-Regulus Earns $2.5 Million Milestone Payment from AstraZeneca-

-RG-125 (AZD4076) is Regulus’ 3rd Clinical Candidate, Achieving Key ‘Clinical Map Initiative’ Goal for 2015-

regulus therapeutics logoLA JOLLA, Calif., April 7, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today the selection of RG-125 (AZD4076), a GalNAc-conjugated anti-miR targeting microRNA-103/107 (“miR-103/107″) for the treatment of Non Alcoholic Steatohepatitis (“NASH) in patients with type 2 diabetes/pre-diabetes, as a clinical candidate by AstraZeneca under the companies’ strategic alliance to discover, develop and commercialize microRNA therapeutics.  RG-125 (AZD4076) is the first compound from the alliance to be selected for clinical development by AstraZeneca. In connection with the candidate selection, AstraZeneca will pay Regulus $2.5 million and will assume development of the program following acceptance of an Investigational New Drug application.  In the near term, Regulus and AstraZeneca plan to submit key preclinical data on the RG-125 (AZD4076) program to be presented at a scientific meeting later this year and expect to initiate a Phase I study of RG-125 (AZD4076) in humans by the end of 2015.

“Regulus is very pleased that AstraZeneca has chosen to advance a microRNA therapeutic candidate from this exciting program toward the clinic.  RG-125 acts as a novel insulin sensitizer which we believe may inform a differentiated development path to treat patients with complicated metabolic disorders,” said Neil W. Gibson, Ph.D., Chief Scientific Officer of Regulus.  “RG-125 represents our third clinical development candidate to arise from our novel technologies in less than two years, which confirms the productivity of our platform, achieves a key goal under our ‘Clinical Map Initiative’, and underscores our leadership in the microRNA therapeutics field.”

Marcus Schindler, Head of Cardiovascular and Metabolic Diseases, Innovative Medicines and Early Development, AstraZeneca, said: “This is a tremendous achievement for our collaboration and an exciting step for AstraZeneca to be [click to continue…]


Regulus Receives Orphan Medicinal Product Designation from the European Commission for RG-012, a microRNA Therapeutic in Development for the Treatment of Alport Syndrome

regulus therapeutics logoLA JOLLA, Calif., March 25, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that the European Commission has granted orphan medicinal product designation for RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 (“miR-21″) for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy. In July 2014, the U.S. Food & Drug Administration granted orphan drug designation to RG-012 for the treatment of Alport syndrome.

“We are pleased to have received orphan medicinal product designation in the European Union for RG-012, a key microRNA therapeutic program under our ‘Clinical Map Initiative’,” said Paul Grint, M.D., Chief Medical Officer of Regulus. “Alport syndrome is a life threatening disease and patients have very limited treatment options because there is currently no approved therapy.  We believe that RG-012 represents an opportunity to make a significant impact in the lives of patients with Alport syndrome and we look forward to advancing this program into the clinic.”

Regulus is currently enrolling patients in a natural history of disease study called ATHENA to gather information about the changes in renal function over time in patients with Alport syndrome.  Data from [click to continue…]


Atheroprotective miRNA Signaling Pathways

by Doug Dluzen on January 22, 2015

in Publications

Atheroprotective miRNA-Signaling Figure 5

MicroRNA (miRNA) are continually being identified as essential regulators of gene expression in a wide variety of different cellular contexts. A study published in March of 2012 in Nature Cell Biology1 identified an atheroprotective miRNA signaling pathway in which the miR-143/145 gene cluster is upregulated in endothelial cells and transferred to underlying smooth muscle cells (SMCs) via extracellular vesicles. Increased sheer stress of endothelial cells in vitro induced miR-143/145 gene expression via the transcription factor KLF2. In turn, miR-143/145 were packaged into extracellular vesicles that signaled to nearby SMCs. miRs 143 and 145 targeted and repressed several genes in SMCs, including ELK1 and KLF4, in an atheroprotective manner. Taking these studies a step farther, ApoE-/- mice were used to study the in vivo development of atherosclerotic [click to continue…]


Abcam acquires FireflyBio - LogosCambridge UK, 21 January, 2015: Abcam plc, a global leader in the supply of life science research tools, today announces that it has entered into a definitive agreement to acquire the entire issued share capital of Firefly BioWorks Inc (“Firefly”) (“the Acquisition”) on a cash-free/debt-free basis for £18.5m ($28.0m). The consideration, which is payable in cash on completion, will be funded from Abcam’s existing resources [1].

Firefly has developed a novel multiplex assay platform for the detection of biomarkers, based on a microfabrication technology developed by the founders at Massachusetts Institute of Technology and has developed its first product for detection of microRNAs (miRNAs). Firefly has 15 employees and is based in Cambridge, Massachusetts.

The Acquisition fits well with Abcam’s strategy outlined in September to establish new growth platforms and supports the Company’s mission to enable scientists to discover more. Firefly will strengthen [click to continue…]


Regulus Announces Key Goals Under its ‘Clinical Map Initiative’ for 2015

Accelerates RG-101 for HCV with Dual-Track Clinical Development Strategy; Top-Line, Single Dose, 4 mg/kg Results as Well as 2mg/kg

Extended Follow Up Results from Ongoing Study to be Reported in Early February 2015

Regulus Therapeutics LogoLA JOLLA, Calif., Jan. 8, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced key goals for 2015 under its ‘Clinical Map Initiative’ to advance its microRNA therapeutics portfolio and biomarkers platform.

“Regulus enters 2015 with the scientific and financial strength to realize the transformative potential of microRNAs.  As such, we’ve set aggressive goals for the year focused on creating a clear path to value for what we believe to be our greatest opportunities,” said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of Regulus.  “Under our ‘Clinical Map Initiative’, we are focusing our near term efforts on accelerating RG-101 for HCV with a Phase II dual-track clinical development strategy, while advancing our overall therapeutics pipeline and aligning our biomarker efforts to streamline our clinical development decisions.”

Key Goals Under Regulus’ ‘Clinical Map Initiative’ for 2015

  • ‘Clinical Map’ of RG-101 for HCV Defined: Dual-Track Strategy Accelerates Phase II Development; Multiple Data Read-Outs in 2015.  Following the favorable interim results reported inOctober 2014 from its ongoing clinical study, Regulus has accelerated development of RG-101, a wholly-owned, GalNAc-conjugated anti-miR targeting microRNA-122 (“miR-122″) for the treatment of HCV.  Regulus is pursuing a Phase II dual-track development strategy (i) to investigate RG-101 in combination with oral agents to potentially shorten treatment durations, optimize clinical outcomes and potentially improve responses in certain underserved HCV patient populations; and (ii) to investigate RG-101 further as a single agent to determine [click to continue…]


SomaGenics Receives $2,275,000 in NIH Funding to Develop its RNAi Therapeutics and microRNA Technologies

December 18, 2014

SANTA CRUZ, Calif., Dec. 18, 2014 /PRNewswire/ — SomaGenics has recently been awarded $2,275,000 in Small Business Innovation Research (SBIR) grants from the NIH to further develop and expand its RNA-based technologies in the areas of wound healing, hepatitis delta virus (HDV) therapeutics, microRNA (miRNA) expression profiling, and next-generation sequencing. SomaGenics’ wound healing program is […]

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Novel Biomarkers for Drug-induced Liver Injury

December 12, 2014

MicroRNAs have emerged as promising biomarkers of organ damage including injury of the liver. In this study the new generation of high-throughput sequencing technology was applied to detect circulating  miRNAs in serum from patients with accidental acetaminophen overdose and identified a set of 33 known miRNAs and 3 novel miRNA-like small RNAs that are functionally […]

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miRWalk 2.0 available

December 2, 2014

miRWalk2.0 (http://zmf.umm.uni-heidelberg.de/apps/zmf/mirwalk2/) which is a significantly improved and upgraded version of miRWalk database is now available for the scientific community. miRWalk2.0 was developed with the aim of providing a regularly updated, freely accessible, comprehensive archive to supply the biggest available collection of predicted and experimentally verified miRNA-target interactions with various novel and unique features to […]

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Upcoming Webinar – Recent Work & Current Methods for Profiling Extracellular microRNAs

September 23, 2014

Date: Tuesday Sept 30, 2014 In the past, differential profiling and functional studies of microRNA (miRNA) has been performed mainly using tissues samples collected by invasive methods. However, in a clinical setting, more convenient and non-invasive methods are required, such as collection of peripheral blood or other bodily fluids. Recently, it has been demonstrated that […]

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Implication of Sperm RNA in Transgenerational Inheritance of the Effects of Early Trauma in Mice

June 4, 2014

Recent studies have suggested that early-life stressors and traumatic experiences can cause heritable changes in gene expression in future generations. Researchers at the University of Zurich’s Brain Research Institute hypothesized that early-life stressors in male mice may cause heritable changes in the small, non-coding RNA content (including miRNAs) in the sperm of traumatized mice and […]

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