Biomolecular Medicine and Advanced therapies- focus on delivery of RNA therapeutics – Research group Samir EL Andaloussi

Our research efforts focus on one of the greatest challenges facing modern therapeutics - delivery. Despite the unprecedented knowledge of diseases and their mechanisms due to advances in biomedical sciences, many promising therapeutic approaches are still clinically unavailable. This is simply because there is no efficient means of delivering these therapeutics to the right organ with the right dose.

Our research

Our research efforts focus on one of the greatest challenges facing modern therapeutics; delivery. Despite the unprecedented knowledge of diseases and their mechanisms due to advances in biomedical sciences; many promising therapeutic approaches are still clinically unavailable. This is simply because there is no efficient means of delivering these therapeutics to the right organ with the right dose.

Such therapeutics include proteins for replacement therapy or antibodies for anti-inflammatory treatment as well as nucleic acids for gene therapy of diseases including muscular dystrophies and neurodegenerative disorders. We develop innovative drug delivery technologies that are able to carry protein and/or gene therapies to the target tissues safely and efficiently. We have developed a promising technology based on reprogramming exosomes, which are vesicles that are naturally used by cells to communicate, to carry therapeutic proteins and nucleic acids. We engineer exosomes with enhanced delivery and targeting capacities and screen our wide array of exosome designs in disease-relevant cellular and animal models. We are also developing methods for large-scale production and purification of such exosome therapeutics. Additionally, our lab develops methods for gene therapy based on modified cell-penetrating peptides that are efficient carriers of nucleic acids and proteins across biological barriers. We test our different technologies in models of inflammatory diseases as well as neurodegenerative and neuromuscular disorders. Our main aim is to one day unlock the potential of protein and gene therapy of such diseases by the development of novel and efficient delivery technologies.

DELIVER project

In the ERC Consolidator project DELIVER, we propose a platform development using synthetic nanocarriers to transiently engineer hepatic cells in vivo and harness extracellular vesicles to functionally deliver drugs to unreachable organs, focusing on the brain. Genetic constructs will be developed that allow for transient in situ engineering of cells in vivo and release of cargo-laden extracellular vesicles for further delivery. The same strategy will be exploited using nanoparticles injected locally in brain to secrete extracellular vesicles loaded with the disease-relevant proteins as a treatment strategy for Parkinson’s disease. Long-term this novel project has enormous potential, as any engineered extracellular vesicles could be produced in situ.

External funding

Swedish Medical Research Council, SSF-IRC FormulaEx, H2020 EXPERT, KI central faculty funding, Swedish Society of Medical Research, Vinnova, ONO Pharma, KID funding, EvoxTherapeutics, US Army.

Research in media

Publications

Selected publications

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