Exosomes in neurological diseases
This project will utilize the unique expertise in CNS molecular movement biology and neuropathology developed at University of Southampton to explore the CNS biodistribution and potential CNS clinical applications of engineered exosomes developed by Codiak Biosciences. The project will clarify subcellular exosome molecular interactions in brain tissue and also study human neuropathology specimens for specific targets and biological pathways of interest to Codiak.
Biodistribution of Alnylam pharmaceutical RNAi Agents in Mouse brain
Alnylam pharmaceuticals has led the translation of RNAi (RNA interference) from Nobel Prize-winning discovery into an innovative, entirely new class of medicines. Founded in 2002 by a team of distinguished life sciences leaders, Alnylam’s vision is to harness the potential of RNAi therapeutics to transform the lives of people living with diseases for which there are limited or inadequate treatment options.
In collaboration with Alnylam we are working to assess the potential of custom designed siRNA’s as therapeutic agents for neurological diseases. We are administering siRNA’s intrathecally and characterising there biodistribution in different brain regions, cell types and subcellular compartments by assessing siRNA accumulation in neuron, microglia, astrocytes and oligodendrocytes, endothelial and vascular smooth muscle cells.
Targeted AAV therapy of CNS disorders
Alcyone therapeutics are mission-driven to overcome the challenges in CNS therapy development through applying precision CNS delivery & dosing technologies to novel gene therapy technologies. Alcyone’s pipeline currently includes 12 AAV (adeno-associated virus) gene therapy programs and four gene therapy platform technologies targeting severe CNS disorders
In collaboration with Alcyone we are working to assess the biodistribution of intrathecally administered adeno associated viruses within the central nervous system.