Skip to main content

Design of targeted gene pharmaceutics using self-assembling functional entities

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

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

Participants (4)

SYDDANSK UNIVERSITET
Denmark
Address
Campusvej 55
Odense
EUROGENTEC SA
Belgium
Address
Liege Science Park Rue Bois Saint-jean 5
Seraing
BIOSPACE MESURES
France
Address
10 Rue Mercoeur
Paris
AVARIS AB
Sweden
Address
C/o Karolinska Innovations
Stockholm