Objective During the last years, developments in genomics have enabled an unprecedented acquisition of new knowledge with relevance for human disease. In parallel, the biotech industry has invented novel ways of generating drugs in order to meet the demands from the healthcare sector. Here, we outline the development of a novel Gene Pharmaceutics prototype through translational research. This can be accomplished by the combined efforts of three research-intensive biotech companies together with two academic groups, representing state-of-the-art skills and proprietary technologies in complementary areas. These include nucleic acid analogue chemistry, short interfering RNA (siRNA) technology, gene transfer and gene therapy, as well as imaging instrument manufacturing, and methods for self-assembling supramolecular complexes. This will lead to the design of new self-assembling Gene Pharmaceutics and bringing them to the stage of clinical grade. The aim is to develop plasmid and siRNA-based drugs that target the liver, since this organ is affected in many disorders involving a genetic component. Three diseases have been selected for this purpose, hepatitis B and C infections and an inherited form of bleeding disorder, haemophilia A (FactorVIII deficiency). Moreover, many aspects of this platform technology are likely to be applicable to essentially any liver disorder and may also be transferable to other organs systems. Fully developed this technology could result in new, safer and more rational drugs. The objectives of the project: 1. Generation of new forms of DNA-binding synthetic compounds, which will serve as genetic glues 2. Enhancing linking chemistry for biologically active entities coupled to DNA binding compounds 3. Optimising the assembly of Gene Pharmaceutics by in vivo experimental studies 4. Developing standard operating procedures for the manufacturing and assembly of Gene Pharmaceutics according to good manufacturing procedure Fields of science natural sciencesbiological sciencesbiochemistrybiomoleculesnucleic acidsmedical and health sciencesmedical biotechnologygenetic engineeringgene therapynatural sciencesbiological sciencesgeneticsDNAmedical and health scienceshealth sciencesinfectious diseasesnatural sciencesbiological sciencesgeneticsRNA Programme(s) FP6-LIFESCIHEALTH - Life sciences, genomics and biotechnology for health: Thematic Priority 1 under the Focusing and Integrating Community Research programme 2002-2006. Topic(s) LSH-2005-1.2.1-3 - Rational and accelerated development of new, safer, more effective drugs including pharmacogenomics approaches Call for proposal FP6-2005-LIFESCIHEALTH-7 See other projects for this call Funding Scheme STREP - Specific Targeted Research Project Coordinator KAROLINSKA INSTITUTET Address Nobels väg 5 Stockholm Sweden See on map Links Website Opens in new window EU contribution € 0,00 Participants (4) Sort alphabetically Sort by EU Contribution Expand all Collapse all SYDDANSK UNIVERSITET Denmark EU contribution € 0,00 Address Campusvej 55 Odense See on map Links Website Opens in new window EUROGENTEC SA Belgium EU contribution € 0,00 Address Liege science park rue bois saint-jean 5 Seraing See on map Links Website Opens in new window BIOSPACE MESURES France EU contribution € 0,00 Address 10 rue mercoeur Paris See on map Links Website Opens in new window AVARIS AB Sweden EU contribution € 0,00 Address C/o karolinska innovations Stockholm See on map Links Website Opens in new window
