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RNAi therapeutics: addressing targets?

2 August 2008 | By

Gene silencing by RNA interference (RNAi) uses double-stranded RNA to shut down gene expression in cells. This provides the possibility that this new methodology could be used in the treatment of disease symptoms and disease processes. A particular attraction of RNAi (as well as other gene knockdown methods of treatment,…

Personalised medicine: are we ready for the revolution?

19 March 2008 | By

The impact of biomarker technology and biomarker strategies in pharmaceutical development is still in its infancy; but the impact is already proving significant. Biomarker strategy forms the basis for personalised medicine, the industry/regulatory focus centres on improving the success rate and reducing the high attrition rate often encountered in early…

The expanding world of small RNA: from germ cells to cancer

21 September 2007 | By Eric A. Miska, The Wellcome Trust/Cancer Research UK Gurdon Institute and Department of Biochemistry, University of Cambridge, Cambridge, UK

Over the last ten years a small RNA revolution has swept biology. In 1998 RNA interference (RNAi) was discovered as an experimental tool by Andy Fire and Craig Mello, a finding that was awarded with the 2006 Nobel Prize for Physiology or Medicine. Although the biology of RNAi is still…

In vivo drug target validation using RNAi

21 July 2007 | By Jost Seibler, Head of Technology Development, Artemis Pharmaceuticals and Frieder Schwenk, Principal Scientist, University of Applied Science, Department of Applied Natural Sciences, Gelsenkirchen, Germany

Among the genetic model organisms, the laboratory mouse (Mus musculus) has a predominant role in the study of human disease and in pre-clinical drug development. Apart from the high degree of sequence homology of mouse and human genomes, and similarities in many physiological aspects, advanced targeting technologies make the crucial…

How will MicroRNAs affect the drug discovery landscape?

21 July 2007 | By Dr. Neil Clarke and Dr. Mark Edbrooke, GlaxoSmithKline Research and Development, Hertfordshire, UK

The archetypal microRNAs, lin-4 and let-7, were discovered in the nematode worm Caenorhabditis elegans over a decade ago and, at that time, no one would have predicted that they would be anything other than an interesting feature of worm developmental biology. However, in recent years there has been an explosion…

RNAi: an attractive choice for future therapeutics

23 May 2007 | By John J. Rossi, Division of Molecular Biology, Beckman Research Institute of the City of Hope, Graduate School of Biological Sciences, Duarte, United States

RNA interference (RNAi) is a regulatory mechanism of most eukaryotic cells that uses small double stranded RNA (dsRNA) molecules as triggers to direct homology-dependent control of gene activity (Almeida and Allshire 2005).

Biomarker discovery and validation in clinical proteomics

23 May 2007 | By Professor Stephen R Pennington, Proteome Research Centre, Conway Institute of Biomolecular and Biomedical Sciences, University College Dublin

Until recently the use of proteomics in the biomedical arena has included programmes aimed at the elucidation of cellular responses to extracellular stimuli by known and potential drugs. It has been anticipated that these will lead to the elucidation of the basic mechanisms of cellular responses, potential identification of new…

Biomarkers in neurodegenerative diseases

27 March 2007 | By Claudio Carini, MD,PhD,FRCPath, F. VP of Translational Medicine, MDS Pharma

Biomarkers are useful characteristics to evaluate disease progress and targets of therapeutic agents. They are objectively measured and obtained by non-invasive procedures collecting readily accessible matrixes (Blood, CSF). Biomarkers should be easy to detect, specific and reproducible. Most importantly when detected early in the course of a disease they should…

Designing a program for early CNS development

28 November 2006 | By Irina Antonijevic, Douglas Craig and Christophe Gerald, Lundbeck Research USA, Inc.

Only a few innovations have been made in recent decades with regard to psychiatric, and particularly antidepressant, drugs (Insel et al., 2006) (Figure 1). This conundrum reflects, at least partly, the lack of understanding of the disease biology. This poses a challenge not only to inventive drug development, but also…

Biomarkers of efficacy and safety

22 August 2005 | By Dr Graham R Betton, Senior Principal Scientist, Safety Assessment, AstraZeneca Pharmaceuticals

This article reviews the types of biomarkers currently available and approaches to discovering new biomarkers.

The promise of pancreatic beta cells

20 May 2005 | By William Carley, Ph.D., Experimental Medicine, Pfizer Global R&D

Biomarker application during drug development is driven by the need to define disease and therapeutic efficacy/safety earlier, better and with reduced cost.

Applications in drug development

7 March 2005 | By Thorir D. Bjornsson, MD, PhD, Translational Development, Wyeth Research

The past decade has witnessed a growing interest in biomarkers, previously referred to as pharmacodynamic markers, PD markers, or pharmacologic read-outs. This increasing interest has been largely driven by evolutionary changes in drug discovery and development and in regulatory science1,2,3. One key driver has involved the increasing need to reach…

Who nose?

7 March 2005 | By Professor Richard Dalby and Yang Guo, University of Maryland School of Pharmacy, Julie Suman, Next Breath LLC

In 2003 worldwide sales of allergic rhinitis nasal sprays alone were valued at US$2.4B (IMS, Valois), up from 2002 worldwide sales of all over-the-counter (OTC) nasal sprays except steroids, estimated at US$820M (IMS, Valois). That’s a significant amount of drug administered via the nose every year (Figure 1). With a…

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