Silence Therapeutics Forms Scientific Advisory Board and Two New Research Collaborations to Further Strengthen Liver Pipeline

Silence Therapeutics Forms Scientific Advisory Board and Forms Two New Research Collaborations to Further Strengthen Liver Pipeline

Two world-leading KOLs to offer scientific perspectives and insight in liver disease

Silence Therapeutics plc (AIM: SLN) ("Silence" or "the Company"), a leading RNA interference (RNAi) therapeutics company, today announced the creation of a Scientific Advisory Board (SAB) with the appointment of two Key Opinion Leaders (KOLs) in the area of liver disease, Professor Dr. Dr. Detlef Schuppan, M.D., PhD., and Professor Dr. Massimo Pinzani, M.D., PhD., F.R.C.P. The SAB will provide scientific perspectives and strategic insight to enable Silence to leverage its world-leading gene silencing technologies AtuRNAi and its liver specific liposome delivery technology DBTC, in the liver disease arena. DBTC is a novel lipid-based formulation that functionally delivers siRNA to liver endothelial cells, hepatocytes and other liver cell types with high efficiency.

Professor Schuppan and Professor Pinzani will also collaborate with Silence in hepatocellular carcinoma (HCC) and liver fibrosis with the aim to produce a development pipeline at Silence that specifically targets these disease areas.

Professor Detlef Schuppan is a consultant Gastroenterologist and Hepatologist and Director of Molecular and Translational Medicine and full Professor of Medicine at Mainz University Medical Centre in Germany. He is also Professor of Medicine and Senior Visiting Scientist at Harvard Medical School. Professor Schuppan has been Principal and Co-Investigator in numerous clinical studies that deal with acute and chronic liver diseases, gastrointestinal (GI) cancers and with celiac disease and has published more than 350 Pubmed cited articles and 50 book chapters. He regularly speaks nationally and internationally on chronic liver disease, matrix metabolism, novel therapies for GI and liver cancers, and celiac disease, and has received numerous awards. He is member of the American Gastroenterological Association, American Association for the Study of Liver Disease, the European Association for the Study of Liver Disease, and the United European Gastroenterological Foundation.

Professor Massimo Pinzani is one of the thought leaders in chronic fibrogenic disorders and particularly hepatic fibrogenesis. He is currently Professor of Medicine and Gastroenterology at the University of Florence, Italy. He is also visiting Professor in the Division of Hepatology at Osaka City School of Medicine, Japan and Adjunct Professor of Gastroenterology and Hepatology at Case Western Reserve University. He has received international awards and grants for this research and is a Peer-Reviewer for a range of International Institutions and Academic Journals.

Commenting on the announcement Tony Sedgwick, Chief Executive Officer of Silence, said: "We are privileged to have the opportunity to collaborate closely with Professors Schuppan and Pinzani,who are world renowned thought-leaders in gastroenterology and hepatology, and we look forward to employing their expertise to identify novel targets for Silence.  Both KOLs haveextensive clinical and academic experience that will ensure that Silence is going to generate and develop an enhanced high quality pipeline in liver fibrosis and HCC by leveraging Silence' leading RNAi and delivery technologies."

Professor Schuppan commented: "Both the generation of highly effective siRNAs and the cell and organ specific siRNA delivery systems proprietary to Silence are most promising platforms to address currently untreatable or difficult-to-treat conditions of liver fibrosis/cirrhosis and HCC. At the same time these siRNA therapeutics are largely devoid of off-target effects and thus side effects. Moreover, synergistic siRNA combinations will be validated without increased side effects."

Professor Pinzani added: "Silence Therapeutics is characterised by a novel array of biotechnological platforms in the field of siRNA structure and delivery. The quality of the products has been achieved and consolidated by extensive exploitation over several years. The current applicability to human diseases, particularly liver diseases, is great and potentially leading to major advances."

Notes for editors

About Silence Therapeutics plc (

Silence Therapeutics plc (AIM: SLN) is a leading biotechnology company dedicated to the discovery, development and delivery of targeted, systemic RNA interference (RNAi) therapeutics for the treatment of serious diseases. Silence offers one of the most comprehensive short interfering RNA (siRNA) therapeutic platforms available today based on a strong intellectual property portfolio and large clinical safety database. Silence's clinical siRNA product pipeline is one of the broadest in the industry. The Company possesses multiple proprietary siRNA delivery technology platforms including AtuPLEX™, DACC and DBTC. AtuPLEX enables the broad functional delivery of siRNA molecules to targeted diseased tissues and cells, while increasing their bioavailability and intracellular uptake. The DACC delivery system allows functional delivery of siRNA molecules selectively to the lung endothelium with a long duration of target mRNA and protein knock-down. The DBTC delivery system enables functional delivery of siRNA molecules selectively to liver cells including hepatocytes. Additionally, the Company has a platform of novel siRNA molecules based around its AtuRNAi chemical modification technology, which provides a number of advantages over conventional siRNA molecules. Silence's unique RNAi assets also include structural features for RNAi molecules and specific design rules for increased potency and reduced off-target effects of siRNA sequences.

The Company's lead internal drug candidate is Atu027, a liposomal formulation in clinical development for systemic cancer indications and one of the most clinically advanced RNAi therapeutic candidates in the area of oncology. Atu027 incorporates two of the Company's technologies, AtuRNAi and AtuPLEX™. Silence is currently conducting an open-label, single-centre, dose-escalation Phase I study with Atu027 in patients with advanced solid tumors involving single, as well as repeated, intravenous administration. Encouraging interim safety and pharmacokinetic data were presented at the American Society of Clinical Oncology Annual Meeting in June 2011. The study is expected to be completed in the first half of 2012.

The Company's RNAi therapeutic platform has received key validation through multiple partnerships with pharmaceutical companies including AstraZeneca, Dainippon Sumitomo, Pfizer/Quark, and Novartis/Quark. Silence is actively pursuing the establishment of additional partnerships. Silence Therapeutics has operations in both Berlin and London.