CNS delivery of complex biopharmaceuticals (CRISPR/Cas9)

Lead Research Organisation: University College London
Department Name: School of Pharmacy

Abstract

Complex biopharmaceuticals include large proteins, antibodies, nucleic acids derived therapies and combinations thereof. These types of therapies are of increasing importance due to the highly specific nature of their disease modifying interactions. The majority of brain conditions remain ill served by pharmacological treatments compared to many other organs due to the due to presence of the blood-brain-barrier. The CRISPR-Cas9 gene editing system has shown immense potential for the genetic treatment of disease due to its specific ability to edit cellular genes (Jain et al. 2019; Xu et al. 2019). The ability to treat CNS diseases at the cellular level in a highly specific and potentially permanent manner would bring a dramatic improvement. However, CRISPR-Cas9 is a multi-component system (i.e. having a protein and RNA component) with unfavourable properties for in-vivo delivery, making it a prime example of complex pharmaceuticals which require the novel biomaterials engineering approaches to start to address these challenges systematically. We will first engineer novel polymer-based biomaterials to allow an inert and reversible encapsulation of complex biopharmaceuticals; the next stage we will build upon this basis to add specific functional components to enhance intracellular delivery and facilitate delivery to the CNS.

Publications

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Studentship Projects

Project Reference Relationship Related To Start End Student Name
EP/S023054/1 01/10/2019 31/03/2028
2236204 Studentship EP/S023054/1 23/09/2019 21/09/2023 Omar Adjani Mokrane