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 (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- natural sciences biological sciences biochemistry biomolecules nucleic acids
- medical and health sciences medical biotechnology genetic engineering gene therapy
- natural sciences biological sciences genetics DNA
- medical and health sciences health sciences infectious diseases
- natural sciences biological sciences genetics RNA
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
FP6-2005-LIFESCIHEALTH-7
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Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Coordinator
STOCKHOLM
Sweden
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.