Ziel
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
Wissenschaftliches Gebiet
- natural sciencesbiological sciencesbiochemistrybiomoleculesnucleic acids
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- natural sciencesbiological sciencesgeneticsDNA
- medical and health scienceshealth sciencesinfectious diseases
- natural sciencesbiological sciencesgeneticsRNA
Programm/Programme
Aufforderung zur Vorschlagseinreichung
FP6-2005-LIFESCIHEALTH-7
Andere Projekte für diesen Aufruf anzeigen
Finanzierungsplan
STREP - Specific Targeted Research ProjectKoordinator
STOCKHOLM
Schweden