mirna therapeutics

New Nature Reviews Drug Discovery publication in the Series “RNA-based therapies”:

MicroRNA therapeutics: towards a new era for the management of cancer and other diseases

Summary of the key steps in the development of miRNA therapeutics.Abstract: In just over two decades since the discovery of the first microRNA (miRNA), the field of miRNA biology has expanded considerably. Insights into the roles of miRNAs in development and disease, particularly in cancer, have made miRNAs attractive tools and targets for novel therapeutic approaches. Functional studies have confirmed that miRNA dysregulation is causal in many cases of cancer, with miRNAs acting as tumour suppressors or oncogenes (oncomiRs), and miRNA mimics and molecules targeted at miRNAs (antimiRs) have shown promise in preclinical development. Several miRNA-targeted therapeutics have reached clinical development, including a mimic of the tumour suppressor miRNA miR-34, which reached phase I clinical trials for treating cancer, and antimiRs targeted at miR-122, which reached phase II trials for treating hepatitis. In this article, we describe recent advances in our understanding of miRNAs in cancer and in other diseases and provide an overview of current miRNA therapeutics in the clinic. The authors also discuss the challenge of identifying the most efficacious therapeutic candidates and provide a perspective on achieving safe and targeted delivery of miRNA therapeutics.

Rupaimoole R, Slack FJ.
MicroRNA therapeutics: towards a new era for the management of cancer and other diseases. Nat Rev Drug Discov. 2017 Mar;16(3):203-222.
doi: 10.1038/nrd.2016.246. Review. PubMed PMID: 28209991.

Full article: http://www.nature.com/nrd/journal/v16/n3/full/nrd.2016.246.html



Series D is Mirna’s Largest Financing to Date With Ten Institutional and Strategic Investors

miRNA Therapeutics Inc. LogoAUSTIN, TX–(Marketwired – April 30, 2015) – Mirna Therapeutics (Mirna), a private, clinical-stage biopharmaceutical and immuno-oncology company focused on the development of microRNA-based cancer therapeutics, today announced the completion of a $41.8 million Series D financing. The company’s second institutional financing was led by Baxter Ventures, joined by other new investors, Eastern Capital, Santé Ventures, Morningside Ventures, Rock Springs Capital, and Celgene Corporation. Existing investors Sofinnova Ventures, New Enterprise Associates, Pfizer Ventures, Osage University Partners, Correlation Ventures, and others, also participated in the financing. The funding will enable Mirna to advance its lead microRNA (miRNA) therapeutic product candidate, MRX34, into Phase 1b and Phase 2 trials in 2016. Indications for further development will be selected at the completion of Mirna’s ongoing Phase 1 trial in patients with hepatocellular carcinoma, other solid tumors and hematological malignancies. The Company also plans to advance a second miRNA therapeutic candidate into clinical trials with the proceeds of this financing, as well as embark on a combination therapy development program.

“Mirna is the industry leader in microRNA Replacement Therapy and we are excited to have the opportunity to invest,” said Geeta Vemuri, Vice President and Head of Baxter Ventures. “The Company has generated [click to continue…]


Mirna Broadens Rights to Its Lead Cancer Therapeutic Candidate MRX34

Marina Biotech LogoBOSTON, MA and AUSTIN, TX–(Marketwired – Jan 2, 2014) – Marina Biotech, Inc. (PINKSHEETS: MRNA), a leading oligonucleotide-based drug discovery and development company, and Mirna Therapeutics, Inc. (Mirna), a privately-held biotechnology company pioneering microRNA (miRNA) replacement therapy for cancer, announced today that they have amended their license agreement regarding the development and commercialization of microRNA-based therapeutics utilizing Mirna’s proprietary microRNAs and Marina Biotech’s novel SMARTICLES liposomal delivery technology. Under terms of the amendment, Mirna paid certain pre-payments to Marina Biotech and now has additional rights to its lead program, MRX34, currently in Phase 1 clinical development for patients with unresectable primary liver cancer or metastatic cancer with liver involvement. In addition, under the terms of the license agreement, Mirna has optioned exclusivity on several additional miRNA targets. Further terms of the Agreement were not disclosed.

“Mirna and Marina Biotech have enjoyed a successful, durable collaboration by leveraging the systemic delivery of our proprietary, double-stranded, miRNA mimics with the SMARTICLES delivery technology,” said Paul Lammers, President and CEO of Mirna Therapeutics. “Amendment of the license agreement strengthens [click to continue…]


Expands the Company’s Patent Portfolio in Oncology Therapeutics

Rosetta Genomics LogoPRINCETON, NJ and REHOVOT, ISRAEL–(Marketwired – Nov 7, 2013) – Rosetta Genomics Ltd. (NASDAQ: ROSG), a leading developer and provider of microRNA-based molecular diagnostics, announces receipt of a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) related to U.S. Patent Application No. 13/481,105 titled “Targeting microRNAs for the Treatment of Liver Cancer.”

This allowance is for a therapeutic patent that protects a method for treating hepatocellular carcinoma (HCC), or liver cancer, and covers the administration of a compound comprising a modified oligonucleotide that is an anti-miR-222. In addition, various modifications of the nucleotide are claimed. There is an additional claim which refers to the reduction of the levels of serum alpha-fetoprotein or serum des-gamma-carboxyprothrombin in the subject to be treated.

“HCC is the fifth most common cancer in the world and the third leading cause of cancer deaths. Because standard chemotherapy usually has no beneficial outcome on HCC patients, there is significant need for new therapeutic modalities and novel therapeutic [click to continue…]


Regulatory Application for RG-101 to be Filed in the Near Term: Clinical Studies in Man Expected to Commence in Early 2014

regulus therapeutics logoLA JOLLA, Calif., Nov. 4, 2013 /PRNewswire/ — Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that data demonstrating the positive preclinical profile of RG-101 will be presented in a late-breaking poster session at the 64th Annual Meeting of the American Association for the Study of Liver Disease (AASLD) being held in Washington, D.C. on Monday, November 4, 2013 from8:00 a.m. Eastern Standard Time (EST) to 5:30 p.m. EST. The poster is available on the Company’s website at http://www.regulusrx.com.

“RG-101 utilizes a unique mechanism of action by targeting microRNA-122, a liver-specific host factor for stability, replication and translation of HCV.  We believe that therapies that target host-encoded factors essential for HCV replication may act as attractive combination agents because they may demonstrate activity across [click to continue…]


Microlin Bio Inc. Licenses The Ohio State University’s miRNA Patent Portfolio to Advance Cancer Diagnostics & Therapeutics

September 26, 2013

COLUMBUS, Ohio, and NEW YORK, Sept. 26, 2013 /PRNewswire/ — The Ohio State University today announced the signing of an exclusive world-wide agreement with Microlin Bio Inc., licensing a large portfolio of Ohio State’s groundbreaking cancer discoveries. The portfolio includes nearly 100 issued and pending microRNA patents that could lead to entirely new, more effective and more targeted ways to diagnose and treat […]

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miRagen Therapeutics: First Milestone in Strategic Alliance with Servier

May 23, 2013

Servier selects third cardiovascular target triggering the first milestone under the collaboration SURESNES, France, and BOULDER, Colo., May 23, 2013 – Servier, a privately-run French research-based pharmaceutical company and a major player in Europe and emerging markets having expertise in the development of treatments for cardiovascular diseases, and miRagen Therapeutics, Inc., a biopharmaceutical company developing […]

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Mirna Therapeutics is First to Advance MicroRNA into the Clinic for Cancer

May 15, 2013

Phase 1 Clinical Trial of Anticancer Drug MRX34 Underway AUSTIN, TX (May 13, 2013)– Mirna Therapeutics, Inc., a biotechnology company pioneering microRNA (miRNA) replacement therapies for cancer, announced today that it has initiated a Phase 1 clinical study of MRX34, the first miRNA to advance into a human clinical trial for cancer. The Phase 1 […]

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Santaris Pharma A/S and miRagen Therapeutics Inc. expand worldwide strategic alliance to discover and develop LNA-drugs against disease causing microRNAs

January 8, 2013

HOERSHOLM, Denmark and BOULDER, Colo., Jan. 8, 2013 /PRNewswire/ — Santaris Pharma A/S and miRagen Therapeutics, Inc. today announced that they have expanded their existing partnership in the field of microRNA therapeutics. Under the expanded agreement, miRagen obtains a broad non-exclusive license in the miRNA therapeutics field for therapeutics research and world-wide exclusive rights to research, […]

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Mirna Therapeutics Secures $34.5 Million Series C Financing to Advance Oncology microRNA Pipeline into the Clinic

October 24, 2012

Institutional Investment Is One of the Largest Ever for a Texas Biotechnology Company Austin, 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 […]

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