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Disease therapeutic targets, multidisciplinary parallel validation, accelerated drug discovery

Objective

Altered gene expression or function in disease indicates disturbed cellular processes and potential new molecular targets for therapeutic intervention. The drug development pipeline however, is plagued by high target attrition rates and extended timelines. Further, advances in genomics have not yet delivered on the promise of a healthcare revolution, even in the context of a completed human genome sequence. Inadequate capabilities to validate prospective targets and leads for functional and especially medical relevance, is a plausible explanation for this failure. In addition, R & D pipelines do not efficiently combine powerful technology platforms. When multiple technologies are combined, it is often with insufficient accompanying expertise so that potential for improvement in drug target development timelines is not met. The PONT consortium will optimize and link together state-of-the-art technology platforms with associated expertise for co-development of drug target candidates by 3 SMEs and 1 larger company. Highly disease deregulated genes identified in human tissues will be interrogated. Parallel informatic analysis will select target subsets with properties consistent with those expected for effective and safe therapeutics. In an innovative parallel drug development approach that also includes 2 academic partners, selected targets will be examined for protein expression profiles with human tissue microarrays, protein structures determined at atomic resolution, functions evaluated with in vitro and in vivo models, and small molecules binding specifically to targets identified with a label-free binding assay. Leads will be optimized with structural analysis of targets complexed with specific binding molecules. Potential aptamer and antibody therapeutics are also built-in to the proposal. Combination of these technologies creates a unique multidisciplinary meta-platform that will reduce early stage drug development times by two years or more.

Call for proposal

FP6-2002-LIFESCIHEALTH
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Coordinator

ORIDIS BIOMED FORSCHUNGS-UND ENTWICKLUNGSGMBH
EU contribution
No data
Address
Elisabethstrasse, 84
GRAZ
Austria

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Total cost
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Participants (5)