-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
http://stm.sciencemag.org/content/4/121/121ra18
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…]
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Patent Protects Use of UNAs in Multiple Nucleic Acid Constructs Including siRNAs, microRNA Mimics, Antagomirs and Single-Stranded Constructs
BOTHELL, 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…]
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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 potential of Silence’s proprietary DBTC delivery system with miRagen’s novel microRNA- (miRNA-) based therapeutics. MiRagen is a pre-clinical stage biopharmaceutical company founded to develop innovative miRNA-based therapeutics for the treatment of cardiovascular and muscle disease.
Under the terms of the agreement, miRagen will provide Silence with specific miRNA sequences, which Silence will formulate with its proprietary DBTC delivery system in order to develop multiple candidate drugs. MiRagen will undertake in vitro and in vivo studies of the candidate drugs developed under the agreement and select lead candidates for further evaluation. Financial terms of the collaboration are not disclosed.
DBTC is a proprietary RNAi delivery system developed by Silence. It is a novel lipid-based formulation that functionally delivers short interfering RNA (siRNA) to liver endothelial cells, hepatocytes and other liver cell types with high efficiency.
Thomas Christely, Chief Executive Officer of Silence Therapeutics, said: “We are delighted to be collaborating with miRagen. This is the third collaboration that [click to continue…]
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-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 of glioblastoma multiforme (GBM), the most common and aggressive brain tumor in humans. Regulus will apply its expertise in microRNA therapeutics to discover chemically modified oligonucleotide anti-miRs for testing at the Samsung Medical Center in preclinical models that mimic human brain cancer. Accelerate Brain Cancer Cure (ABC2), a non-profit organization dedicated to accelerating therapies for brain cancer patients, has awarded Regulus a grant to support the research.
“GBM is a devastating disease with limited treatment options,” said Neil W. Gibson, Ph.D. Chief Scientific Officer of Regulus Therapeutics. “At Regulus, we have successfully targeted microRNAs in multiple disease settings and believe that targeting dysregulated microRNAs using [click to continue…]
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- 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 of the cost
- Miragen to receive regulatory and commercial milestones of approximately $352 million and double-digit royalties on product sales in markets outside the U.S. and Japan
- Total deal value approximately $1 billion including development support and payments to Miragen
SURESNES, France, and BOULDER, Colo., October 18, 2011 – Les Laboratoires Servier, a leading European pharmaceutical company with expertise in innovative treatments for cardiovascular diseases, and Miragen Therapeutics, Inc., a preclinical-stage biopharmaceutical company focused on improving patients’ lives by developing innovative microRNA (miRNA)-based therapeutics for cardiovascular and muscle disease, announced today an agreement for advancing the research, development and commercialization of three drug candidates, including two of miRagen’s lead programs (miR-208 and miR-15/195) and one additional target yet to be identified, for cardiovascular disease. This partnership provides worldwide rights, excluding the U.S. and Japan, to Servier.
Under the terms of the agreement, Miragen will receive up to $45 million in total upfront, research support and near-term milestone payments over the next three years, as well as royalties on sales, based on the successful outcome of the collaboration. Additional clinical and commercial milestones, as well as clinical development support for the successful development of the three compounds, would value the deal at approximately $1 billion. Miragen and Servier will collaborate on the research and development effort, while Servier alone will be responsible for all costs associated with the global development, regulatory approval and commercialization of the three product candidates [click to continue…]
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