MicroRNA therapeutics: towards a new era for the management of cancer and other diseases
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
AUSTIN, 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…]
Regulatory Application for RG-101 to be Filed in the Near Term: Clinical Studies in Man Expected to Commence in Early 2014
LA 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…]