics Corporation General Research Laboratory Intradigm Corporation MDRNA Mirna Therapeutics MiRagen Therapeutics Nitto Denko Technical Corp Opko Health Oxford BioMedica Polyplus Transfection Quark Pharmaceuticals Regulus Therapeutics RXi Therapeutics Santaris Pharma Senesco Technologies Silence Therapeutics Siranoamics siRNAsense […]

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