Funktionell leverans av nukleotid-baserade läkemedel

Research Project, 2017 – 2024

Nucleotide-based therapies promises a major paradigm shift in several therapeutic areas. However, the full potential of this novel approach is hampered by the lack of delivery vehicles efficiently transporting the therapeutic agent to the cellular sites where it exerts its biological/pharmacological function. This in turn is due to gaps in understanding the mechanisms which govern cellular uptake, transport and release of nucleotide drugs. The overall objective of the research in the proposed centre is to bridge this knowledge gap and develop novel nucleotide drug formulations with the potential of improving efficacy, safety and overall clinical utility of these therapies. This will be accomplished by merging a multidisciplinary team from industry and academia who in a concerted action will contribute novel formulations and hitherto unmatched analytical methods to help unravel the mode of action of novel nucleotide delivery vehicles. By taking a leading position in this emerging field, we foresee an impact on Swedish society that goes far beyond the benefit of the partners, including a new arena for education, entrepreneurial and standardisation activities. Long term this will enable the generation of safe and efficacious drug vehicle formulations for functional nucleotide based drug modalities, thus enhancing the Swedish Life Science sector’s long-term competitiveness as well as enabling development of novel therapeutics improving human health.