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
HOERSHOLM, 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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by Christoph on February 11, 2013
in News
Dr. Davide Marini, co-founder and Chief Executive Officer of Firefly BioWorks announced today the creation of the Firefly Frontiers Grant:
“We are excited to provide financial support to researchers for expanding the horizons of microRNA research. We encourage applicants to explore high-impact questions and those high-risk explorations that would not be normally funded through a traditional academic route”.
The Grant
The Firefly Frontiers Grant is a $10,000 award in products and services to support exploratory studies in the area of microRNAresearch. It is open to principal investigators, postdoctoral and graduate students in academic and non-profit research institutions. The company’s flagship product, FirePlex™ miRSelect, enables high-throughput and targeted microRNA profiling on a flow cytometer. FirePlex™ miRSelect scales easily [click to continue…]
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A phosphate switch to fine-tune the protein production in the cells:
Fast-Forward Genetics Identifies Plant CPL Phosphatases as Regulators of miRNA Processing Factor HYL1
MicroRNAs are essential regulators of the genetic program in multicellular organisms. Because of their potent effects, the production of these small regulators has itself to be tightly controlled. That is the key finding of a new study performed by Tübingen scientists at the Max Planck Institute for Developmental Biology. They identified a new component that modulates the production of micro RNAs in thale cress, Arabidopsis thaliana, by the removal of phosphate residues from a micro RNA-biogenesis enzyme. This can be as quick as the turn of a switch, allowing the plant to adapt to changing conditions. In this study, the scientists combined advanced imaging for facile detection of plants with defective microRNA activity with whole genome sequencing for rapid identification of new mutations.
The cell seems to thwart itself: Reading the DNA, a mobile messenger RNA is produced in the cell nucleus, exported to the cytoplasm where it serves as a blueprint for the production of proteins. At the same time, the cell is able to produce micro RNAs that, by binding to specific messenger RNAs, can block protein production or even initiate its destruction. But why does the cell start a costly process and immediately stops it? “Well, the answer lies on the fine balance the cell has to achieve between [click to continue…]
Title: Recent Work & Current Methods in Cancer microRNA Research – The 2nd in a series of microRNA Webinars presented by LC Sciences
Date: Wed 7/11/12
Please register for the webinar by selecting the presentation time that is most convenient for your timezone.
North America – 1:00 Central Daylight Time
Europe – 1:00 PM Greenwich Mean Time
Far East – 10:00 AM China Standard Time (GMT +8hrs)
Webinar Outline
- Brief review of microRNA basics: history, biogenesis, function
- Recent developments of microRNA research in the field of cancer genomics
- Current methods for microRNA discovery and profiling
- Case studies and application examples
After registering, you will receive a confirmation email containing information about joining the webinar.
After completion of the webinar, you will receive an email containing a link to the archived webinar.
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In this week’s issue of Nature , two researchers at the Cancer Biology and Genetics Program, Memorial Sloan-Kettering Cancer Center have published a review discussing the diverse contribution microRNA networks exhibit in cancer biology. Lujambio et. al. discuss both the oncogenic and tumor-suppressor roles microRNA have been attributed in the initiation and progression of multiple cancers. Discussion also focuses on microRNA as a driving/initiation factor in tumor biology and provides a thorough review of the dynamic between several microRNA genes and the TP53 tumor suppressor. The mouse model system is highlighted as a very useful tool validating and investigating the in vivo role specific microRNA contribute to tumor development and risk. The review concludes with a discussion concerning the potential roles of RNAi technology and its use as a therapeutic tool in cancer treatment.
The authors present insights into several areas of microRNA cancer research that requires further investigation in the near future, including: the impact of expression variation of several components of the microRNA biogenesis machinery, the direct role of microRNA epigenetic regulation of chromosomal DNA, and designing effective and safe microRNA drug-delivery systems. These three areas are highlighted by the authors as important steps into elucidating the total impact and use of microRNA biology in cancer development and treatment. Undoubtedly, further research in these areas will greatly enhance what is already know about microRNA regulation in cancer biology and may provide novel avenues for drug therapies.
Lujambio, A. and S.W. Lowe, The microcosmos of cancer.
Nature. 482(7385): p. 347-55.
http://www.ncbi.nlm.nih.gov/pubmed?term=the%20microcosmos%20of%20cancer
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